A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 (XIRIUS)

December 22, 2023 updated by: Biogen

A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in participants with X-linked retinitis pigmentosa (XLRP).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
50

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • Manchester, United Kingdom
        • Research Site
      • Oxford, United Kingdom
        • Research Site
      • Southampton, United Kingdom
        • Research Site
  • United States
    • Florida
      • Gainesville, Florida, United States, 32607
        • Research Site
      • Miami, Florida, United States, 33136
        • Research Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Research Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Research Site
    • Texas
      • Dallas, Texas, United States, 75231
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

Part 1:

  • Participants with genetically confirmed diagnosis of XLRP (with RPGR mutation).
  • Participant with active disease clinically visible within the macular region in both eyes.

Part 2:

- Participant with mean total retinal sensitivity in the study eye as assessed by microperimetry ≥ 0.1 dB and ≤8 dB.

Key exclusion Criteria:

Parts 1 and 2:

  • Participant with history of amblyopia in either eye.
  • Participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks or received a gene/cell-based therapy at any time previously.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Part 1: BIIB112 Dose 1
Participants will receive a single Dose 1 of BIIB112 by sub-retinal injection on Day 0.
Biological: BIIB112
Administered as specified in the treatment arm.
Other Names:
  • AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 2
Participants will receive a single Dose 2 of BIIB112 by sub-retinal injection on Day 0.
Biological: BIIB112
Administered as specified in the treatment arm.
Other Names:
  • AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 3
Participants will receive a single Dose 3 of BIIB112 by sub-retinal injection on Day 0.
Biological: BIIB112
Administered as specified in the treatment arm.
Other Names:
  • AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 4
Participants will receive a single Dose 4 of BIIB112 by sub-retinal injection on Day 0.
Biological: BIIB112
Administered as specified in the treatment arm.
Other Names:
  • AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 5
Participants will receive a single Dose 5 of BIIB112 by sub-retinal injection on Day 0.
Biological: BIIB112
Administered as specified in the treatment arm.
Other Names:
  • AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 6
Participants will receive a single Dose 6 of BIIB112 by sub-retinal injection on Day 0.
Biological: BIIB112
Administered as specified in the treatment arm.
Other Names:
  • AAV8-RPGR
Experimental: Part 2: BIIB112 High Dose
Participants will receive a single high dose of BIIB112 by sub-retinal injection.
Biological: BIIB112
Administered as specified in the treatment arm.
Other Names:
  • AAV8-RPGR
Experimental: Part 2: BIIB112 Low Dose
Participants will receive a single low dose of BIIB112 by sub-retinal injection.
Biological: BIIB112
Administered as specified in the treatment arm.
Other Names:
  • AAV8-RPGR
No Intervention: Part 2: Untreated Group
Participants will receive no intervention to allow for a controlled comparison.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Part 1: Number of Participants With Dose-Limiting Toxicities (DLTs)
Time Frame: Up to Month 24
DLTs are defined as any of the following events considered to be related to study drug: Sustained decrease in best-corrected visual acuity (BCVA) of ≥30 letters on the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart compared to baseline (sustained is defined as lasting 48 hours or more until recovery, with recovery defined as visual acuity (VA) returning to within 10 letters of baseline VA. An exception is made for surgery-related events occurring in close temporal association {within <24 hours} of the surgery); Vitreous inflammation, vitritis (>Grade 3 using standardized Nussenblatt vitreous inflammation scale grading); Any clinically significant retinal damage observed that is not directly attributed to complications of surgery; Any clinically relevant suspected unexpected serious adverse reaction, with the exception of vision loss or vision threatening.
Up to Month 24
Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time Frame: Day 0 (surgery) in Part 1 of the study up to 24 months
An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. TEAEs are defined as the AEs starting or worsening on or after the day of the first surgery.
Day 0 (surgery) in Part 1 of the study up to 24 months
Part 2: Percentage of Study Eyes With ≥7 Decibels (dB) Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry
Time Frame: Month 12
MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0', or a positive integer. The point labelled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points out of the 16 central points.
Month 12
Part 2: Number of Participants With TEAEs
Time Frame: Day 0 (surgery) in Part 2 of the study up to 12 months
An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. TEAEs are defined as AEs starting on or after the day of the first surgery.
Day 0 (surgery) in Part 2 of the study up to 12 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Part 1: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0' or a positive integer. The point labelled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points out of the 16 central points.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 68 Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0' or a positive integer. The point labelled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in whole grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points of the grid as a whole (68 points).
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Mean Sensitivity of the 16 Central Loci Points Assessed by MAIA Microperimetry
Time Frame: Baseline, Months 1, 3, 6, 9, 12, 18, and 24
MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0' or a positive integer. The point labelled as '< 0' is assigned a value of '-1' by MAIA in the calculation. Improvement in mean sensitivity in center grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points out of the 16 central points. Here negative values indicate a decline in retinal sensitivity.
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Mean Sensitivity of the 68 Central Loci Points Assessed by MAIA Microperimetry
Time Frame: Baseline, Months 1, 3, 6, 9, 12, 18, and 24
MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0' or a positive integer. The point labelled as '< 0' is assigned a value of '-1' by MAIA in the calculation. Improvement in mean sensitivity in whole grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points of the grid as a whole (68 points). Here negative values indicate a decline in retinal sensitivity.
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Mean Best Corrected Visual Acuity (BCVA) Reported as Letters
Time Frame: Baseline, Months 1, 3, 6, 9, 12, 18, and 24
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was reported as number of letters read correctly by the participants using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Here negative values indicate decline in BCVA.
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Mean Low Luminance Visual Acuity (LLVA) Reported as Letters
Time Frame: Baseline, Months 1, 3, 6, 9, 12, 18, and 24
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Here negative values indicate decline in LLVA.
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥15 Letters Increase From Baseline for BCVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥15 letters increase from baseline for BCVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥15 Letters Increase From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥15 letters increase from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥10 Letters Increase From Baseline for BCVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥10 letters increase from baseline for BCVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥10 Letters Increase From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥10 letters increase from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥5 Letters Increase From Baseline for BCVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters increase from baseline for BCVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥5 Letters Increase From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters increase from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥15 Letters Loss From Baseline for BCVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥15 letters loss from baseline for BCVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥15 Letters Loss From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥15 letters loss from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥10 Letters Loss From Baseline for BCVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥10 letters loss from baseline for BCVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥10 Letters Loss From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥10 letters loss from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥5 Letters Loss From Baseline for BCVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters loss from baseline for BCVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With a ≥5 Letters Loss From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters loss from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With Change From Baseline > -5 Letters for BCVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Percentage of eyes with a change from baseline of ≥ -5 letters for BCVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Eyes With Change From Baseline > -5 Letters for LLVA
Time Frame: Months 1, 3, 6, 9, 12, 18, and 24
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Percentage of eyes with a change from baseline of ≥ -5 letters for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Central Ellipsoid Area
Time Frame: Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Spectral Domain Optical Coherence Tomography (SD-OCT) was used to assess change in central ellipsoid area. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates a decline in central ellipsoid area.
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Central Horizontal Ellipsoid Width
Time Frame: Baseline, Months 1, 3, 6, 9, 12, 18, and 24
SD-OCT was used to assess change in central horizontal ellipsoid width. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates a decline in central horizontal ellipsoid width.
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Fundus Autofluoroscence- Mean Total Area of Preserved Autofluoroscence
Time Frame: Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Fundus Autofluoroscence was used to assess the total area of preserved autofluoroscence. Areas of preserved AF were identified as well-demarcated regions of relative hyper autofluorescence (hyper AF) compared with the background areas of surrounding atrophy. A negative change from baseline indicates a decline in total area of preserved autofluoroscence.
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Fundus Autofluoroscence- Mean Distance From Foveal Center (FC) to Nearest Border of Preserved Autofluoroscence
Time Frame: Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Fundus Autofluoroscence was used to assess the distance from FC to nearest border of preserved autofluoroscence. A negative change from baseline indicates a decline in the total area of preserved autofluoroscence.
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Mean Volume of 30-Degree Hill of Vision
Time Frame: Baseline, Months 6, 12, and 24
Visual field testing was performed to assess the volume of 30-degree hill vision, reported as dBs. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Only reliable measurements were included for analysis of this outcome measure. Here negative values indicate decline in volume of 30-degree hill vision.
Baseline, Months 6, 12, and 24
Part 1: Change From Baseline in Mean Volume of Full Field Hill of Vision
Time Frame: Baseline, Months 6, 12, and 24
Visual field testing was performed to assess the volume of full field of hill vision, reported as dBs. RF =number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Only reliable measurements were included for analysis of this outcome measure. Here negative values indicate decline in volume of 30-degree hill vision.
Baseline, Months 6, 12, and 24
Part 1: Change From Baseline in Contrast Sensitivity (CS) Score
Time Frame: Baseline, Months 3, 6, 12, and 24
Change in CS was assessed by Pelli-Robson chart which uses a single large letter size (20/60 optotype), with contrast varying across groups of letters. Chart uses letters (6 per line), arranged in groups whose contrast varies from high to low. Participants read the letters, starting with the highest contrast, until they were unable to read two or three letters in a single group. Each group had three letters of the same contrast level, so there were three trials per contrast level. Participant is assigned a score based on the contrast of the last group in which two or three letters were correctly read. Score is a measure of the participant's log contrast sensitivity ranging from 0-2.25, with 0 being no letters read, and 2.25 being all letters read. Total CS score = [(total letters correct - 3) x 0.05].
Baseline, Months 3, 6, 12, and 24
Part 2: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry
Time Frame: Months 1, 2, 3, 6, and 9
MAIA microperimetry assessment was measured in dBs using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0' or a positive integer. The point labelled as '< 0' is assigned a value of '-1' by MAIA in the calculation. Improvement in mean sensitivity in center grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points out of the 16 central points.
Months 1, 2, 3, 6, and 9
Part 2: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 68 Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry
Time Frame: Months 1, 2, 3, 6, 9, and 12
MAIA microperimetry assessment was measured in dBs using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0' or a positive integer. The point labelled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in mean sensitivity in whole grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points of the grid as a whole (68 points).
Months 1, 2, 3, 6, 9, and 12
Part 2: Change From Baseline in Mean Sensitivity of the 16 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry
Time Frame: Baseline, Months 1, 2, 3, 6, 9, and 12
MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0' or a positive integer. The point labelled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Mean Sensitivity in center grid was defined as the mean in dB of the 16 points located in the center of the grid. Here negative values indicate a decline in retinal sensitivity.
Baseline, Months 1, 2, 3, 6, 9, and 12
Part 2: Change From Baseline in Mean Sensitivity of the 68 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry
Time Frame: Baseline, Months 1, 2, 3, 6, 9, and 12
MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as '< 0', '0' or a positive integer. The point labelled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in mean ensitivity in whole grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points of the grid as a whole (68 points). Here negative values indicate a decline in retinal sensitivity.
Baseline, Months 1, 2, 3, 6, 9, and 12
Part 2: Change From Baseline in BCVA Reported as Letters
Time Frame: Baseline, Months 1, 2, 3, 6, 9, and 12
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was reported as number of letters read correctly by the participant, using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Here negative values indicate a decline in BCVA.
Baseline, Months 1, 2, 3, 6, 9, and 12
Part 2: Change From Baseline in Mean LLVA Reported as Letters
Time Frame: Baseline, Months 1, 3, 6, 9, and 12
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the participant, using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Here negative values indicate a decline in LLVA.
Baseline, Months 1, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥15 Letter Increase From Baseline for BCVA
Time Frame: Months 1, 2, 3, 6, 9, and 12
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥15 letters increase from baseline for BCVA were reported for study and non-study eyes.
Months 1, 2, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥15 Letter Increase From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, and 12
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥15 letters increase from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥10 Letter Increase From Baseline for BCVA
Time Frame: Months 1, 2, 3, 6, 9, and 12
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Percentage of eyes with a ≥10 letters increase from baseline for BCVA were reported for study and non-study eyes.
Months 1, 2, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥10 Letter Increase From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, and 12
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥10 letters increase from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥5 Letter Increase From Baseline for BCVA
Time Frame: Months 1, 2, 3, 6, 9, and 12
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters increase from baseline for BCVA were reported for study and non-study eyes.
Months 1, 2, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥5 Letter Increase From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, and 12
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters increase from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥15 Letters Loss From Baseline for BCVA
Time Frame: Months 1, 2, 3, 6, 9, and 12
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters loss from baseline for BCVA were reported for study and non-study eyes.
Months 1, 2, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥15 Letters Loss From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, and 12
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥15 letters loss from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥10 Letters Loss From Baseline for BCVA
Time Frame: Months 1, 2, 3, 6, 9, and 12
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥10 letters loss from baseline for BCVA were reported for study and non-study eyes.
Months 1, 2, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥10 Letters Loss From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, and 12
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥10 letters loss from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥5 Letters Loss From Baseline for BCVA
Time Frame: Months 1, 2, 3, 6, 9, and 12
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters loss from baseline for BCVA were reported for study and non-study eyes.
Months 1, 2, 3, 6, 9, and 12
Part 2: Percentage of Eyes With a ≥5 Letters Loss From Baseline for LLVA
Time Frame: Months 1, 3, 6, 9, and 12
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a ≥5 letters loss from baseline for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, and 12
Part 2: Percentage of Eyes With Change From Baseline >-5 Letters for BCVA
Time Frame: Months 1, 2, 3, 6, 9, and 12
BCVA was assessed for both eyes using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a change from baseline of ≥ -5 letters for BCVA were reported for study and non-study eyes.
Months 1, 2, 3, 6, 9, and 12
Part 2: Percentage of Eyes With Change From Baseline >-5 Letters for LLVA
Time Frame: Months 1, 3, 6, 9, and 12
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of eyes with a change from baseline of ≥ -5 letters for LLVA were reported for study and non-study eyes.
Months 1, 3, 6, 9, and 12
Part 2: Change From Baseline in Volume of 30-Degree Hill of Vision Assessed by Octopus 900
Time Frame: Baseline, Months 3, 6, and 12
Visual field testing was performed to assess the volume of 30-degree hill vision, reported as dBs. RF=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Only reliable measurements were included for analysis of this outcome measure. Here negative values indicate decline in the volume of 30-degree hill vision.
Baseline, Months 3, 6, and 12
Part 2: Change From Baseline in Volume of Full Field Hill of Vision Assessed by Octopus 900
Time Frame: Baseline, Months 3, 6, and 12
Visual field testing was performed to assess the volume of full field of hill vision, reported as dBs. RF=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Only reliable measurements were included for analysis of this outcome measure. Here negative values indicate decline in volume of full field hill vision.
Baseline, Months 3, 6, and 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Biogen

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Biogen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 8, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 18, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 18, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 29, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 13, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 14, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 18, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 22, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 274RP101
  • 2016-003852-60 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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Sponsors and Collaborators

Medical Conditions

Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

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