Natural History of the Progression of Choroideremia Study (NIGHT)

February 9, 2023 updated by: NightstaRx Ltd, a Biogen Company
The objective of this natural history study is to gain a better understanding of the progression of choroideremia (CHM) and add to the knowledge base for this rare disease.

Study Overview

Status

Completed

Conditions

Detailed Description

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

Study Type

Observational

Enrollment (Actual)

319

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

  • Brazil
      • São Paulo, Brazil, 04552-050
        • Research Site
  • Canada
      • Montréal, Canada, H3A 0E7
        • Research Site
      • Vancouver, Canada, V5Z 3N9
        • Research Site
    • Alberta
      • Edmonton, Alberta, Canada
        • Research Site
  • Denmark
      • Glostrup, Denmark
        • Research Site
  • Finland
      • Helsinki, Finland, 00290
        • Research Site
  • France
      • Montpellier, France, 74103
        • Research Site
  • Germany
      • Bonn, Germany, 53127
        • Research Site
      • Tübingen, Germany
        • Research Site
  • Netherlands
      • Nijmegen, Netherlands
        • Research Site
  • United Kingdom
      • London, United Kingdom, EC1V 2PD
        • Research Site
      • Manchester, United Kingdom, M13 9WL
        • Research Site
      • Oxford, United Kingdom, OX3 9DU
        • Research Site
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Research Site
    • Florida
      • Miami, Florida, United States, 33136
        • Research Site
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Research Site
    • New York
      • New York, New York, United States, 10032
        • Research Site
    • Oregon
      • Portland, Oregon, United States, 97232
        • Research Site
    • Texas
      • Dallas, Texas, United States, 75231
        • Research Site
    • Wisconsin
      • Madison, Wisconsin, United States, 53705
        • 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

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

Description

Key Inclusion Criteria:

  • Are willing and able to provide informed consent for participation in the study.
  • Have a clinical phenotype and confirmed genetic diagnosis of CHM.
  • Have active disease clinically visible within the macular region.
  • Are willing and able to undergo ophthalmic examinations once every 4 months for up to 20 months.
  • Have a BCVA better than or equal to 6/60 (20/200; decimal 0.1; LogMAR 1.0; 34-38 ETDRS letters) in at least one eye.

Key Exclusion Criteria:

  • Have a history of amblyopia in the eligible eye.
  • Have any other significant ocular or non-ocular disease/disorder in the eligible eye which, in the opinion of the investigator, may put the subject at risk because of participation in the study, influence the results of the study or influence the subject's ability to participate in the study.
  • Have participated in an interventional research study in the past 6 months.

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Best-Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (EDTRS)
Time Frame: Up to Month 20
BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.
Up to Month 20

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline in Reading Performance using International Reading Speed Texts (IReST)
Time Frame: Baseline up to Month 12
Reading performance will be evaluated prior to pupil dilation.
Baseline up to Month 12
Change from Baseline in Color Vision
Time Frame: Baseline up to Month 12
Colour vision will be tested prior to pupil dilation. Each eye will be tested separately and in the same order.
Baseline up to Month 12
Change from Baseline in Visual Fields
Time Frame: Baseline up to Month 12
The progression of defects in visual fields will be assessed in both eyes using perimetry equipment.
Baseline up to Month 12
Change from Baseline in Contrast Sensitivity
Time Frame: Baseline up to Month 12
Contrast sensitivity will be measured for both eyes, prior to pupil dilation, using a Pelli Robson chart.
Baseline up to Month 12
Change from Baseline in Retinal Thinning Using Spectral Domain Optical Coherence Tomography (SD-OCT)
Time Frame: Baseline up to Month 20
SD-OCT measurements will be performed after dilation of the participant's pupil.
Baseline up to Month 20
Change from Baseline in Area of Viable Retinal Tissue Using Fundus Autofluorescence
Time Frame: Baseline up to Month 20
Fundus Autofluorescence will be performed after dilation of the participant's pupil to assess changes in the area of viable retinal tissue.
Baseline up to Month 20
Change from Baseline in Retinal Sensitivity Using Microperimetry
Time Frame: Baseline up to Month 20
Macular analyser integrity assessment (MAIA) microperimetry will be conducted for both eyes to assess changes in retinal sensitivity within the macula.
Baseline up to Month 20

Collaborators and Investigators

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

Sponsor

NightstaRx Ltd, a Biogen Company

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)

June 30, 2015

Primary Completion (ACTUAL)

October 1, 2020

Study Completion (ACTUAL)

October 1, 2020

Study Registration Dates

First Submitted

November 24, 2017

First Submitted That Met QC Criteria

November 30, 2017

First Posted (ACTUAL)

December 2, 2017

Study Record Updates

Last Update Posted (ACTUAL)

February 10, 2023

Last Update Submitted That Met QC Criteria

February 9, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 273CH001
  • NSR-CHM-OS1 (OTHER: NightstaRx Ltd, a Biogen Company)

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 http://clinicalresearch.biogen.com/

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

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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