A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel

September 10, 2018 updated by: Neurotech Pharmaceuticals

A Phase 2 Multicenter Randomized Clinical Trial of Ciliary Neurotrophic Factor (CNTF) for Macular Telangiectasia Type 2 (MacTel)

This study is a phase 2, randomized, multi-center, single-masked study to evaluate the efficacy and safety of the NT-501 implants in participants with Mactel.

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.

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.
67

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

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

  • Australia
      • East Melbourne, Australia
        • Centre For Eye Research Australia
      • Nedlands, Australia
        • Lions Eye Institute
    • New South Wales
      • Sydney, New South Wales, Australia
        • Save Sight Institute
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Jules Stein Eye Institute
    • Florida
      • Miami, Florida, United States, 33136
        • Bascom Palmer
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • National Eye Institute
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts Eye and Ear Infirmary, Retina Service
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • University of Michigan, Kellogg Eye Center
    • Ohio
      • Beachwood, Ohio, United States, 44122
        • Retina Associates of Cleveland, Inc.
    • Wisconsin
      • Madison, Wisconsin, United States, 53705
        • University of Wisconsin

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

17 years to 76 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Participant must be offered sufficient opportunity to review and to understand the informed consent form, agree to the form's contents and sign the protocol's informed consent
  • Participant must have at least one study eye with a positive diagnosis of MacTel Type 2
  • Participant must have an IS/OS PR break in the study eye(s) and en face ellipsoid zone (area of IS/OS loss) as measured by SDOCT between 0.16 mm2 and 4.00 mm2
  • If female, participant must be incapable of pregnancy
  • If male, participant must agree to use an effective form of birth control during the study

Exclusion Criteria:

  • Participant is unable to provide informed consent
  • Participant is less than 21 years of age or greater than 80 years of age
  • Participant is medically unable to comply with study procedures or follow-up visits
  • Participant was a study subject in any other clinical trial of an intervention (drug or device) within the last 6 months
  • Participant is pregnant or breastfeeding

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: Parallel Assignment
  • Masking: Single

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: NT-501 Implant procedure
The investigational product is the NT-501 encapsulated cell system which consists of cells encapsulated within a semi-permeable polymer membrane and supportive matrices. NT-501 contains NTC-201 cells that were derived from the NTC-200 cell line by genetic modification so as to secrete recombinant human ciliary neurotrophic factor (CNTF).
Biological: Ciliary neurotrophic factor (CNTF)
Ciliary neurotrophic factor released from NT-501 encapsulated cell implant
Device: NT-501 Implant
NT-501 encapsulated cell implant
Procedure: NT-501 Implant procedure
Surgery to implant device for NT-501 encapsulated cell implant releasing human ciliary neurotrophic factor arm
Sham Comparator: Sham procedure
Non-penetrating sham procedure to mimic implant procedure
Procedure: Sham procedure
Sham surgery for Sham arm

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
Ellipsoid zone (area of IS/OS loss) as measured by en face imagining by SDOCT in study eye(s)
Time Frame: 24 months
Change in the ellipsoid zone (area of IS/OS loss) from baseline to month 24 as measured by en face imaging by SDOCT in study eye(s)
24 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
Ellipsoid zone
Time Frame: 12 months
Change in the ellipsoid zone from baseline to Month 12.
12 months
Retinal sensitivity (dB) as measured by microperimetry
Time Frame: 12 and 24 months
Change in retinal sensitivity (dB) as measured by microperimetry from baseline to Months 12 and 24.
12 and 24 months
Increase in ellipsoid zone
Time Frame: 12 and 24 months
Proportion of study eyes with a 35% or more increase from baseline in the ellipsoid zone at Months 12 and 24.
12 and 24 months
Visual Acuity
Time Frame: 12 and 24 months
Change in best corrected visual acuity (BCVA) from baseline to Months 12 and 24.
12 and 24 months
Visual Acuity
Time Frame: 12 and 24 Months
Proportion of study eyes with 15 or more letter loss from baseline in BCVA at Months 12 and 24.
12 and 24 Months
Visual Acuity
Time Frame: 12 and 24 Months
Proportion of study eyes with 10 or more letter loss from baseline in BCVA at Months 12 and 24.
12 and 24 Months
Reading Speed
Time Frame: 12 and 24 Months
Change in reading speed as measured by the IReST from baseline to Months 12 and 24.
12 and 24 Months

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Cone density as measured by AOSLO
Time Frame: 12 and 24 Months
Change in cone density as measured by AOSLO from baseline to Months 12 and 24, in selected participants.
12 and 24 Months
National Eye Institute Visual Functioning Questionnaire
Time Frame: 12 and 24 months
Change in NEI VFQ (overall and subscale) from baseline to Months 12 and 24.
12 and 24 months
Electroretinogram changes
Time Frame: 6, 12 and 24 Months
Electroretinogram (ERG) changes from baseline to Months 6, 12 and 24, in selected clinics/participants.
6, 12 and 24 Months

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.
Neurotech Pharmaceuticals

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
The Emmes Company, LLC
The Lowy Medical Research Institute Limited

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

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.
April 1, 2014

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.
April 14, 2017

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.
May 22, 2017

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).
September 19, 2013

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.
September 19, 2013

First Posted (Estimate)

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.
September 24, 2013

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.
September 12, 2018

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.
September 10, 2018

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.
September 1, 2018

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.
  • NTMT-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

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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