CompletedPhase 1/2

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

United States, United Kingdom50 participantsStarted 2017-03-08

Plain-language summary

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).

Who can participate

Age range

10 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Part 1: Number of Participants With Dose-Limiting Toxicities (DLTs)

Timeframe: Up to Month 24

Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Timeframe: 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

Timeframe: Month 12

Part 2: Number of Participants With TEAEs

Timeframe: Day 0 (surgery) in Part 2 of the study up to 12 months

Trial details

NCT IDNCT03116113

SponsorBiogen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-11-18

Results submitted2023-11-16

View on ClinicalTrials.gov More X-Linked Retinitis Pigmentosa trials