Trials / Completed
CompletedNCT03116113
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
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)
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 50 (actual)
- Sponsor
- Biogen · Industry
- Sex
- Male
- Age
- 10 Years
- Healthy volunteers
- Not accepted
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).
Detailed description
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | BIIB112 | Administered as specified in the treatment arm. |
Timeline
- Start date
- 2017-03-08
- Primary completion
- 2020-11-18
- Completion
- 2020-11-18
- First posted
- 2017-04-14
- Last updated
- 2024-01-18
- Results posted
- 2024-01-18
Locations
8 sites across 2 countries: United States, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03116113. Inclusion in this directory is not an endorsement.