Trials / Completed
CompletedNCT04312672
Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Long-term Follow-up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 42 (actual)
- Sponsor
- Janssen Research & Development, LLC · Industry
- Sex
- Male
- Age
- 5 Years
- Healthy volunteers
- Not accepted
Summary
This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | AAV5-hRKp.RPGR | Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up. |
Timeline
- Start date
- 2017-07-31
- Primary completion
- 2025-09-17
- Completion
- 2025-09-17
- First posted
- 2020-03-18
- Last updated
- 2026-01-21
Locations
5 sites across 2 countries: United States, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04312672. Inclusion in this directory is not an endorsement.