Trials / Active Not Recruiting
Active Not RecruitingNCT06646289
A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
An Open-label, Multicenter, Phase 2 Follow-on Study for Second Eye Treatment of Patients Previously Treated With 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
- Active Not Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 24 (actual)
- Sponsor
- Janssen Research & Development, LLC · Industry
- Sex
- Male
- Age
- 5 Years
- Healthy volunteers
- Not accepted
Summary
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | AAV5-hRKp.RPGR | AAV5-hRKp.RPGR will be administered sub-retinally. |
| OTHER | No intervention (Follow-Up assessment) | Participants will not receive any intervention and will undergo follow-up assessment. |
Timeline
- Start date
- 2024-10-10
- Primary completion
- 2030-10-24
- Completion
- 2030-10-24
- First posted
- 2024-10-17
- Last updated
- 2026-04-13
Locations
3 sites across 2 countries: United States, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06646289. Inclusion in this directory is not an endorsement.