Phase 2
N=45
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
X-Linked Retinitis Pigmentosa
Bottom Line
View on ClinicalTrials.gov: NCT03252847 ↗Enrolled (actual)
45
Serious AEs
5.2%
Results posted
Jan 2025
Primary outcome: Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Product (ATIMP), Not Surgery Alone. — 0; 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AAV5-RPGR (Genetic)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- Male
- Sponsor
- MeiraGTx UK II Ltd
- Primary completion
- Nov 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Product (ATIMP), Not Surgery Alone. |
0; 0; 0; 0 | — |
| SECONDARY Improvements in Visual Function as Assessed by Visual Acuity |
0.6; -3.2 | — |
| SECONDARY Improvements in Retinal Function as Assessed by Static Perimetry |
6; 2 | — |
| SECONDARY Quality of Life Measured by the Low Luminance Questionnaire (LLQ) Emotional Distress Domain Score |
2.344; -1.389 | — |
| SECONDARY Quality of Life Measured by the Low Luminance Questionnaire (LLQ) Extreme Lighting Domain Score |
2.956; -8.059 | — |
| SECONDARY Quality of Life Measured by the Low Luminance Questionnaire (LLQ) General Dim Lighting Domain Score |
4.115; 1.852 | — |
| SECONDARY Quality of Life Measured by the Low Luminance Questionnaire (LLQ) Mobility Domain Score |
5.729; 0.926 | — |
| SECONDARY Quality of Life Measured by the Low Luminance Questionnaire (LLQ) Peripheral Vision Domain Score |
1.563; -1.852 | — |
Summary
Phase 1 of the study is a dose escalation of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR. Participants enrolled in Phase 1 were assigned to a dose group based on when they enrolled (i.e., sequential assignment).
Phase 2 of the study is a cohort expansion of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety and efficacy of this vector in participants with XLRP caused by mutations in RPGR. Participants enrolled in Phase 2 were randomized to immediate or deferred treatment.
Eligibility Criteria
Key inclusion Criteria
- Males aged 5 years or older
- Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)
Key exclusion Criteria
- Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
Data sourced from ClinicalTrials.gov (NCT03252847). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.