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Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)

NCT03252847 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 49

Last updated 2025-01-08

Study results available
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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.

Conditions

  • X-Linked Retinitis Pigmentosa

Interventions

GENETIC

AAV5-RPGR

Single, subretinal administration of AAV5-RPGR

Sponsors & Collaborators

  • Syne Qua Non Limited

    collaborator INDUSTRY

  • Bionical Emas

    collaborator INDUSTRY

  • MeiraGTx UK II Ltd

    lead INDUSTRY

Principal Investigators

  • James Bainbridge, Prof · University College, London

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
5 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-07-31
Primary Completion
2021-11-18
Completion
2021-11-18
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03252847 on ClinicalTrials.gov