NCT05926583Active, not recruitingPhase 3
A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa
Phase 3 Study to Evaluate the Safety and Efficacy of AAV5-hRKp.RPGR for the Treatment of Japanese X-linked Retinitis Pigmentosa Associated With Pathogenic Variants in Retinitis Pigmentosa GTPase Regulator (RPGR)
Source: ClinicalTrials.gov · Last updated 25 Sept 2026
Brief summary (registry, in English)
The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid human rhodopsin kinase promoter. retinitis pigmentosa guanosine triphosphatase regulator (AAV5-hRKp.RPGR).
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 4 (actual)
- First submitted
- 23 Jun 2023
- Start date
- 12 Sept 2023
- Primary completion date
- 30 Jun 2027
- Lead sponsor
- MeiraGTx Ocular UK Ltd
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT05926583
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 4
- From 12 Sept 2023 to the primary completion date, 30 Jun 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the AAV5-hRKp.RPGR administration
Other
- Is unable to perform the imaging assessments as required (for example: reliable static perimetry \[reliability factor less than or equal to {\<=}19\], optical coherence tomography \[OCT\], or fundus autofluorescence \[FAF\]).
- Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
Who the study is looking for
- Participants who are Japanese male or female aged 5 years or older
- Participants diagnosed as X-linked retinitis pigmentosa (XLRP) (generalized rod-cone dystrophy) associated with pathogenic or likely pathogenic variants in the retinitis pigmentosa guanosine triphosphatase regulator(RPGR) gene
- Has evidence of preserved retinal function as defined by a mean retinal sensitivity of greater than or equal to (\>=) 2 decibel (dB) by Octopus static perimetry and evidence of preserved outer retinal structure (namely the presence of discernible ellipsoid zone) as determined by spectral domain-optical coherence tomography (SD-OCT) in both eyes
- Otherwise, healthy participant on the basis of clinical laboratory tests performed at screening. If the results of the serum chemistry panel or hematology outside the normal reference ranges, the participant may be included only if the investigator judges the abnormalities or deviations from normal to be not clinically significant or to be appropriate and reasonable for the population under study. This determination must be recorded in the participant's source documents and initialed by the investigator
Age in the registry: 5 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| National Hospital Organization Tokyo Medical Center Coordinator of this site? Confirm its status | Meguro-Ku | Japan | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.