Treatment for Japanese patients with X-linked Retinitis Pigmentosa using gene therapy
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)
This study is testing a new gene therapy to see if it can safely improve vision in Japanese patients with X-linked Retinitis Pigmentosa.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 4 (estimated) |
| Ages | 5 Years and up |
| Sex | All |
| Sponsor | Janssen Pharmaceutical K.K. Industry-sponsored |
| Locations | 1 site (Meguro-ku) |
| Trial ID | NCT05926583 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and tolerability of a gene therapy approach using an adeno-associated virus vector (AAV5-hRKp.RPGR) delivered subretinally to treat X-linked Retinitis Pigmentosa in Japanese participants. The intervention aims to restore retinal function in patients with specific genetic mutations associated with this condition. Participants will be monitored for their retinal sensitivity and outer retinal structure to assess the therapy's effectiveness and safety over time.
Who should consider this trial
Good fit: Ideal candidates are Japanese males and females aged 5 years or older diagnosed with X-linked Retinitis Pigmentosa and showing preserved retinal function.
Not a fit: Patients with advanced retinal degeneration or those without preserved retinal function may not benefit from this treatment.
Why it matters
Potential benefit: If successful, this treatment could significantly improve vision and quality of life for patients suffering from X-linked Retinitis Pigmentosa.
How similar studies have performed: Other studies using gene therapy for retinal diseases have shown promising results, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:
* 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
Exclusion Criteria:
* 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
* 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
Where this trial is running
Meguro-ku
- National Hospital Organization Tokyo Medical Center — Meguro-ku, Japan (Recruiting)
Study contacts
- Study coordinator: Study Contact
- Email: Participate-In-This-Study1@its.jnj.com
- Phone: 844-434-4210
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.