Second treatment for patients with X-linked retinitis pigmentosa who received prior gene therapy
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)
PHASE2 · Janssen Research & Development, LLC · NCT06646289
This study is testing a second gene therapy treatment for people with X-linked retinitis pigmentosa who have already received a similar therapy to see if it is safe and well-tolerated.
Quick facts
| Phase | PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 39 (estimated) |
| Ages | 5 Years and up |
| Sex | Male |
| Sponsor | Janssen Research & Development, LLC (industry) |
| Locations | 2 sites (Boston, Massachusetts and 1 other locations) |
| Trial ID | NCT06646289 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and tolerability of a second subretinal delivery of gene therapy using an Adeno-associated Virus Vector (AAV5 hRKp.RPGR) in individuals who have previously been treated for X-linked retinitis pigmentosa. Participants must have completed or be currently enrolled in a related study. The study aims to gather data on the effects of this follow-on treatment in both adults and children. The methodology includes monitoring participants for safety and tolerability over time.
Who should consider this trial
Good fit: Ideal candidates are individuals who have previously received AAV5-hRKp.RPGR gene therapy and are willing to participate in long-term follow-up.
Not a fit: Patients who have not been treated with AAV5-hRKp.RPGR gene therapy will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could enhance vision preservation or restoration in patients with X-linked retinitis pigmentosa.
How similar studies have performed: Other studies involving gene therapy for retinal diseases have shown promising results, indicating potential for success in this follow-on approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Have been treated with AAV5-hRKp.RPGR in study MGT009 and have completed or is currently enrolled in Study MGT010 * Must sign an informed consent form indicating that they understand the purpose and procedures of the study and is willing to participate in the study * Willing to adhere to the protocol and long-term follow-up Exclusion Criteria: - There are no specific exclusion criteria to enroll in this study
Where this trial is running
Boston, Massachusetts and 1 other locations
- Massachusetts Eye and Ear Infirmary — Boston, Massachusetts, United States (RECRUITING)
- University of Michigan Kellogg Eye Center — Ann Arbor, Michigan, United States (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.
Conditions: X-Linked Retinitis Pigmentosa