A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)

Last updated: April 1, 2025
Sponsor: Janssen Research & Development, LLC
Overall Status: Active - Recruiting

Phase

2

Condition

Eye Disorders/infections

Retinitis Pigmentosa

Posterior Uveitis

Treatment

AAV5-hRKp.RPGR

No intervention (Follow-Up assessment)

Clinical Study ID

NCT06646289
74765340RPG2001
74765340RPG2001
  • Ages > 5
  • Male

Study Summary

The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Have been treated with AAV5-hRKp.RPGR in study MGT009 and have completed or iscurrently enrolled in Study MGT010

  • Must sign an informed consent form indicating that they understand the purpose andprocedures of the study and is willing to participate in the study

  • Willing to adhere to the protocol and long-term follow-up

Exclusion

Exclusion Criteria:

  • There are no specific exclusion criteria to enroll in this study

Study Design

Total Participants: 39
Treatment Group(s): 2
Primary Treatment: AAV5-hRKp.RPGR
Phase: 2
Study Start date:
October 10, 2024
Estimated Completion Date:
October 24, 2030

Connect with a study center

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • University of Michigan Kellogg Eye Center

    Ann Arbor, Michigan 48105
    United States

    Active - Recruiting

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