Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Condition(s) studied

Investigational drug(s) / intervention(s)

Genetic: AAV5-hRKp.RPGR Intermediate Dose: Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Genetic: AAV5-hRKp.RPGR Low Dose: Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Study summary

A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Eligibility

Primary outcome measure(s)

• Number of Participants with Ocular and Non-ocular Adverse Events — Day 1 - Month 60
  Number of participants with ocular and non-ocular adverse events will be assessed.
• Number of Participants With Abnormalities in Laboratory Assessments — Day 1 - Month 60
  Number of participants with abnormalities in laboratory assessments will be assessed.
• Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment — From Baseline - Month 60
  Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
• Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment — From Baseline- Month 60
  Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.

Trial sites (28)

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