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

Condition(s) studied

Investigational drug(s) / intervention(s)

AAV5-hRKp.RPGR: AAV5-hRKp.RPGR will be administered sub-retinally.

No intervention (Follow-Up assessment): Participants will not receive any intervention and will undergo follow-up assessment.

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

Primary outcome measure(s)

• Number of Participants With Adverse Events (AE) — From baseline up to 5.5 years
  An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non investigational) product, whether or not related to that medicinal (investigational or non investigational) product.
• Change from Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Scores — Baseline, Months 12, 24, 36, 48 and 60
  Change from baseline in BCVA by ETDRS chart letter scores in monocular assessment of the second treated eye will be assessed. BCVA was measured using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. An increase in the number of letters read correctly means that vision has improved and vice-versa.
• Change from Baseline in Low Luminance Visual Acuity (LLVA) by ETDRS Chart Letter Score — Baseline, Months 12, 24, 36, 48 and 60
  Change in LLVA by ETDRS chart letter scores from baseline in monocular assessment of second treated eye will be assessed. Visual function assessments included LLVA assessment in each eye by ETDRS letters. The letter score ranges from 0 (worse score) to 100 (best score), and a gain in LLVA from baseline indicates an improvement in visual acuity.

Trial sites (3)

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