A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

Launched by JANSSEN PHARMACEUTICAL K.K. · Jun 23, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment for X-linked retinitis pigmentosa (XLRP), a genetic eye condition that can lead to vision loss. The treatment being tested is called AAV5-hRKp.RPGR, which is delivered directly into the eye using a method that places it beneath the retina. The main goals of the trial are to ensure that this treatment is safe and that people can tolerate it well.

To participate in this study, candidates must be Japanese males or females aged 5 years or older who have been diagnosed with XLRP and have specific genetic markers related to this condition. Participants should have some preserved vision and a healthy overall status based on medical tests. Those who have had recent eye surgeries or are unable to complete required eye tests may not be eligible. If someone joins the study, they can expect regular check-ins to monitor their health and the treatment’s effects. This trial is currently recruiting participants, so it’s a chance to contribute to important research that could help others with this condition in the future.

Gender

ALL

Eligibility 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