A Study of AAV5-hRKp.RPGR for the Treatment ofJapanese Participants with X-linked Retinitis Pigmentosa
- Conditions
- X-Linked Retinitis Pigmentosa
- Registration Number
- JPRN-jRCT2031230141
- Lead Sponsor
- umaguchi Hirotaka
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- All
- Target Recruitment
- 4
Participants diagnosed as X-linked retinitis pigmentosa (XLRP) (generalized rod-cone dystrophy) associated with 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
- Has a mean retinal sensitivity of less than or equal to (<=)20 dB by Octopus static perimetry
- Otherwise, healthy participant on the basis of physical examination, medical history, and vital signs, performed at screening. Any abnormalities, must be consistent with the underlying illness in the study population and this determination must be recorded in the participant's source documents and initialed by the investigator
- 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
- 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
- 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
- Has undergone prior retinal surgery involving the macula, vitrectomy, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery (except cataract surgery)
- History of an ocular implant, with the exception of an intraocular lens
- Uncontrolled hypertension defined as a systolic value greater than or equal to >=140 millimeter of mercury (mm Hg) or diastolic value >=90 mm Hg at screening
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method umber of Participants with Adverse Event (AEs):<br>Up to 60 Months<br>An AE is any untoward medical occurrence in a clinical study participant administered a investigational or non-investigational medicinal product. An AE does not necessarily have a causal relationship with the treatment.<br><br>Number of Participants with Abnormalities in Clinical Laboratory Assessments:<br>Up to 60 Months<br>Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported.
- Secondary Outcome Measures
Name Time Method