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Gene Therapy Trial Report

Summary

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


NCTID NCT04671433 (View at clinicaltrials.gov)
Description
Development Status Active
Indication X-Linked Retinitis Pigmentosa (XLRP)
Disease Ontology Term DOID:0110414
Compound Name Botaretigene sparoparvovec
Compound Alias proper name
Compound Description AAV2/5.hRKp.RPGR
Sponsor Janssen Research & Development, LLC
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 🔄 105 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant RPGR
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2/5
Editor Type none
Dose 1 2.0E10 vg/mL
Dose 2 2.0E11 vg/mL
Dose 3 2.0E12 vg/mL

Study Record Dates


Current Stage Phase3
Submit Date 2020-11-05
Completion Date 2024-09-30
Last Update 🔄 2026-03-16

Participation Criteria


Eligible Age >=3 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Male or female * 3 years of age or older * Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory Exclusion Criteria: * Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention 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, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 28
Locations Canada,Netherlands,Belgium,United States,Denmark,Italy,United Kingdom,Israel,France,Switzerland,Spain

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates Phase 3 dosing complete, phase 3 study did not meet its primary efficacy endpoint

Resources/Links