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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
Drug Product Name Botaretigene sparoparvovec
Drug Product Alias proper name
Drug Product 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
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type AAV2/5
Dose 1 2.0E10 vg/mL
Dose 2 2.0E11 vg/mL
Dose 3 2.0E12 vg/mL

Study Record Dates


Current Phase 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