Clinical trial NCT04671433: Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Official title: Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: X-Linked Retinitis Pigmentosa
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2020-12-17
Enrollment: 97
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
Primary purpose: TREATMENT

Brief summary: A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)


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

Primary outcomes:
measure: Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze
timeFrame: From Baseline to 52 Weeks
Secondary outcomes:
measure: Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52
timeFrame: From Baseline to Week 52
measure: Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52
timeFrame: From Baseline to Week 52
measure: Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
timeFrame: From Baseline to Week 52
measure: Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52
timeFrame: From Baseline to Week 52
measure: Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52
timeFrame: From Baseline to Week 52
measure: Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52
timeFrame: From Baseline to Week 52
measure: Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52
timeFrame: From Baseline to Week 52
measure: Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52
timeFrame: From Baseline to Week 52
measure: Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52
timeFrame: From Baseline to Week 52
measure: Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52
timeFrame: From Baseline to Week 52
measure: Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52
timeFrame: From Baseline to Week 52
measure: Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52
timeFrame: From Baseline to Week 52
measure: Number of Participants with Ocular and Non-ocular Adverse Events
timeFrame: Day 1 - Week 52
measure: Number of Participants With Abnormalities in Laboratory Assessments
timeFrame: Day 1 - 52 Weeks
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Shiley Eye Institute Jacobs Retina Center
state: California
city: Los Angeles
country: United States
facility: Childrens Hospital
state: California
city: Palo Alto
country: United States
facility: Stanford Health Care
state: California
city: Gainesville
country: United States
facility: VitreoRetinal Associates, PA
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Boston
country: United States
facility: Massachusetts General Hospital - Center for Celiac Research and Treatment
state: Massachusetts
city: Ann Arbor
country: United States
facility: Univ of Michigan Medical Center
state: Michigan
city: Durham
country: United States
facility: Duke Eye Center
state: North Carolina
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center (UPMC)
state: Pennsylvania
city: Gent
country: Belgium
facility: UZ Gent
city: Toronto
country: Canada
facility: Hospital For Sick Children
state: Ontario
city: Glostrup
country: Denmark
facility: Rigshospitalet Glostrup
city: Paris
country: France
facility: Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
city: Jerusalem
country: Israel
facility: Hadassah Medical Center
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Careggi
city: Milano
country: Italy
facility: Ospedale San Paolo
city: Napoli
country: Italy
facility: Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
city: Roma
country: Italy
facility: IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
city: Amsterdam
country: Netherlands
facility: VUMC Amsterdam
city: Nijmegen
country: Netherlands
facility: Radboudumc
city: Madrid
country: Spain
facility: Hosp Univ Fund Jimenez Diaz
city: Basel
country: Switzerland
facility: University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
city: Lausanne
country: Switzerland
facility: Universite de Lausanne, Hopital ophtalmique Jules-Gonin
city: Edinburgh
country: United Kingdom
facility: NHS Lothian
city: Glasgow
country: United Kingdom
facility: Gartnavel General Hospital
city: Leeds
country: United Kingdom
facility: St James University Hospital
city: London
country: United Kingdom
facility: Moorfields Eye Hospital
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: