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

X-Linked Retinitis Pigmentosa Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04671433
• Other study ID #: CR109258
• Secondary ID: MGT-RPGR-0212020
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: December 4, 2020
• Est. completion date: September 20, 2024

Study information

• Verified date: April 2024
• Source: Janssen Research & Development, LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 97
• Est. completion date: September 20, 2024
• Est. primary completion date: September 20, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• X-Linked Retinitis Pigmentosa

Intervention

Biological:
• Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
• Genetic: AAV5-hRKp.RPGR
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)

Locations

Country	Name	City	State
Belgium	UZ Gent	Gent
Canada	Hospital For Sick Children	Toronto	Ontario
Denmark	Rigshospitalet Glostrup	Glostrup
France	Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts	Paris
Israel	Hadassah Medical Center	Jerusalem
Italy	Azienda Ospedaliero Universitaria Careggi	Firenze
Italy	Ospedale San Paolo	Milano
Italy	Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli	Napoli
Italy	IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS	Roma
Netherlands	VUMC Amsterdam	Amsterdam
Netherlands	Radboudumc	Nijmegen
Spain	Hosp. Univ. Fund. Jimenez Diaz	Madrid
Switzerland	University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical	Basel
Switzerland	Universite de Lausanne, Hopital ophtalmique Jules-Gonin	Lausanne
United Kingdom	NHS Lothian	Edinburgh
United Kingdom	Gartnavel General Hospital	Glasgow
United Kingdom	St James University Hospital	Leeds
United Kingdom	Moorfields Eye Hospital	London
United States	Univ of Michigan Medical Center	Ann Arbor	Michigan
United States	Emory University	Atlanta	Georgia
United States	Retina Consultants of Houston	Bellaire	Texas
United States	Massachusetts General Hospital - Center for Celiac Research and Treatment	Boston	Massachusetts
United States	Duke Eye Center	Durham	North Carolina
United States	VitreoRetinal Associates, PA	Gainesville	Florida
United States	Shiley Eye Institute Jacobs Retina Center	La Jolla	California
United States	Childrens Hospital	Los Angeles	California
United States	Stanford Health Care	Palo Alto	California
United States	University of Pittsburgh Medical Center (UPMC)	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Janssen Research & Development, LLC

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  Denmark,  France,  Israel,  Italy,  Netherlands,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze.	From Baseline to 52 Weeks
Secondary	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	Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed.	From Baseline to Week 52
Secondary	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	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 will be assessed.	From Baseline to Week 52
Secondary	Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52	Pointwise response in full visual field at Week 52 will be assessed.	From Baseline to Week 52
Secondary	Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52	Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed.	From Baseline to Week 52
Secondary	Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52	Pointwise response in the central 30 degrees visual field at Week 52 will be assessed.	From Baseline to Week 52
Secondary	Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52	Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed.	From Baseline to Week 52
Secondary	Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52	Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed.	From Baseline to Week 52
Secondary	Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52	Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52.	From Baseline to Week 52
Secondary	Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52	Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52.	From Baseline to Week 52
Secondary	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	Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52.	From Baseline to Week 52
Secondary	Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52	Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52.	From Baseline to Week 52
Secondary	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	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.	From Baseline to Week 52
Secondary	Number of Participants with Ocular and Non-ocular Adverse Events	Number of participants with ocular and non-ocular adverse events will be assessed.	Day 1 - Week 52
Secondary	Number of Participants With Abnormalities in Laboratory Assessments	Number of participants with abnormalities in laboratory assessments will be assessed.	Day 1 - 52 Weeks