Follow-up 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 Follow-up 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: NCT04794101
• Other study ID #: MGT-RPGR-022
• Secondary ID: MGT-RPGR-0222020
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: December 4, 2020
• Est. completion date: December 19, 2029

Study information

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

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 97
• Est. completion date: December 19, 2029
• Est. primary completion date: September 20, 2029
• 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:

• None

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• X-Linked Retinitis Pigmentosa

Intervention

Biological:
• Genetic: AAV5-hRKp.RPGR Intermediate Dose
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
• Genetic: AAV5-hRKp.RPGR Low Dose
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Locations

Country	Name	City	State
Belgium	Ghent University Hospital	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	Rome
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 Eye and Ear Infirmary	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	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 in vision-guided mobility assessment (VMA), as measured by the ability of the participant to navigate through a VMA maze, after bilateral subretinal delivery of AAV5-hRKp.RPGR	Change from baseline in vision-guided mobility assessment (VMA), as measured by the ability of the participant to navigate through a VMA maze, after bilateral subretinal delivery of AAV5-hRKp.RPGR	From Baseline to Month 60
Primary	Number of participants with Ocular and Non-ocular Adverse Events of AAV5-hRKp.RPGR in Participants with RPGR-XLRP	Number of participants with ocular and non-ocular adverse events of AAV5-hRKp.RPGR in participants with RPGR-XLRP will be assessed.	Day 1 - Month 60
Primary	Number of Participants With Abnormalities in Laboratory Assessments	Number of participants with abnormalities in laboratory assessments will be assessed.	Day 1 - Month 60
Secondary	Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (MRS10) in Static Perimetry	Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be assessed.	From Baseline to Month 60
Secondary	Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma (MRS10) in Static Perimetry	Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be assessed.	From Baseline to Month 60
Secondary	Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field	Pointwise response in full visual field will be assessed.	From Baseline to Month 60
Secondary	Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field	Pointwise response in worse-seeing eye in full visual field will be assessed.	From Baseline to Month 60
Secondary	Change in Retinal Function as Assessed by Pointwise Response in the Central 30-degree Visual Field	Pointwise response in the central 30-degree visual field will be assessed.	From Baseline to Month 60
Secondary	Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30-degree Visual Field	Pointwise response in worse-seeing eye in the central 30-degree visual field will be assessed.	From Baseline to Month 60
Secondary	Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry	Change from baseline in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be assessed.	From Baseline to Month 60
Secondary	Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" as Assessed by VMA	Vision-guided mobility assessment response in the "worse-seeing eye" as assessed by VMA will be assessed.	From Baseline to Month 60
Secondary	Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain Score	Change from baseline in the mLLQ extreme lighting domain score will be assessed.	From Baseline to Month 60
Secondary	Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment	Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.	From Baseline- Month 60
Secondary	Change From Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Monocular Assessment	Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.	From Baseline - Month 60
Secondary	Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Worse-seeing Eye	Change from baseline in low luminance visual acuity by ETDRS chart letter score in worse-seeing eye will be assessed.	From Baseline to Month 60