Long-term Follow-up Gene Therapy Study for RPGR- XLRP

X-Linked Retinitis Pigmentosa Clinical Trial

Official title:

Long-term Follow-up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04312672
• Other study ID #: MGT010
• Secondary ID: MGT0102018-00042
• Status: Active, not recruiting
• Start date: July 31, 2017
• Est. completion date: November 18, 2026

Study information

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

Clinical Trial Summary

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 42
• Est. completion date: November 18, 2026
• Est. primary completion date: November 18, 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

Inclusion Criteria:

• Received AAV5-hRKp.RPGR in the MGT009 Study

Exclusion Criteria:

• Unwilling or unable to meet with the requirements of the study

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• X-Linked Retinitis Pigmentosa

Intervention

Biological:
• AAV5-hRKp.RPGR
Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up.

Locations

Country	Name	City	State
United Kingdom	Leeds Teaching Hospitals NHS Trust	Leeds
United Kingdom	Moorfields Eye Hospital	London
United States	Univ of Michigan Medical Center	Ann Arbor	Michigan
United States	Massachusetts Eye and Ear Infirmary	Boston	Massachusetts
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Janssen Research & Development, LLC

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants	Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported.	60 Months
Secondary	Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)	Change in functional vision of walk time after treatment administration in VMA (Version 1.0) will be reported.	Up to 60 Months
Secondary	Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)	Change in functional vision of LLQ domain scores in PRO will be reported. The instrument is scored by domain, computed by scaling individual items from 0 to 100 and then averaging the individual items for each domain. A higher score reflects higher functional level.	Up to 60 Months
Secondary	Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score	The longer-term efficacy of AAV5-hRKp.RPGR in change in BCVA using ETDRS chart letter score will be reported.	Up to 60 Months
Secondary	Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions	Change in LLVA as assessed by ETDRS chart under low luminance conditions will be reported.	Up to 60 Months
Secondary	Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry	Change in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported.	Up to 60 months
Secondary	Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field	Change in retinal sensitivity by pointwise comparison of data in static perimetry within the full visual field will be reported.	Up to 60 months
Secondary	Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field	Change in retinal sensitivity by pointwise comparison of data in static perimetry within the Central 30 Degrees visual field will be reported.	Up to 60 months
Secondary	Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry	Change in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be reported.	Up to 60 months