Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.

• Register: EUCTR
• Last refreshed on: 4 April 2022
• Main ID: EUCTR2020-002255-37-ES
• Date of registration: 02/11/2021
• Prospective Registration: Yes
• Primary sponsor: MeiraGTx UK II Limited
• Public title: Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.
• Scientific 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 - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR
• Date of first enrolment: 28/03/2022
• Target sample size: 66
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-002255-37
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: Deferred treatment grp & long-term safety. Efforts to mask vision assessors to treatment assignment If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Dominic Fitzsimmons
• Affiliation: MeiraGTx II UK Limited

• Name: Dominic Fitzsimmons
• Affiliation: MeiraGTx II UK Limited

Key inclusion & exclusion criteria

Inclusion criteria:
1.Previously completed participation in Study MGT-RPGR-021.
2.Must reconfirm that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 60
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
There are no specific exclusion criteria.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
MedDRA version: 20.0 Level: PT Classification code 10038914 Term: Retinitis pigmentosa System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Eye Diseases [C11]

Intervention(s)

Product Name: AAV5-hRKp.RPGR
Pharmaceutical Form: Solution for injection
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-hRKp.RPGR
Concentration unit: vector genomes (vg)/mL
Concentration type: equal
Concentration number: 400000000000-

Product Name: AAV5-hRKp.RPGR
Pharmaceutical Form: Solution for injection
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-hRKp.RPGR
Concentration unit: vector genomes (vg)/mL
Concentration type: equal
Concentration number: 200000000000-

Primary Outcome(s)

Secondary Objective: To assess changes in all participants after treatment in: retinal function, functional vision, visual function and to assess the safety and tolerability of bilateral subretinal delivery of AAV5-hRKp.RPGR.

Main Objective: To assess the long-term safety of AAV5-hRKp.RPGR in individuals with RPGR-XLRP
To assess the long-term efficacy of treatment with AAV5-hRKp.RPGR in individuals with RPGR-XLRP based on assessments of retinal function.

Primary end point(s): Efficacy: Change in retinal sensitivity by pointwise comparison of individual loci in a 185-point customized grid over 60 months

Timepoint(s) of evaluation of this end point: For participants in the long-term follow-up group; (Month 18, 24, 36, 48, and 60)
For participants in the MGT-RPGR-022 treatment group, from baseline to week 52; and follow up at Months 18, 24, 36, 48 and 60.

Secondary Outcome(s)

Secondary end point(s): Major secondary efficacy endpoints will include the following:
•Retinal Function assessed by
-Static perimetry: V30 from Visual Field Modeling Analysis (VFMA) modeling
-Average threshold by microperimetry, under mesopic condition
•Functional Vision assessed by
-visual mobility assessment
-Impact of Vision Impairment on Adults (IVI-A) – Reading and Accessing Information domain score – for adults (age 18 years and older)
•Visual Function assessed by
-Low luminance BCVA by ETDRS chart letter score
-Contrast sensitivity by Pelli-Robson chart in LogMAR

Timepoint(s) of evaluation of this end point: Major secondary efficacy endpoints up to 60 months

Secondary ID(s)

2020-002255-37-IE

MGT-RPGR-022

Source(s) of Monetary Support

MeiraGTx UK II Limited

Ethics review

Status: Approved
Approval date: 22/03/2022
Contact: