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.

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-002255-37-IE

Enrollment

Registered

2021-10-15

Start date

2022-05-26

Completion date

Unknown

Last updated

2023-10-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

Product Name: Botaretigene Sparoparvovec Product Code: AAV5-hRKp.RPGR Pharmaceutical Form: Solution for injection INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR

Eligibility

Sex/Gender

All

Inclusion 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 90 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: There are no specific exclusion criteria.

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the long-term safety and tolerability 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 functional vision as measured by vision-guided mobility assessment (VMA).;Secondary Objective: To assess changes in all participants after treatment in: retinal function, functional vision, visual function. ;Primary end point(s): Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse events Laboratory Assessment ;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.	—

Measure

Time frame

Main Objective: To assess the long-term safety and tolerability 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 functional vision as measured by vision-guided mobility assessment (VMA).;Secondary Objective: To assess changes in all participants after treatment in: retinal function, functional vision, visual function. ;Primary end point(s): Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse events Laboratory Assessment ;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.

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Secondary

Measure	Time frame
Secondary end point(s): Major secondary efficacy endpoints will include the following: •Retinal Function assessed by -Static perimetry: a.Mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) b. Pointwise responder in full visual field c. Pointwise responder in the central 30-degree visual field d. Mean retinal sensitivity within the full visual field excluding scotoma (MRS90) •Functional Vision assessed by -Vision-guided mobility response in the “worse-seeing eye” as assessed by VMA - Low Luminance Questionnaire (LLQ) in patient-reported outcome – Extreme lighting domain score •Visual Function assessed by - Low luminance visual acuity by ETDRS chart letter score in binocular assessment - Best corrected visual acuity (BCVA) by ETDRS chart letter score in binocular assessment;Timepoint(s) of evaluation of this end point: Major secondary efficacy endpoints up to 60 months	—

Measure

Time frame

Secondary end point(s): Major secondary efficacy endpoints will include the following: •Retinal Function assessed by -Static perimetry: a.Mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) b. Pointwise responder in full visual field c. Pointwise responder in the central 30-degree visual field d. Mean retinal sensitivity within the full visual field excluding scotoma (MRS90) •Functional Vision assessed by -Vision-guided mobility response in the “worse-seeing eye” as assessed by VMA - Low Luminance Questionnaire (LLQ) in patient-reported outcome – Extreme lighting domain score •Visual Function assessed by - Low luminance visual acuity by ETDRS chart letter score in binocular assessment - Best corrected visual acuity (BCVA) by ETDRS chart letter score in binocular assessment;Timepoint(s) of evaluation of this end point: Major secondary efficacy endpoints up to 60 months

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Countries

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

Contacts

Public ContactDominic Fitzsimmons

MeiraGTx II UK Limited

dominic.fitzsimmons@meiragtx.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026