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ong term follow-up study of gene therapy trial for X-linked Retinitis Pigmentosa

Phase 1
Active, not recruiting
Conditions
X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
MedDRA version: 20.0Level: PTClassification code 10038914Term: Retinitis pigmentosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Eye Diseases [C11]
Registration Number
EUCTR2018-000425-31-Outside-EU/EEA
Lead Sponsor
Janssen-Cilag International NV
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Active
Sex
Male
Target Recruitment
Not specified
Inclusion Criteria

Inclusion in the study will be limited to individuals who meet the following criteria:
• Are able to give informed consent or assent, with or without the guidance of their parent/guardian where appropriate
• Received AAV5-hRKp.RPGR by sub retinal administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-003967-21)
• Are willing to adhere to the protocol and long-term follow-up
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 41
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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