Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: FT-002.
- Who it may be relevant to
- Registry conditions: X-Linked Retinitis Pigmentosa. Basic parameters: 8 years — 45 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.
Overview
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
Interventions
- Genetic FT-002
Comparison of different dosages of FT-002
Primary outcome measures
- Incidence of AEs [Time frame: from FT-002 administration through up to 1 years]
Secondary outcome measures (2)
- Change in visual function [Time frame: from FT-002 administration through up to 1 years]
- Change in retinal structure as assessed by Optical Coherence Tomography [Time frame: from FT-002 administration through up to 1 years]
Eligibility criteria
Inclusion criteria
1.Subjects that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ;
Exclusion criteria
1.Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 2 centers
- Peking Union Medical College Hospital — Beijing
- Eye & ENT hospital of Fudan university — Shanghai
Identifiers
NCT: NCT05874310 · FT002RP-1