Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100142, China
Location status: Recruiting
Location contact
Ruifang Sui, Professor
CONTACT
Recruiting
NCT Number: NCT06492850
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).
Recruiting
Interested in participating?
Request InfoKey information
Conditions
Age range
8 year–45 year
Sex eligibility
Male
Study type
Interventional
Phase
Phase 1 / Phase 2
Primary location
Who can participate
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
- Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form;
- Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF;
- Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.;
Exclusion criteria
- 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.
Treatment and study plan
FT-002
GeneticIntraocular injection of a single dose
Primary outcomes
-
To evaluate the safety and tolerance of FT-002
Time frame: from FT-002 administration through up to 1 years
Incidence and severity of AEs
Secondary outcomes
-
To evaluate the efficacy of FT-002
Time frame: from FT-002 administration through up to 1 years
Changes in visual sensitivity /FST/BCVAfrom baseline
Study contacts
Contact information is provided by the study sponsor or research team.
Minghui Xue
CONTACT
Xinyan Li
CONTACT
Sponsors and collaborators
Lead sponsor
Frontera Therapeutics
Industry
Registry information
Official study title
A Phase I/II Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of FT-002 Subretinal Injection in Subjects With RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa.
Important dates
- Study start
- 2024
- Primary completion
- 2025
- Study completion
- 2026
- First posted
- Jul 9, 2024
- Registry last updated
- Jul 9, 2024
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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