Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100142, China
Location status: Recruiting
Location contact
Ruifang Sui, Professor
CONTACT
Recruiting
NCT Number: NCT05858983
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
Recruiting
Interested in participating?
Request InfoKey information
Age range
8 year–45 year
Sex eligibility
All sexes
Study type
Interventional
Phase
Phase 1 / Phase 2
Primary location
About this study
This study is a multi-center, open-label, phase I/II clinical study to evaluate the safety, tolerability, efficacy, immunogenicity, and in vivo biodistribution characteristics of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.
Who can participate
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
- Subjects who are able to understand and sign the ICF
- Female or male aged 8-45 years old when signing the ICF
- Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy
Exclusion criteria
- Other interfering eye diseases
- Presence of any systemic or ocular disease that can cause or likely to cause vision loss
- There is evidence of obviously uncontrolled concomitant diseases
- Known to have active or suspected autoimmune diseases
- With active systemic infection under treatment
- Pregnant or lactating women
- Other conditions unsuitable for the study as determined by the investigator
Treatment and study plan
FT-001 Low Dose
GeneticComparison of different dosages of FT-001
FT-001 Mid Dose
GeneticComparison of different dosages of FT-001
FT-001 High Dose
GeneticComparison of different dosages of FT-001
Primary outcomes
-
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
Time frame: 52 weeks
Incidence of ocular and non-ocular AEs and SAEs
Secondary outcomes
-
Changes in visual function from baseline
Time frame: 52 weeks
Changes in visual function from baseline as assessed by FST
-
Changes in visual function from baseline
Time frame: 52 weeks
Changes in visual function from baseline as assessed by Mobility courses
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 Multi-center, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of FT-001 Administered Via Subretinal Injection in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Important dates
- Study start
- 2022
- Primary completion
- 2025
- Study completion
- 2029
- First posted
- May 15, 2023
- Registry last updated
- May 15, 2023
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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Biallelic RPE65 Mutation-associated Retinal Dystrophy
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View Trial Details