Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
NCT ID: NCT05858983
Last Updated: 2023-05-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
9 participants
INTERVENTIONAL
2022-11-30
2029-11-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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FT-001 Dose 1
Intraocular administration of a single low dose of range FT-001
FT-001 Low Dose
Comparison of different dosages of FT-001
FT-001 Dose 2
Intraocular administration of a single Mid dose of range FT-001
FT-001 Mid Dose
Comparison of different dosages of FT-001
FT-001 Dose 3
Intraocular administration of a single High dose of range FT-001
FT-001 High Dose
Comparison of different dosages of FT-001
Interventions
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FT-001 Low Dose
Comparison of different dosages of FT-001
FT-001 Mid Dose
Comparison of different dosages of FT-001
FT-001 High Dose
Comparison of different dosages of FT-001
Eligibility Criteria
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Inclusion Criteria
2. Female or male aged 8-45 years old when signing the ICF
3. Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy
Exclusion Criteria
2. Presence of any systemic or ocular disease that can cause or likely to cause vision loss
3. There is evidence of obviously uncontrolled concomitant diseases
4. Known to have active or suspected autoimmune diseases
5. With active systemic infection under treatment
6. Pregnant or lactating women
7. Other conditions unsuitable for the study as determined by the investigator
8 Years
45 Years
ALL
No
Sponsors
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Frontera Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Ruifang Sui
Role: PRINCIPAL_INVESTIGATOR
Peking Union Medical College Hospital
Locations
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Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
Countries
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Central Contacts
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Facility Contacts
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Ruifang Sui, Professor
Role: primary
Other Identifiers
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FT001-C101
Identifier Type: -
Identifier Source: org_study_id
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