Biallelic RPE65 Mutation-associated Retinal DystrophyPhase 1/2NCT05858983
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
A Phase 1/2 study testing FT-001 Low Dose, FT-001 Mid Dose and FT-001 High Dose in people with Biallelic RPE65 Mutation-associated Retinal Dystrophy, run by Frontera Therapeutics at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 8 to 45 years
- Conditions the study is for
- Biallelic RPE65 Mutation-associated Retinal Dystrophy
- What is being tested?
- FT-001 Low Dose, FT-001 Mid Dose and FT-001 High Dose
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 30 Nov 2022to 30 Nov 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100142
Ruifang Sui, Professor
About this study
Official 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
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.
Read the detailed description
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.
- Being tested
- FT-001 Low Dose, FT-001 Mid Dose, FT-001 High Dose
- Keywords
- FT-001, Biallelic RPE65 Mutation-associated Retinal Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FT-001 Low DoseGenetic
Comparison of different dosages of FT-001
FT-001 Mid DoseGenetic
Comparison of different dosages of FT-001
FT-001 High DoseGenetic
Comparison of different dosages of FT-001
| Study group | Role | What they receive |
|---|---|---|
| FT-001 Dose 1 | Experimental | Intraocular administration of a single low dose of range FT-001 Receives: Genetic: FT-001 Low Dose |
| FT-001 Dose 2 | Experimental | Intraocular administration of a single Mid dose of range FT-001 Receives: Genetic: FT-001 Mid Dose |
| FT-001 Dose 3 | Experimental | Intraocular administration of a single High dose of range FT-001 Receives: Genetic: FT-001 High Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
Incidence of ocular and non-ocular AEs and SAEs
Measured: 52 weeks
Secondary outcomes (2)
Changes in visual function from baseline
Changes in visual function from baseline as assessed by FST
Measured: 52 weeks
Changes in visual function from baseline
Changes in visual function from baseline as assessed by Mobility courses
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2023
- Study start30 Nov 2022
- Primary completion30 Nov 2025 (estimated)
- Study completion30 Nov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Biallelic RPE65 Mutation-associated Retinal DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Frontera Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ruifang Sui, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05858983
- Sponsor's protocol ID
- FT001-C101
- Status last verified
- Oct 2022
- First submitted
- 29 Apr 2023
- Last updated
- 15 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05858983
- Acronym / sponsor ID
- FT001-C101
- Keywords
- FT-001, Biallelic RPE65 Mutation-associated Retinal Dystrophy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Frontera TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Nov 2022 (actual)
- Primary completion
- 30 Nov 2025 (estimated)
- Study completion
- 30 Nov 2029 (estimated)
- First posted
- 15 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| FT-001 Dose 1 | Experimental | Genetic: FT-001 Low DoseIntraocular administration of a single low dose of range FT-001 |
| FT-001 Dose 2 | Experimental | Genetic: FT-001 Mid DoseIntraocular administration of a single Mid dose of range FT-001 |
| FT-001 Dose 3 | Experimental | Genetic: FT-001 High DoseIntraocular administration of a single High dose of range FT-001 |
Who may qualify
- Ages
- 8 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FT-001 Low DoseComparison of different dosages of FT-001 | Genetic | FT-001 Dose 1 |
| FT-001 Mid DoseComparison of different dosages of FT-001 | Genetic | FT-001 Dose 2 |
| FT-001 High DoseComparison of different dosages of FT-001 | Genetic | FT-001 Dose 3 |
Primary outcome
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
2 secondary outcomes
Changes in visual function from baseline
Time frame: 52 weeks
Changes in visual function from baseline
Time frame: 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2023.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ruifang Sui principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FT-001 Low Dose, FT-001 Mid Dose and FT-001 High Dose in people with Biallelic RPE65 Mutation-associated Retinal Dystrophy, run by Frontera Therapeutics at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xinyan Li (+86-021-58206061, Xinyan.li@fronteratherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs) (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 May 2023; AzTrials last refreshed its copy on 7 Nov 2026.