X-Linked Retinitis Pigmentosa (XLRP)Phase 1/2NCT06492850
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
A Phase 1/2 study testing FT-002 in people with X-Linked Retinitis Pigmentosa (XLRP), run by Frontera Therapeutics at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 8 to 45 years, men only
- Conditions the study is for
- X-Linked Retinitis Pigmentosa (XLRP)
- What is being tested?
- FT-002
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteChina
- Study dates
- 1 Apr 2024to 1 Feb 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.;
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100142
Ruifang Sui, Professor
About this study
Official 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.
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).
- Conditions
- X-Linked Retinitis Pigmentosa (XLRP)
- Being tested
- FT-002
- Keywords
- X-Linked Retinitis Pigmentosa (XLRP), FT-002
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FT-002Genetic
Intraocular injection of a single dose
| Study group | Role | What they receive |
|---|---|---|
| FT-002 dose 1 | Experimental | Low dose FT-002 Receives: Genetic: FT-002 |
| High dose FT-002 | Experimental | High dose FT-002 Receives: Genetic: FT-002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerance of FT-002
Incidence and severity of AEs
Measured: from FT-002 administration through up to 1 years
Secondary outcomes (1)
To evaluate the efficacy of FT-002
Changes in visual sensitivity /FST/BCVAfrom baseline
Measured: from FT-002 administration through up to 1 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2024
- Study start1 Apr 2024
- Primary completion1 Aug 2025 (estimated)
- Study completion1 Feb 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked Retinitis Pigmentosa (XLRP)Studies 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
- 32 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
- NCT06492850
- Sponsor's protocol ID
- FT002-C101
- Status last verified
- Jul 2024
- First submitted
- 1 Jul 2024
- Last updated
- 9 Jul 2024
- 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 9 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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.
- ClinicalTrials.gov ID
- NCT06492850
- Acronym / sponsor ID
- FT002-C101
- Conditions
- X-Linked Retinitis Pigmentosa (XLRP)
- Keywords
- X-Linked Retinitis Pigmentosa (XLRP), FT-002
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Frontera TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Apr 2024 (actual)
- Primary completion
- 1 Aug 2025 (estimated)
- Study completion
- 1 Feb 2026 (estimated)
- First posted
- 9 Jul 2024 (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 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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| FT-002 dose 1 | Experimental | Genetic: FT-002Low dose FT-002 |
| High dose FT-002 | Experimental | Genetic: FT-002High dose FT-002 |
Who may qualify
- Ages
- 8 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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.;
Key exclusion criteria (1 listed)
- 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.
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-002Intraocular injection of a single dose | Genetic | FT-002 dose 1, High dose FT-002 |
Primary outcome
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
1 secondary outcome
To evaluate the efficacy of FT-002
Time frame: from FT-002 administration through up to 1 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2024.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
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-002 in people with X-Linked Retinitis Pigmentosa (XLRP), 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 1 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: status not confirmed, as of the sponsor's last update on 9 Jul 2024. 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?
Results are not available in the source record.
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?
To evaluate the safety and tolerance of FT-002 (time frame: from FT-002 administration through up to 1 years). 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 9 Jul 2024.