X-Linked Retinitis PigmentosaEarly Phase 1NCT05874310
Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
A Early Phase 1 study testing FT-002 in people with X-Linked Retinitis Pigmentosa, run by Frontera Therapeutics at 2 sites in China.
Trial summary
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (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
- 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
- 18 participants
- Where
- 2 sitesChina
- Study dates
- 1 Feb 2023to 1 Nov 2027
- Phase
- Early Phase 1
- 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
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 1.Subjects that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ;
Who cannot join
- 1.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
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100142
Ruifang Sui, Professor
- Recruiting
Eye & ENT hospital of Fudan university
Shanghai, Shanghai Municipality, 200032
Gezhi Xu, Professor
Jihong Wu, Professor
About this study
Official title: An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
- Conditions
- X-Linked Retinitis Pigmentosa
- Being tested
- FT-002
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FT-002Genetic
Comparison of different dosages of FT-002
| Study group | Role | What they receive |
|---|---|---|
| Low dose FT-002 | Experimental | Intraocular injection of a single low dose of FT-002 Receives: Genetic: FT-002 |
| Intermediate dose FT-002 | Experimental | Intraocular injection of a single Intermediate dose of FT-002 Receives: Genetic: FT-002 |
| High dose FT-002 | Experimental | Intraocular injection of a single High dose of FT-002 Receives: Genetic: FT-002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of AEs
Number and proportion of Adverse Events
Measured: from FT-002 administration through up to 1 years
Secondary outcomes (2)
Change in visual function
Change in retinal function as assessed by mean retinal sensitivity within the 30-degree visual field
Measured: from FT-002 administration through up to 1 years
Change in retinal structure as assessed by Optical Coherence Tomography
Change in Outer Nuclear Layer Thickness from baseline as assessed by Optical Coherence Tomography
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 posted24 May 2023
- Study start1 Feb 2023
- Primary completion31 Oct 2023 (estimated)
- Study completion1 Nov 2027 (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 PigmentosaStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- X-linked retinitis pigmentosa patients
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Frontera Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Eye & ENT Hospital of Fudan University, Peking Union Medical College Hospital, Henan Provincial People's Hospital
- Responsible party
- Sponsor
- Investigators
- Gezhi Xu, Principal investigator, Eye & ENT Hospital of Fudan UniversityRuifang Sui, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05874310
- Sponsor's protocol ID
- FT002RP-1
- Status last verified
- Aug 2022
- First submitted
- 29 Apr 2023
- Last updated
- 24 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Researchers qualified can request the dataset, including de-identified individual subject data.
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.
- ClinicalTrials.gov ID
- NCT05874310
- Acronym / sponsor ID
- FT002RP-1
- Conditions
- X-Linked Retinitis Pigmentosa
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Frontera TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Eye & ENT Hospital of Fudan University, Peking Union Medical College Hospital, Henan Provincial People's Hospital
- Study start
- 1 Feb 2023 (actual)
- Primary completion
- 31 Oct 2023 (estimated)
- Study completion
- 1 Nov 2027 (estimated)
- First posted
- 24 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.
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- X-linked retinitis pigmentosa patients
| Arm | Type | Receives |
|---|---|---|
| Low dose FT-002 | Experimental | Genetic: FT-002Intraocular injection of a single low dose of FT-002 |
| Intermediate dose FT-002 | Experimental | Genetic: FT-002Intraocular injection of a single Intermediate dose of FT-002 |
| High dose FT-002 | Experimental | Genetic: FT-002Intraocular injection of a single High dose of FT-002 |
Who may qualify
- Ages
- 8 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- 1.Subjects that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ;
Key exclusion criteria (1 listed)
- 1.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-002Comparison of different dosages of FT-002 | Genetic | High dose FT-002, Intermediate dose FT-002, Low dose FT-002 |
Primary outcome
Incidence of AEs
Time frame: from FT-002 administration through up to 1 years
Number and proportion of Adverse Events
2 secondary outcomes
Change in visual function
Time frame: from FT-002 administration through up to 1 years
Change in retinal structure as assessed by Optical Coherence Tomography
Time frame: from FT-002 administration through up to 1 years
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 24 May 2023.
- Individual participant dataSharing planned: No. Researchers qualified can request the dataset, including de-identified individual subject data.
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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.
Gezhi Xu principal investigator
Eye & ENT Hospital of Fudan University
Ruifang Sui principal investigator
Peking Union Medical College Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing FT-002 in people with X-Linked Retinitis Pigmentosa, run by Frontera Therapeutics at 2 sites 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 1 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: looking for participants now, as of the sponsor's last update on 24 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 2 sites 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Incidence of AEs (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 24 May 2023.