4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
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ClinicalTrials.gov Identifier: NCT04517149 |
Recruitment Status :
Active, not recruiting
First Posted : August 18, 2020
Last Update Posted : January 31, 2023
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Tracking Information | |||||
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First Submitted Date ICMJE | August 14, 2020 | ||||
First Posted Date ICMJE | August 18, 2020 | ||||
Last Update Posted Date | January 31, 2023 | ||||
Actual Study Start Date ICMJE | June 9, 2020 | ||||
Estimated Primary Completion Date | June 2026 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Incidence and severity of TEAEs and serious adverse events (SAEs), including clinically significant changes in safety parameters [ Time Frame: 24 months to 60 Months ] | ||||
Original Primary Outcome Measures ICMJE |
Incidence and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs) [ Time Frame: 24 months ] Incidence and severity of TEAEs and SAEs, including clinically significant changes in safety parameters.
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Change History | |||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||
Original Secondary Outcome Measures ICMJE | Not Provided | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) | ||||
Official Title ICMJE | An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males With X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene | ||||
Brief Summary | This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR). | ||||
Detailed Description | This Phase 1/2 study will gather data in an observational phase Natural History Cohort to further characterize and evaluate natural disease progression in male patients with genetically-confirmed X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR). The study will also evaluate the safety and tolerability, as assessed by frequency and severity of ocular and systemic adverse events, as well as preliminary clinical efficacy of a single intravitreal (IVT) injection of 4D-125 at two dose levels in this patient population in one or both eyes (the contralateral eye dose provided the subject is eligible and provides consent). 4D-125 has been developed as a gene replacement therapy for XLRP. After receiving 4D-125, patients will be followed for 24 months with continued safety follow-up and 36 additional months of long-term follow-up. Secondary endpoints will assess preliminary efficacy measures over time after 4D-125 administration. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Sequential Assignment Intervention Model Description: In Phase 1, up to 2 dose exploration cohorts will be enrolled; each cohort will initially recruit up to 3 patients to receive a single uniocular IVT injection of 4D-125 in a standard 3+3 design. The cohort will be expanded in the event of a dose limiting toxicity (DLT). Any cohort may be expanded by an additional 3 subjects (to a maximum of 12 patients) to provide additional safety information and/or to confirm the selected dose for expansion. Once the dose level has been selected, the Phase 2 Dose Expansion Cohort will be opened to dose an additional 6-12 adult patients, and includes a pediatric sub-population of up to 6 patients. For adult participants only, the contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent. Masking: None (Open Label)Primary Purpose: Treatment |
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Condition ICMJE | X-Linked Retinitis Pigmentosa | ||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Actual Enrollment ICMJE |
21 | ||||
Original Estimated Enrollment ICMJE |
37 | ||||
Estimated Study Completion Date ICMJE | May 2029 | ||||
Estimated Primary Completion Date | June 2026 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Natural History Key Inclusion Criteria:
Interventional Key Inclusion Criteria:
Key Exclusion Criteria (all cohorts)
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Sex/Gender ICMJE |
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Ages ICMJE | 12 Years and older (Child, Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT04517149 | ||||
Other Study ID Numbers ICMJE | 4D-125-C001 | ||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | 4D Molecular Therapeutics | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | 4D Molecular Therapeutics | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | 4D Molecular Therapeutics | ||||
Verification Date | January 2023 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |