Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
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ClinicalTrials.gov Identifier: NCT04483440 |
Recruitment Status :
Active, not recruiting
First Posted : July 23, 2020
Last Update Posted : May 9, 2024
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Choroideremia | Biological: 4D-110 | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 13 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential Assignment |
Intervention Model Description: | Each dose escalation cohort will initially recruit up to 3 patients to receive a single uniocular intravitreal injection of 4D-110 in a standard 3+3 design. The cohort will be expanded in the event of a dose limiting toxicity (DLT). Once the maximum tolerated dose (MTD)/maximum feasible dose (MFD) has been identified, up to 4 additional patients may be enrolled at that dose level to provide additional safety information. |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia |
Actual Study Start Date : | June 2, 2020 |
Estimated Primary Completion Date : | June 2024 |
Estimated Study Completion Date : | June 2027 |
Arm | Intervention/treatment |
---|---|
Experimental: 4D-110 Dose 1
4D-110 IVT injection
|
Biological: 4D-110
4D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene. |
Experimental: 4D-110 Dose 2
4D-110 IVT injection
|
Biological: 4D-110
4D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene. |
Experimental: 4D-110 Dose 3
4D-110 IVT injection
|
Biological: 4D-110
4D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene. |
- Frequency and severity of ocular and systemic adverse events (AEs) [ Time Frame: 24 months ]Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.
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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
- Both eyes must have ≥ 34 ETDRS letters (~20/200)
Key Exclusion Criteria:
- Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye
- Patient has previously received any AAV treatment
- Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04483440
United States, Texas | |
Retina Foundation of the Southwest | |
Dallas, Texas, United States, 75251 | |
United States, Utah | |
Moran Eye Center, University of Utah | |
Salt Lake City, Utah, United States, 84132 |
Study Director: | Schonmei Lee, MD | 4D Molecular Therapeutics |
Responsible Party: | 4D Molecular Therapeutics |
ClinicalTrials.gov Identifier: | NCT04483440 |
Other Study ID Numbers: |
4D-110-CP-0001 |
First Posted: | July 23, 2020 Key Record Dates |
Last Update Posted: | May 9, 2024 |
Last Verified: | May 2024 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
CHM transgene gene therapy AAV |
Choroideremia Eye Diseases, Hereditary Eye Diseases Choroid Diseases |
Uveal Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |