Back to resultsDose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
Primary Purpose
Choroideremia
Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
4D-110
Sponsored by
About this trial
This is an interventional treatment trial for Choroideremia focused on measuring CHM, transgene, gene therapy, AAV
Eligibility Criteria
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)
Sites / Locations
- Retina Foundation of the Southwest
- Moran Eye Center, University of Utah
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
4D-110 Dose 1
4D-110 Dose 2
Arm Description
4D-110 IVT injection
4D-110 IVT injection
Outcomes
Primary Outcome Measures
Frequency and severity of ocular and systemic adverse events (AEs)
Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.
Secondary Outcome Measures
Full Information
NCT ID
NCT04483440
First Posted
July 20, 2020
Last Updated
January 18, 2023
Sponsor
4D Molecular Therapeutics
1. Study Identification
Unique Protocol Identification Number
NCT04483440
Brief Title
Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
Official Title
Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia
Study Type
Interventional
2. Study Status
Record Verification Date
January 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 2, 2020 (Actual)
Primary Completion Date
June 2024 (Anticipated)
Study Completion Date
June 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
4D Molecular Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).
Detailed Description
This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at two dose levels in male patients with genetically-confirmed CHM.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Choroideremia
Keywords
CHM, transgene, gene therapy, AAV
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
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 3 additional patients may be enrolled at that dose level to provide additional safety information.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
13 (Actual)
8. Arms, Groups, and Interventions
Arm Title
4D-110 Dose 1
Arm Type
Experimental
Arm Description
4D-110 IVT injection
Arm Title
4D-110 Dose 2
Arm Type
Experimental
Arm Description
4D-110 IVT injection
Intervention Type
Biological
Intervention Name(s)
4D-110
Intervention Description
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.
Primary Outcome Measure Information:
Title
Frequency and severity of ocular and systemic adverse events (AEs)
Description
Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.
Time Frame
24 months
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Schonmei Lee, MD
Organizational Affiliation
4D Molecular Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
Retina Foundation of the Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75251
Country
United States
Facility Name
Moran Eye Center, University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
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