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Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04483440
Recruitment Status : Active, not recruiting
First Posted : July 23, 2020
Last Update Posted : January 19, 2023
Sponsor:
Information provided by (Responsible Party):
4D Molecular Therapeutics

Study Description
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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).

Condition or disease Intervention/treatment Phase
Choroideremia Biological: 4D-110 Phase 1

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.
Study Design
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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 3 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

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Arms and Interventions
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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.


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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)
Contacts and Locations
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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


Locations
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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
Sponsors and Collaborators
4D Molecular Therapeutics
Investigators
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Study Director: Schonmei Lee, MD 4D Molecular Therapeutics
More Information
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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: January 19, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by 4D Molecular Therapeutics:
CHM
transgene
gene therapy
AAV
Additional relevant MeSH terms:
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Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
 Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked