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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04483440
Other study ID # 4D-110-CP-0001
Secondary ID
Status Active, not recruiting
Phase Phase 1
First received
Last updated
Start date June 2, 2020
Est. completion date June 2027

Study information

Verified date May 2024
Source 4D Molecular Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


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 three dose levels in male patients with genetically-confirmed CHM.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 13
Est. completion date June 2027
Est. primary completion date June 2024
Accepts healthy volunteers No
Gender Male
Age group 18 Years and older
Eligibility 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)

Study Design


Related Conditions & MeSH terms


Intervention

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.

Locations

Country Name City State
United States Retina Foundation of the Southwest Dallas Texas
United States Moran Eye Center, University of Utah Salt Lake City Utah

Sponsors (1)

Lead Sponsor Collaborator
4D Molecular Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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. 24 months
See also
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Recruiting NCT05258032 - Structural and Functional Characterization of Rare Ocular Diseases
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Recruiting NCT01866371 - High Resolution Retinal Imaging
Completed NCT01603576 - Pilot Study of a Suprachoroidal Retinal Prosthesis N/A
Enrolling by invitation NCT03584165 - Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa Phase 3
Completed NCT03359551 - Natural History of the Progression of Choroideremia Study
Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry
Completed NCT02671539 - THOR - Tübingen Choroideremia Gene Therapy Trial Phase 2
Completed NCT02670980 - Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy N/A
Completed NCT02341807 - Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations Phase 1/Phase 2
Completed NCT03406416 - Study of a Suprachoroidal Retinal Prosthesis N/A
Enrolling by invitation NCT05158049 - Longitudinal Study of a Bionic Eye
Terminated NCT02994368 - "Natural History" Study of Choroideremia
Terminated NCT01654562 - The Short-term Effects of Simvastatin on the Vision of Males Affected by Choroideremia Phase 1/Phase 2
Active, not recruiting NCT00427180 - IRIS PILOT - Extended Pilot Study With a Retinal Implant System N/A
Completed NCT04750785 - A Study to Assess Choroideremia (CHM) Health Outcomes