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

Phase 1

Active, not recruiting

• Conditions: Choroideremia
• Interventions: Biological: 4D-110

• Registration Number: NCT04483440
• Lead Sponsor: 4D Molecular Therapeutics

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

Study Timeline

• Study Start: 2020-06-02
• Study First Submitted: 2020-07-20
• Study First Submitted QC: 2020-07-20
• Study First Posted: 2020-07-23
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-07
• Last Update Posted: 2024-05-09
• Primary Completion: 2024-06
• Study Completion: 2027-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 13

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

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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)

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
4D-110 Dose 2	4D-110	4D-110 IVT injection
4D-110 Dose 3	4D-110	4D-110 IVT injection
4D-110 Dose 1	4D-110	4D-110 IVT injection

Primary Outcome Measures

Name	Time	Method
Frequency and severity of ocular and systemic adverse events (AEs)	24 months	Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.

Trial Locations

Locations (2)

Moran Eye Center, University of Utah; 🇺🇸 Salt Lake City, Utah, United States

Retina Foundation of the Southwest; 🇺🇸 Dallas, Texas, United States