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

4D Molecular Therapeutics (4DMT)

Status and phase: Active, not recruiting; Phase 1

Conditions: Choroideremia

Treatments: Biological: 4D-110

Study type: Interventional

Funder types: Industry

Identifiers

NCT04483440

4D-110-CP-0001

Details and patient eligibility

About

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).

Full 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.

Enrollment: 13 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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)

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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13 participants in 3 patient groups

4D-110 Dose 1

Experimental group

Description:

4D-110 IVT injection

Treatment:

Biological: 4D-110

4D-110 Dose 2

Experimental group

Description:

4D-110 IVT injection

Treatment:

Biological: 4D-110

4D-110 Dose 3

Experimental group

Description:

4D-110 IVT injection

Treatment:

Biological: 4D-110