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

Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04483440

Enrollment

Registered

2020-07-23

Start date

2020-06-02

Completion date

2027-08-31

Last updated

2025-05-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Choroideremia

Keywords

CHM, transgene, gene therapy, AAV

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.

Interventions

BIOLOGICAL4D-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.

Study design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

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 4 additional patients may be enrolled at that dose level to provide additional safety information.

Eligibility

Sex/Gender

MALE

Age

18 Years to No maximum

Healthy volunteers

Inclusion 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)

Design outcomes

Primary

Measure	Time frame	Description
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.

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 24, 2026