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

Plain-language summary

This study is evaluating escalating doses of 4D-110, a gene therapy injected into the eye (intravitreal), in patients with choroideremia. Patients with a genetically confirmed disease-causing mutation in the CHM gene are eligible. The study is in Phase 1 and is active but not currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Active, not recruiting

Sponsor

4D Molecular Therapeutics

Intervention

4D-110

Start date

6/2/2020

Estimated completion

8/31/2027

Countries

United States

Related genes

Locations

• Dallas, United States
• Salt Lake City, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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)