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

Choroideremia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04483440
• Other study ID #: 4D-110-CP-0001
• Status: Active, not recruiting
• Phase: Phase 1
• 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
• 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)

Related Conditions & MeSH terms

• Choroideremia

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