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NCT Number: NCT04483440

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

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).

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Choroideremia Choroid Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Genetic Diseases, X-Linked Uveal Diseases

Age range

18 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1

Primary location

Retina Foundation of the Southwest, Dallas, Texas, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

4D-110

Biological

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.

Primary outcomes

  1. Frequency and severity of ocular and systemic adverse events (AEs)

    Time frame: 24 months

    Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.

Sponsors and collaborators

Lead sponsor

4D Molecular Therapeutics

Industry

Registry information

Official study title

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

Important dates

Study start
2020
Primary completion
2027
Study completion
2027
First posted
Jul 23, 2020
Registry last updated
May 22, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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