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).

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

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: November 2021

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 two dose levels in male patients with genetically-confirmed CHM.


  • 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.

Arms, Groups and Cohorts

  • Experimental: 4D-110 Dose 1
    • 4D-110 IVT injection
  • Experimental: 4D-110 Dose 2
    • 4D-110 IVT injection

Clinical Trial Outcome Measures

Primary Measures

  • Frequency and severity of ocular and systemic adverse events (AEs)
    • Time Frame: 6 months
    • Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.

Participating in This Clinical Trial

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)

Gender Eligibility: Male

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • 4D Molecular Therapeutics
  • Collaborator
    • Roche Pharma AG
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Robert Kim, MD, Study Director, 4D Molecular Therapeutics
  • Overall Contact(s)
    • Tamim Assifi, 510-505-2680,

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