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An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

NCT04519749 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2024-04-08

No results posted yet for this study

Summary

This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease.

Conditions

Interventions

BIOLOGICAL

4D-310

4D-310 is a novel adeno-associated virus (AAV) gene therapy comprised of two active components: the capsid (4D-C102) and the transgene cassette, which encodes a codon-optimized full length human GLA transgene driven by the CAG promoter. 4D-310 has been engineered so that it cannot replicate (replication incompetent).

Sponsors & Collaborators

  • 4D Molecular Therapeutics

    lead INDUSTRY

Principal Investigators

  • Alan H Cohen, MD · 4D Molecular Therapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-01
Primary Completion
2026-01-31
Completion
2030-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04519749 on ClinicalTrials.gov