Trials / Active Not Recruiting
Active Not RecruitingNCT04519749
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
- Status
- Active Not Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 18 (estimated)
- Sponsor
- 4D Molecular Therapeutics · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Detailed description
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
| Type | Name | Description |
|---|---|---|
| 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). |
Timeline
- Start date
- 2020-09-01
- Primary completion
- 2026-01-01
- Completion
- 2030-06-01
- First posted
- 2020-08-20
- Last updated
- 2024-04-08
Locations
4 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04519749. Inclusion in this directory is not an endorsement.