4D-310 in Adults With Fabry Disease and Cardiac Involvement
NCT05629559 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 18
Last updated 2025-02-21
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 and cardiac involvement
Conditions
Interventions
- BIOLOGICAL
-
4D-310
Single IV administration of 4D-310
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
- Max Age
- 65 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2022-10-16
- Primary Completion
- 2026-06-30
- Completion
- 2030-06-30
- FDA Drug
- Yes
Countries
- Australia
- Taiwan
Study Locations
More Related Trials
-
Long-Term Follow-up of Subjects Who Were Treated With ST-920
NCT05039866 ·Status: ENROLLING_BY_INVITATION
-
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
NCT06614569 ·Status: ACTIVE_NOT_RECRUITING
-
Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
NCT06207552 ·Status: RECRUITING ·Phase: EARLY_PHASE1
-
Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease
NCT06114329 ·Status: RECRUITING ·Phase: PHASE2
-
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
NCT06907875 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
NCT06657859 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE2
-
A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome
NCT01894958 ·Status: COMPLETED ·Phase: PHASE2
-
An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy
NCT07222371 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
NCT00351156 ·Status: COMPLETED
-
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
NCT03425539 ·Status: COMPLETED ·Phase: PHASE3
-
Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
NCT02531217 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
NCT04455230 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Study of STG-001 in Subjects With Stargardt Disease
NCT04489511 ·Status: COMPLETED ·Phase: PHASE2
-
A Fabry Disease Gene Therapy Study
NCT04040049 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy
NCT03866954 ·Status: WITHDRAWN ·Phase: PHASE2
-
Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing
NCT05699265 ·Status: TERMINATED
-
The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
NCT06819514 ·Status: NOT_YET_RECRUITING ·Phase: PHASE1/PHASE2
-
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
NCT00283959 ·Status: COMPLETED ·Phase: PHASE2
-
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
NCT03692312 ·Status: COMPLETED ·Phase: PHASE2/PHASE3
-
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
NCT06663358 ·Status: RECRUITING
-
Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders
NCT03812055 ·Status: UNKNOWN
-
Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
NCT02489344 ·Status: COMPLETED ·Phase: PHASE2
-
Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
NCT04552691 ·Status: APPROVED_FOR_MARKETING
-
Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia
NCT04117711 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
NCT05698901 ·Status: RECRUITING