An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
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ClinicalTrials.gov Identifier: NCT04519749 |
Recruitment Status :
Active, not recruiting
First Posted : August 20, 2020
Last Update Posted : January 31, 2023
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Condition or disease | Intervention/treatment | Phase |
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Fabry Disease | Biological: 4D-310 | Phase 1 Phase 2 |
Study Type : | Interventional |
Estimated Enrollment : | 18 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease |
Actual Study Start Date : | September 1, 2020 |
Estimated Primary Completion Date : | June 2023 |
Estimated Study Completion Date : | June 2027 |

Arm | Intervention/treatment |
---|---|
Experimental: 4D-310 Dose Level 1 - AAV Neutralizing Antibody (NAb) Group A
Single IV administration of 4D-310 Dose Level 1 - AAV NAb Titer Group A patients
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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). |
Experimental: 4D-310 Dose Level 1 - AAV NAb Titer Group B
Single IV administration of 4D-310 Dose Level 1 - AAV NAb titer Group B patients
|
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). |
Experimental: 4D-310 Dose Level 2 - AAV NAb Titer Group A and/or B
Single IV administration of 4D-310 at Dose Level 2 in AAV NAb titer Group A and/or B patients
|
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). |
Experimental: 4D-310 Dose Expansion
Dose expansion cohort of single IV administration of 4D-310 at the selected dose and selected AAV Nab titer group(s) patients
|
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). |
- Incidence and severity of adverse events [ Time Frame: 1 year ]Safety and tolerability of 4D-310 following a single IV dose, as assessed by incidence and severity of adverse events, serious adverse events and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters
- Change from baseline in serum AGA activity [ Time Frame: 1 year ]Change from baseline in serum AGA activity
- Change from baseline serum globotriaosylsphingosine (lysoGb3) [ Time Frame: 1 year ]Change from baseline serum globotriaosylsphingosine (lysoGb3)

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female ≥ 18 years of age
- Pathogenic GLA mutation consistent with Fabry Disease
- Confirmed diagnosis of classic or late-onset Fabry disease
- Individuals on ERT must be on a stable dose for at least 6 months (and a minimum of 12 months total exposure) prior to study enrollment
- Agree to use highly effective contraception
Exclusion Criteria:
- Presence of high titer neutralizing antibody to 4D-310 capsid, or presence of high antibody titer to AGA
- eGFR <45 mL/min/1.73 m2
- Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis
- HIV, active or chronic hepatitis B or C,
- Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control
- History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism)
- Contraindication to systemic corticosteroid therapy or immunosuppressive therapy
- Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.
- Moderately severe to severe cardiovascular disease or uncontrolled hypertension
- Left ventricular ejection fraction of <45% on echocardiogram (ECHO)
- Currently receiving investigational drug, device or therapy or having ever received gene therapy
- History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation
- History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent)
- Pregnant or breast-feeding

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04519749
United States, Alabama | |
University of Alabama at Birmingham | |
Birmingham, Alabama, United States, 35294 | |
United States, California | |
University of California at San Diego | |
La Jolla, California, United States, 92037 | |
University of California at Los Angeles | |
Los Angeles, California, United States, 90025 | |
United States, Georgia | |
Emory University | |
Atlanta, Georgia, United States, 30322 | |
United States, New Jersey | |
Hackensack University | |
Hackensack, New Jersey, United States, 07601 | |
United States, Pennsylvania | |
Children's Hospital of Pittsburgh of UPMC | |
Pittsburgh, Pennsylvania, United States, 15224 | |
United States, Utah | |
University of Utah | |
Salt Lake City, Utah, United States, 84108 | |
United States, Virginia | |
Lysosomal & Rare Disorders Research & Treatment Center, Inc | |
Fairfax, Virginia, United States, 22030 |
Study Director: | Mitra Tavakkoli, MD, PharmD | 4D Molecular Therapeutics |
Responsible Party: | 4D Molecular Therapeutics |
ClinicalTrials.gov Identifier: | NCT04519749 |
Other Study ID Numbers: |
4D-310-C001 |
First Posted: | August 20, 2020 Key Record Dates |
Last Update Posted: | January 31, 2023 |
Last Verified: | January 2023 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Lysosomal Storage Diseases Nervous System Brain Diseases Inborn Brain Diseases Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Vascular Diseases Cardiovascular Diseases Genetic Diseases |
Metabolic Diseases Lipid Metabolism Disorders Metabolic X-Linked Inborn Sphingolipidoses Metabolism Inborn Errors Lipodoses Lipid Metabolism |
Fabry Disease Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Vascular Diseases |
Cardiovascular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders Sphingolipidoses Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors |