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Gene Therapy

Gene Therapy for Fabry Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by 4D Molecular Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of classic or late-onset Fabry disease
Pathogenic GLA mutation consistent with Fabry Disease
Must not have
HIV, active or chronic hepatitis B or C
Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug for Fabry Disease in adult males. The goal is to see if it is safe and effective.

Who is the study for?
Adults with Fabry Disease can join this trial. They must have a confirmed diagnosis, be on stable enzyme replacement therapy if applicable, and not have severe diseases like liver or lung disease, recent strokes, or uncontrolled diabetes. Participants need to agree to use effective contraception and cannot be pregnant or breastfeeding.
What is being tested?
The trial is testing the safety and effects of a single IV dose of gene therapy called 4D-310 in adults with Fabry Disease. It's an open-label study where everyone knows they're getting the treatment, which will vary in dosage as researchers find the best level.
What are the potential side effects?
Possible side effects may include reactions related to immune response due to gene therapy such as fever, fatigue, muscle pain; potential liver issues since patients with existing liver conditions are excluded; also infusion-related responses similar to those seen with enzyme replacement therapies.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with classic or late-onset Fabry disease.
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I have a genetic mutation linked to Fabry Disease.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I do not have HIV or active/chronic hepatitis B or C.
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I have severe liver, lung disease, or poorly controlled diabetes.
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I am currently in a trial for a new treatment or have had gene therapy.
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I have serious heart disease or high blood pressure that isn't under control.
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I have not had a stroke, mini-stroke, or major blood clot in the past year.
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I had a bad reaction to enzyme replacement therapy that made me stop it.
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I have been taking oral steroids for 3 months or more in the past year.
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I have had a kidney transplant or am currently on dialysis.
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I cannot take systemic steroids or immunosuppressive drugs due to health reasons.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence and severity of adverse events
Secondary study objectives
Change from baseline in serum AGA activity
Change from baseline serum globotriaosylsphingosine (lysoGb3)

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups
Experimental Treatment
Group I: 4D-310 Dose Level 2 - AAV NAb Titer Group A and/or BExperimental Treatment1 Intervention
Single IV administration of 4D-310 at Dose Level 2 in AAV NAb titer Group A and/or B patients
Group II: 4D-310 Dose Level 1 - AAV Neutralizing Antibody (NAb) Group AExperimental Treatment1 Intervention
Single IV administration of 4D-310 Dose Level 1 - AAV NAb Titer Group A patients
Group III: 4D-310 Dose Level 1 - AAV NAb Titer Group BExperimental Treatment1 Intervention
Single IV administration of 4D-310 Dose Level 1 - AAV NAb titer Group B patients
Group IV: 4D-310 Dose ExpansionExperimental Treatment1 Intervention
Dose expansion cohort of single IV administration of 4D-310 at the selected dose and selected AAV Nab titer group(s) patients

Find a Location

Who is running the clinical trial?

4D Molecular TherapeuticsLead Sponsor
7 Previous Clinical Trials
436 Total Patients Enrolled
1 Trials studying Fabry Disease
18 Patients Enrolled for Fabry Disease
Raphael Schiffmann, MD, MHSc, FAANStudy Director4D Molecular Therapeutics
Mitra Tavakkoli, MD, PharmDStudy Director4D Molecular Therapeutics
2 Previous Clinical Trials
58 Total Patients Enrolled
1 Trials studying Fabry Disease
18 Patients Enrolled for Fabry Disease

Media Library

4D-310 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04519749 — Phase 1 & 2
Fabry Disease Research Study Groups: 4D-310 Dose Level 1 - AAV Neutralizing Antibody (NAb) Group A, 4D-310 Dose Level 2 - AAV NAb Titer Group A and/or B, 4D-310 Dose Expansion, 4D-310 Dose Level 1 - AAV NAb Titer Group B
Fabry Disease Clinical Trial 2023: 4D-310 Highlights & Side Effects. Trial Name: NCT04519749 — Phase 1 & 2
4D-310 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04519749 — Phase 1 & 2
~4 spots leftby Jan 2026
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