Gene Therapy for Fabry Disease

Recruiting in Palo Alto (17 mi)

+7 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: 4D Molecular Therapeutics

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

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.

Inclusion Criteria

I am 18 years old or older.

I have been on the same dose of enzyme replacement therapy for at least 6 months.

Agree to use highly effective contraception

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Exclusion Criteria

I do not have HIV or active/chronic hepatitis B or C.

I have severe liver, lung disease, or poorly controlled diabetes.

I haven't had cancer in the past 2 years, except for non-melanoma skin cancer or treated prostate cancer.

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Treatment Details

Interventions

4D-310 (Gene Therapy)

Trial OverviewThe 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.

Participant Groups

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