Gene Therapy for Fabry Disease

Recruiting in Palo Alto (17 mi)

+20 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Sangamo Therapeutics

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial tests ST-920, a treatment using a virus to deliver a gene that helps produce an important enzyme in patients with Fabry disease. The goal is to help these patients by continuously making the enzyme to reduce harmful substances in their bodies. ST-920 is a gene therapy treatment for Fabry disease, which aims to deliver a gene to produce the enzyme alpha-galactosidase A, addressing the enzyme deficiency central to the disease.

Eligibility Criteria

Adults with Fabry disease can join this trial. For the cardiac group, they need heart changes like left ventricular hypertrophy or other signs of worsening disease. The renal group requires specific kidney function levels and a history of declining function. All participants must be COVID-19 vaccinated at least one month before treatment.

Inclusion Criteria

My heart shows signs of thickening or disease progression.

I am 18 years old or older.

I have symptoms like vision changes, pain in hands/feet, less sweating, or skin spots.

+3 more

Exclusion Criteria

You have had problems with alcohol or drugs in the recent past.

You had a bad reaction to ERT in the last 6 months.

My heart condition severely limits my daily activities.

+14 more

Participant Groups

The trial is testing ST-920, a gene therapy designed to produce the enzyme α-Gal A in people with Fabry disease. It's given as a single intravenous dose, aiming to reduce or clear harmful substances in the body caused by the disease over a 52-week period.

2Treatment groups

Experimental Treatment

Group I: Sequential dose escalationExperimental Treatment1 Intervention

ST-920 is administered as a single infusion: 1. Cohort 1: 0.5e13 vg/kg 2. Cohort 2: 1.0e13 vg/kg 3. Cohort 3: 3.0e13 vg/kg 4. Cohort 4: 5.0e13 vg/kg

Group II: Expansion CohortsExperimental Treatment1 Intervention

1. Anti Alpha-Gal A Antibody Positive Cohort 2. Anti Alpha-Gal A Antibody Negative Cohort 3. Female Cohort 4. Renal Cohort 5. Cardiac Cohort