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Gene Therapy

Gene Therapy for Fabry Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by Sangamo Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Additional Inclusion Criteria for Cardiac Cohort: Left ventricular hypertrophy (LVH) in 2D echocardiography or CMR, OR presentation with cardiac changes indicative of disease progression
≥ 18 years of age
Must not have
New York Heart Association Class III or higher
Prior treatment with a gene therapy product
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 12 months after the st-920 infusion
Awards & highlights

Summary

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.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Potential side effects are not detailed but may include reactions related to immune response against the viral vector (AAV6) used for gene delivery or components of ST-920 formulation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My heart shows signs of thickening or disease progression.
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I am 18 years old or older.
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I have symptoms like vision changes, pain in hands/feet, less sweating, or skin spots.
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I have been diagnosed with Fabry disease.
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My kidney function is moderately reduced and getting worse each year.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My heart condition severely limits my daily activities.
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I have previously received gene therapy.
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You have had kidney dialysis or transplantation, recent kidney problems, or have started or changed certain medications for kidney issues. Your urine test shows high protein levels and you are not taking specific medications for it.
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I have had cancer before, but only skin cancer (not melanoma) or prostate cancer that was treated to cure it.
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My kidney function is reduced with an eGFR below 40.
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I cannot take corticosteroids due to health reasons.
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I do not have an active infection with hepatitis A, B, C, HIV, or TB.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 12 months after the st-920 infusion
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 12 months after the st-920 infusion 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 of treatment-emergent adverse events (TEAEs)

Trial Design

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

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The ST-920 trial involves a gene therapy using a recombinant AAV2/6 vector to deliver the cDNA for human alpha-galactosidase A (α-Gal A) into patients. This therapy aims to enable the stable, long-term production of the α-Gal A enzyme, which is deficient in Fabry Disease patients. The presence of functional α-Gal A is crucial as it helps break down globotriaosylceramide (Gb3) and lyso-Gb3, substrates that accumulate in the cells of Fabry Disease patients, leading to various symptoms and organ damage. By restoring the enzyme's activity, this treatment can potentially reduce or clear these harmful substrates, improving patient outcomes and quality of life.

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Who is running the clinical trial?

Sangamo TherapeuticsLead Sponsor
28 Previous Clinical Trials
915 Total Patients Enrolled
1 Trials studying Fabry Disease
48 Patients Enrolled for Fabry Disease
Medical MonitorStudy DirectorSangamo Therapeutics, Inc.
1,673 Previous Clinical Trials
988,401 Total Patients Enrolled
13 Trials studying Fabry Disease
808 Patients Enrolled for Fabry Disease

Media Library

ST-920 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04046224 — Phase 1 & 2
Fabry Disease Research Study Groups: Sequential dose escalation, Expansion Cohorts
Fabry Disease Clinical Trial 2023: ST-920 Highlights & Side Effects. Trial Name: NCT04046224 — Phase 1 & 2
ST-920 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04046224 — Phase 1 & 2
~3 spots leftby Apr 2025
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