What side effects are associated with this type of intervention?

Treatments for Fabry Disease, such as gene therapy and enzyme replacement therapy (ERT), have specific side effects. Gene therapy, like the ST-920 trial, can cause immune reactions to the viral vector, liver enzyme elevations, and potential long-term risks such as insertional mutagenesis. ERT, which involves regular infusions of the α-Gal A enzyme, can lead to infusion-related reactions including fever, chills, rash, and occasionally severe allergic reactions. Both treatments aim to reduce harmful substrate accumulation but come with their own risks.

What prior FDA approvals exist?

The FDA has approved several treatments for Fabry Disease, primarily focusing on enzyme replacement therapies (ERTs). These include agalsidase beta (Fabrazyme) and agalsidase alfa (Replagal), which provide the deficient α-Gal A enzyme. More recently, the FDA approved migalastat (Galafold), an oral pharmacological chaperone that stabilizes the body's own dysfunctional α-Gal A enzyme. While these treatments do not involve gene therapy, they aim to address the enzyme deficiency central to Fabry Disease. The ST-920 trial represents a novel approach by using a recombinant AAV2/6 vector to enable the body to produce α-Gal A enzyme continuously.

What other types of research exist?

Promising novel alternatives to ST-920 for Fabry Disease patients include gene editing techniques such as CRISPR-Cas9, which can potentially provide a permanent correction of the genetic defect. Additionally, RNA silencing therapies using siRNA or antisense oligonucleotides (ASOs) to reduce the production of harmful substrates are also being explored. These approaches aim to offer more durable and effective treatments compared to traditional enzyme replacement therapies.

← Back to Search

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

No Placebo-Only Group

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

Select...

My heart shows signs of thickening or disease progression.

Select...

I am 18 years old or older.

Select...

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

Select...

I have been diagnosed with Fabry disease.

Select...

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:

Select...

My heart condition severely limits my daily activities.

Select...

I have previously received gene therapy.

Select...

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.

Select...

I have had cancer before, but only skin cancer (not melanoma) or prostate cancer that was treated to cure it.

Select...

My kidney function is reduced with an eGFR below 40.

Select...

I cannot take corticosteroids due to health reasons.

Select...

I do not have an active infection with hepatitis A, B, C, HIV, or TB.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 12 months after the st-920 infusion

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)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

0 / 12Eligibility criteria met

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.