Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study on the Safety and Tolerability of ST-920 Gene Therapy for Patients with Fabry Disease

Verified siteInvestigationalNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Fabry Disease, a genetic condition that affects the body's ability to break down a specific type of fat, leading to a buildup in various organs. The study is testing a new treatment called ST-920, which is a type of gene therapy. This treatment uses a specially designed virus to deliver a healthy copy of a gene that helps produce an enzyme called alpha-galactosidase A, which is missing or not working properly in people with Fabry Disease.

The purpose of the study is to assess the safety and tolerability of ST-920. Participants in the study will receive a single dose of this treatment through an injection into a vein. The study will monitor participants over a period of time to see how their bodies respond to the treatment. This includes regular check-ups and tests to ensure the treatment is safe and to observe any changes in the symptoms of Fabry Disease.

Throughout the study, researchers will collect information on any side effects that may occur and will perform various tests, such as blood tests and imaging scans, to monitor the health of participants. The study aims to provide valuable information on whether ST-920 can be a safe and effective treatment option for people living with Fabry Disease.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a diagnosis of Fabry Disease and meeting specific criteria such as age and health status.

    A negative pregnancy test is required for female participants, and all participants must be fully vaccinated against COVID-19 at least one month prior to dosing.

  2. Step 2

    Consent and preparation

    Participants provide signed, written informed consent to participate in the study.

    Participants are informed about the need for effective contraception and restrictions on sperm or egg donation during the study period.

  3. Step 3

    Administration of ST-920

    The study involves a single dose of ST-920, administered intravenously as a solution for injection or infusion.

    The main objective is to assess the safety and tolerability of this gene therapy.

  4. Step 4

    Monitoring and follow-up

    Participants are monitored for treatment-emergent adverse events. This includes routine blood tests, liver function tests, and heart monitoring through ECG and echocardiogram.

    Additional tests such as alpha fetoprotein (AFP) and liver imaging are conducted to monitor for any liver-related issues.

  5. Step 5

    Evaluation of treatment effects

    Over a one-year period, changes from baseline in various health indicators are evaluated. This includes alpha-Gal A activity, levels of certain substances in the blood, and kidney function.

    Heart function is assessed through cardiac imaging, and the clearance of the ST-920 vector is measured in various bodily fluids.

  6. Step 6

    Completion of study

    The study is expected to conclude by March 2025, with all participants completing the required follow-up assessments.

    Final evaluations are conducted to determine the overall safety and effectiveness of the treatment.

Who can join the trial?

16 criteria

  • Must be **18 years of age** or older.
  • Must have a **diagnosis of Fabry disease**.
  • Must have one or more of the following symptoms:
    • **Cornea verticillata**: a pattern on the eye's surface.
    • **Acroparesthesia**: tingling or burning sensations in the hands or feet.
    • **Anhidrosis**: reduced ability to sweat.
    • **Angiokeratoma**: small, dark spots on the skin.
    • Must sign a written **informed consent** form, agreeing to participate in the study.
    • Can be on **ERT** (enzyme replacement therapy), have never received ERT, or have stopped ERT. If on ERT, it must have been taken regularly for at least 6 months.
    • **Male subjects** must agree to use effective contraception and not donate sperm until certain conditions are met after receiving the study treatment.
    • **Female subjects** must not donate eggs until certain conditions are met after receiving the study treatment.
    • **Female subjects** who can become pregnant must have a negative pregnancy test and use effective contraception during the study.
    • Must be **fully vaccinated for COVID-19** at least one month before starting the study.
    • For certain groups, **male subjects** must have classical Fabry disease with very low **α-Gal A activity** in blood tests.
    • For certain groups, **female subjects** must have a known genetic change linked to classical Fabry disease and need treatment.
    • For kidney and heart groups, **male subjects** must have low **α-Gal A activity**, and **female subjects** must have genetic test results showing Fabry disease.
    • For the kidney group, must have a specific range of **eGFR** (a kidney function test) and a certain pattern in eGFR results.

Who cannot join the trial?

3 criteria

  • Patients who are not male cannot participate.
  • Patients who are under 18 years old cannot participate.
  • Patients who are part of a vulnerable population, meaning they might need special protection or care, cannot participate.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

ST-920 is a gene therapy being studied for its potential to treat Fabry Disease. This therapy uses a specially designed virus to deliver a healthy copy of the human alpha-galactosidase A gene into the patient's cells. The goal is to help the body produce the enzyme that is missing or not working properly in people with Fabry Disease, potentially reducing symptoms and improving quality of life.

What is already known about the treatment

ST-920 – This medication is administered as a single-dose gene therapy through intravenous infusion. It is currently in Phase I/II clinical trials, focusing on its safety and tolerability in treating Fabry Disease, a genetic disorder. The main therapeutic indication for ST-920 is Fabry Disease, which is a condition caused by the buildup of a specific type of fat in the body's cells. The mechanism of action involves delivering a functional copy of the human alpha-galactosidase A gene to the patient's cells, aiming to restore the enzyme's activity and reduce the accumulated fat. ST-920 is classified as a gene therapy product, representing a novel approach in the treatment of genetic disorders.

Investigated diseases

Fabry Disease – Fabry Disease is a rare genetic disorder caused by the buildup of a specific type of fat in the body's cells. This condition is due to a deficiency of an enzyme called alpha-galactosidase A, which leads to the accumulation of globotriaosylceramide (Gb3) in various tissues. Symptoms often begin in childhood or adolescence and can include pain in the hands and feet, clusters of small, dark red spots on the skin, and problems with sweating. Over time, the disease can affect the kidneys, heart, and nervous system, leading to more severe complications. The progression of Fabry Disease varies among individuals, with some experiencing mild symptoms and others facing significant health challenges. It is an X-linked disorder, meaning it primarily affects males, although females can also be affected.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-512695-34-00Protocol codeST-920-201Estimated enrolment46 patientsSponsorSangamo Therapeutics Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.