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

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What is this study about?

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.

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 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 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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Careggi Florence Italy

Other Sites

Site Name City Country Status
Utpcnbiwom Mlqtfda Chzpsl Haqgnnxhtvsmyezmv Hamburg Germany
Uscumshmqomcpskyqhawx Wvurigqkj Ate Wuerzburg Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Germany Germany
Not recruiting
23.06.2023
Italy Italy
Not recruiting
23.06.2023

Trial locations

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.

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 ID:
2024-512695-34-00
Protocol code:
ST-920-201
Trial Phase:
Phase I and Phase II (Integrated) – First administration to humans

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