Not recruitingRare disease

Long-Term Safety Study of ST-920 Gene Therapy for Patients with Fabry Disease

Verified siteInvestigationalNo placebo

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What is this trial about?

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

This clinical trial focuses on Fabry Disease, a genetic condition that affects the body's ability to break down certain fatty substances. The study involves a treatment called ST-920, which is a type of gene therapy. This therapy uses a specially designed virus to deliver 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 evaluate the long-term safety of ST-920. Participants in this study have previously received the ST-920 treatment in an earlier trial. The study will monitor these participants over an extended period to observe any potential side effects or adverse events that may occur as a result of the treatment.

Throughout the study, participants will undergo regular check-ups and assessments to ensure their well-being and to gather data on the treatment's safety. This long-term follow-up is crucial to understanding how the treatment affects patients over time and to ensure that it remains a safe option for managing Fabry Disease.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This involves verifying that the individual has previously received ST-920 therapy in a separate parent trial and has consented to participate in this long-term follow-up study.
Step 2
Administration of ST-920
ST-920 is a gene therapy administered as a solution for injection/infusion. The specific dosage and frequency are determined based on the individual's previous treatment in the parent trial. The administration route is categorized as 'other use,' indicating a specialized method tailored to the therapy's requirements.
Step 3
Monitoring and follow-up
The primary objective of the study is to evaluate the long-term safety of ST-920. This involves regular monitoring for any adverse events, which are unexpected medical occurrences that may arise during the study. The severity and frequency of these events are assessed to ensure the safety of the therapy over time.
Step 4
Completion of study
The study is estimated to conclude by March 1, 2040. Throughout the study duration, participants will continue to be monitored to gather comprehensive data on the long-term effects and safety of ST-920 therapy.

Who can join the trial?

4 criteria

Must have received **ST-920 therapy** in a previous related study. This means you participated in an earlier study where you were given a specific treatment called ST-920.
Must agree to take part in this **LTFU study**. LTFU stands for Long-Term Follow-Up, which means the study will continue to check on your health over a longer period.
Must be **male**. Only males are eligible to participate in this study.
Must be within a certain **age range**. The study is open to individuals who are adults, typically aged 18 and older.

Who cannot join the trial?

3 criteria

Only males can participate in the study. Females are not eligible.
Participants must be within certain age ranges, specifically adults and older adults.
Individuals with certain vulnerabilities or special needs are not eligible to participate.

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Verified sites

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Universitaetsklinikum Wuerzburg AöR
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Wuerzburg, Germany

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Investigated drugs

ST-920 is a gene therapy designed to treat Fabry disease. It uses a modified virus to deliver a healthy copy of the gene responsible for producing the enzyme alpha-galactosidase A. This enzyme is crucial for breaking down certain fats in the body. The therapy aims to provide a long-term solution by enabling the patient's own cells to produce the enzyme, potentially reducing symptoms and preventing disease progression.

Isaralgagene Civaparvovec

What is already known about the treatment

ST-920 – This medication is administered as a gene therapy, specifically designed for the treatment of Fabry Disease, a rare genetic disorder. Currently, ST-920 is under clinical investigation to assess its long-term safety and effectiveness. The main therapeutic indication for ST-920 is to address the underlying cause of Fabry Disease by delivering a functional copy of the human alpha-galactosidase A gene. This gene therapy works at the molecular level by using an adeno-associated virus (AAV2/6) vector to introduce the correct gene into the patient's cells, enabling them to produce the enzyme that is deficient in individuals with Fabry Disease. ST-920 is classified under gene therapies, which are a cutting-edge approach in modern medicine aimed at treating genetic disorders by correcting the underlying genetic defects.

Investigated diseases

Fabry Disease – Fabry Disease is a 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. Symptoms often begin in childhood or adolescence and can include pain, particularly in the hands and feet, as well as skin rashes. Over time, the disease can affect various organs, including the kidneys, heart, and nervous system. The progression of Fabry Disease can vary widely among individuals, with some experiencing more severe symptoms than others. It is an X-linked condition, meaning it primarily affects males, although females can also be affected.