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Isaralgagene Civaparvovec

Isaralgagene Civaparvovec, also known as ST-920, is an innovative gene therapy being studied in clinical trials for the treatment of Fabry Disease. This article will provide an overview of the ongoing research, including the study objectives, eligibility criteria, and potential benefits for patients with this rare genetic disorder.

Table of Contents

What is ISARALGAGENE CIVAPARVOVEC?

ISARALGAGENE CIVAPARVOVEC, also known as ST-920, is an innovative gene therapy being developed to treat Fabry disease. It is a recombinant adeno-associated virus 2/6 vector that carries the genetic code for producing a crucial enzyme missing in people with Fabry disease[1]. This treatment is currently being studied in clinical trials to assess its safety and effectiveness.

Fabry Disease: An Overview

Fabry disease is a rare genetic disorder classified as an X-linked lysosomal storage disease. People with this condition lack or have insufficient amounts of an enzyme called alpha-galactosidase A (α-Gal A). This deficiency leads to the buildup of certain fatty substances in various organs, causing a wide range of symptoms and complications[1].

Some common symptoms of Fabry disease include:

  • Cornea verticillata (clouding of the eye’s cornea)
  • Acroparesthesia (burning pain in hands and feet)
  • Anhidrosis (decreased ability to sweat)
  • Angiokeratoma (small, dark red spots on the skin)

How ISARALGAGENE CIVAPARVOVEC Works

ISARALGAGENE CIVAPARVOVEC is designed to deliver a functional copy of the GLA gene, which codes for the α-Gal A enzyme, directly to the patient’s cells. The treatment uses a modified virus (AAV2/6) as a vehicle to carry this genetic material. Once inside the body, the goal is for the cells to use this new genetic information to produce the missing enzyme, potentially alleviating the symptoms of Fabry disease[1].

Clinical Trial Information

The ongoing clinical trial for ISARALGAGENE CIVAPARVOVEC is a Phase I/II study aimed at assessing the safety and tolerability of the treatment in patients with Fabry disease. Here are some key points about the trial[1]:

  • It is a multicenter, open-label, single-dose, dose-ranging study.
  • The main objective is to evaluate the safety and tolerability of ST-920.
  • Secondary objectives include assessing the treatment’s impact on α-Gal A activity, enzyme replacement therapy (ERT) requirements, renal function, and cardiac function.
  • The study will also monitor the presence of the vector in various bodily fluids over time.

Potential Benefits

If successful, ISARALGAGENE CIVAPARVOVEC could offer several potential benefits for Fabry disease patients[1]:

  • Increased α-Gal A enzyme activity in the body
  • Reduction in harmful substance accumulation (Gb3 and lyso-Gb3) in plasma
  • Possible reduction in the frequency of enzyme replacement therapy (ERT) infusions
  • Potential improvements in kidney function
  • Possible enhancements in heart function and structure

Safety and Side Effects

As with any new treatment, safety is a primary concern. The clinical trial is closely monitoring for any adverse events. Some areas of focus include[1]:

  • Routine blood tests to check for any changes in blood cell counts or organ function
  • Regular heart monitoring through ECG and echocardiogram
  • Liver function tests and imaging to ensure liver health
  • Monitoring for any immune reactions to the treatment

Who is Eligible for the Treatment?

The clinical trial has specific eligibility criteria. Some key points include[1]:

  • Patients must be 18 years or older
  • Diagnosed with Fabry disease
  • May be on enzyme replacement therapy (ERT) or ERT-naïve
  • Must have certain Fabry disease symptoms
  • Specific criteria for different study cohorts (e.g., renal function requirements for the renal cohort)

There are also several exclusion criteria, such as certain medical conditions or treatments that might interfere with the study results or pose additional risks.

How is the Treatment Administered?

ISARALGAGENE CIVAPARVOVEC is administered as a single intravenous (IV) infusion. This means the treatment is given directly into a vein, typically in a controlled medical setting. After administration, patients are closely monitored for any immediate reactions and to track the treatment’s effectiveness over time[1].

Conclusion

ISARALGAGENE CIVAPARVOVEC represents a promising advancement in the treatment of Fabry disease. As a gene therapy, it aims to address the root cause of the condition by providing the body with the ability to produce the missing enzyme. While the treatment is still in clinical trials and more research is needed to fully understand its long-term effects and benefits, it offers hope for improved management of Fabry disease in the future.

Aspect Details
Study Type Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging
Main Objective Assess safety and tolerability of ST-920
Secondary Objectives Evaluate enzyme activity, substrate levels, impact on ERT, renal and cardiac function
Key Eligibility Criteria Adults (≥18 years) with Fabry Disease diagnosis, specific criteria for different cohorts
Treatment Administration Single-dose intravenous injection/infusion
Primary Endpoints Incidence of treatment-emergent adverse events, safety evaluations
Secondary Endpoints Changes in enzyme activity, substrate levels, ERT frequency, organ function

FAQ

  • What is Isaralgagene Civaparvovec (ST-920)? Isaralgagene Civaparvovec, or ST-920, is a gene therapy product designed to treat Fabry Disease. It uses a modified virus (AAV2/6) to deliver a functional copy of the alpha-galactosidase A gene to patients' cells, potentially addressing the underlying cause of the disease.
  • What is the main goal of the clinical trial for ST-920? The primary objective of the clinical trial is to assess the safety and tolerability of ST-920 in patients with Fabry Disease. Researchers are also looking at how the therapy affects enzyme activity, substrate levels, and various aspects of organ function.
  • Who is eligible to participate in the ST-920 clinical trial? Eligible participants include adults (18 years or older) with a confirmed diagnosis of Fabry Disease. The trial includes both male and female patients, with specific criteria for different cohorts based on disease severity and organ involvement.
  • How is ST-920 administered to patients? ST-920 is administered as a single-dose treatment through intravenous injection or infusion. The study is designed to test different dose levels to determine the most effective and safe dosage.
  • What are the potential benefits of ST-920 for Fabry Disease patients? If successful, ST-920 could potentially provide long-term production of the alpha-galactosidase A enzyme, which is deficient in Fabry Disease. This may lead to improved organ function, reduced disease symptoms, and a decreased need for enzyme replacement therapy (ERT) in some patients.

Ongoing Clinical Trials on Isaralgagene Civaparvovec

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

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

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

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Germany Italy

Glossary

  • Fabry Disease: A rare genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A, leading to the accumulation of certain fats in various organs and tissues.
  • Alpha-galactosidase A (α-Gal A): An enzyme that breaks down specific fat molecules in the body. Its deficiency is the cause of Fabry Disease.
  • Gene therapy: A treatment approach that involves introducing genetic material into a person's cells to compensate for abnormal genes or to produce a beneficial protein.
  • AAV2/6: A type of adeno-associated virus used as a vector to deliver the therapeutic gene in ST-920.
  • Enzyme Replacement Therapy (ERT): A treatment method for Fabry Disease that involves regular infusions of the missing enzyme to help manage symptoms.
  • Gb3 and lyso-Gb3: Substances that accumulate in the body of Fabry Disease patients due to the lack of alpha-galactosidase A enzyme.
  • eGFR (estimated glomerular filtration rate): A measure of kidney function used to assess the progression of Fabry Disease.
  • Left ventricular mass index (LVMI): A measure of heart muscle thickness, which can be affected in Fabry Disease.
  • Acroparesthesia: Burning pain and tingling sensations in the hands and feet, a common symptom of Fabry Disease.
  • Anhidrosis: Decreased or absent sweating, which can occur in Fabry Disease patients.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-st-920-gene-therapy-for-patients-with-fabry-disease/