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Rebisufligene Etisparvovec

Rebisufligene Etisparvovec, also known as ABO-102 or UX111, is an innovative gene therapy being studied in clinical trials for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA). This rare genetic disorder affects children and is characterized by progressive neurological deterioration. The clinical trials aim to evaluate the safety and effectiveness of this gene therapy, which is administered as a single intravenous injection to potentially restore the metabolic pathway damaged in MPS IIIA patients.

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What is REBISUFLIGENE ETISPARVOVEC?

REBISUFLIGENE ETISPARVOVEC, also known as ABO-102, scAAV9.U1a.hSGSH, or UX111, is a promising gene therapy medication designed to treat Mucopolysaccharidosis Type IIIA (MPS IIIA)[1]. It is a suspension for injection that contains a specially engineered gene therapy vector. This innovative treatment aims to address the underlying genetic cause of MPS IIIA, offering hope to patients and families affected by this rare genetic disorder.

Target Condition: Mucopolysaccharidosis Type IIIA

Mucopolysaccharidosis Type IIIA, also known as Sanfilippo Syndrome Type A, is a rare genetic disorder that affects the body’s ability to break down certain complex sugar molecules[1]. This condition is caused by a deficiency in an enzyme called N-sulfoglucosamine sulfohydrolase (SGSH). As a result, harmful substances accumulate in the body, particularly affecting the brain and nervous system, leading to progressive cognitive decline and various other symptoms.

How REBISUFLIGENE ETISPARVOVEC Works

REBISUFLIGENE ETISPARVOVEC is a gene therapy that works by delivering a functional copy of the SGSH gene to cells in the patient’s body[1]. The therapy uses a modified virus called AAV9 as a vector to carry the corrective gene. This vector is designed to cross the blood-brain barrier (BBB), allowing it to reach cells in the central nervous system.

Once inside the cells, the therapy enables the production of the missing N-sulfoglucosamine sulfohydrolase enzyme. This helps to restore the metabolic pathway that is damaged in MPS IIIA patients, potentially slowing or halting the progression of the disease.

Clinical Trials and Research

REBISUFLIGENE ETISPARVOVEC is currently being studied in clinical trials to evaluate its safety and effectiveness in treating MPS IIIA[1]. The ongoing research includes:

  • A Phase I/II/III integrated clinical trial to assess the drug’s efficacy and safety
  • Studies involving different age groups, from infants to children up to 5 years old
  • Evaluation of various dosages to determine the optimal treatment regimen

Administration and Treatment Process

REBISUFLIGENE ETISPARVOVEC is administered as a single intravenous injection[1]. This means that the medication is given directly into a vein, allowing it to circulate throughout the body. The treatment is designed to be a one-time therapy, potentially providing long-lasting benefits to patients with MPS IIIA.

Safety and Efficacy

The primary goals of the clinical trials for REBISUFLIGENE ETISPARVOVEC are to assess its safety and efficacy[1]. Researchers are monitoring:

  • The incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
  • Changes in cerebrospinal fluid (CSF) heparan sulfate (HS) levels, which is a key indicator of the therapy’s effectiveness
  • Improvements in cognitive function, as measured by standardized tests like the Bayley Scales of Infant and Toddler Development (BSITD-III)
  • Changes in brain structure, such as total cortical volume

Eligibility for Treatment

Eligibility for participation in the clinical trials for REBISUFLIGENE ETISPARVOVEC includes[1]:

  • Confirmed diagnosis of MPS IIIA through genetic testing and enzyme activity analysis
  • Specific age requirements, ranging from infants to children up to 5 years old, depending on the study cohort
  • No prior gene therapy, cell therapy, or enzyme replacement therapy for MPS IIIA
  • Absence of certain medical conditions that could interfere with the treatment or study procedures

Long-Term Follow-Up Study

To further evaluate the long-term safety and effectiveness of REBISUFLIGENE ETISPARVOVEC, a separate long-term follow-up study is being conducted[2]. This study aims to:

  • Monitor the long-term safety and tolerability of the treatment
  • Assess the durability of the treatment’s effects on cognitive function and disease progression
  • Gather additional data on the therapy’s impact on quality of life for patients with MPS IIIA

REBISUFLIGENE ETISPARVOVEC represents a significant advancement in the treatment of Mucopolysaccharidosis Type IIIA. As research continues, this innovative gene therapy offers hope for improved outcomes and quality of life for individuals affected by this rare genetic disorder.

Aspect Details
Drug Name Rebisufligene Etisparvovec (ABO-102, UX111)
Condition Treated Mucopolysaccharidosis Type IIIA (MPS IIIA)
Administration Method Single intravenous injection
Trial Phase Phase I/II/III Integrated Clinical Trial
Primary Objectives Evaluate safety and efficacy
Key Efficacy Measures CSF heparan sulfate levels, cognitive function scores
Safety Monitoring Incidence of adverse events and serious adverse events
Participant Age Range From birth/3 months to 5 years (varies by cohort)
Long-term Follow-up Separate study to monitor long-term safety and efficacy

FAQ

  • What is Rebisufligene Etisparvovec? Rebisufligene Etisparvovec is a gene therapy designed to treat Mucopolysaccharidosis Type IIIA (MPS IIIA). It uses a modified virus to deliver a healthy copy of the N-sulfoglucosamine sulfohydrolase gene to cells, potentially restoring the damaged metabolic pathway in patients with MPS IIIA.
  • How is Rebisufligene Etisparvovec administered? Rebisufligene Etisparvovec is administered as a single intravenous injection. This means it's given directly into the bloodstream through a vein, allowing the therapy to circulate throughout the body.
  • What are the main objectives of the clinical trials? The main objectives of the clinical trials are to evaluate the safety and effectiveness of Rebisufligene Etisparvovec in patients with MPS IIIA. This includes monitoring for any side effects and measuring improvements in cognitive function and certain biomarkers associated with the disease.
  • Who can participate in these clinical trials? Participants must have a confirmed diagnosis of MPS IIIA and meet specific age and cognitive function criteria. The trials include both very young children and those up to 5 years old, depending on the specific cohort.
  • What is the long-term follow-up study? The long-term follow-up study is designed to monitor patients who have previously received Rebisufligene Etisparvovec in earlier clinical trials. It aims to evaluate the long-term safety and effectiveness of the therapy over an extended period.

Ongoing Clinical Trials on Rebisufligene Etisparvovec

  • Study on the Safety and Effectiveness of Rebisufligene Etisparvovec for Patients with Mucopolysaccharidosis Type IIIA

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Spain

  • Long-term safety study of rebisufligene etisparvovec gene therapy in patients previously treated for Mucopolysaccharidosis type IIIA

    Recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Spain

Glossary

  • Mucopolysaccharidosis Type IIIA (MPS IIIA): A rare genetic disorder characterized by the body's inability to break down certain complex sugars, leading to progressive neurological deterioration.
  • Gene Therapy: A technique that uses genes to treat or prevent disease. In this case, it involves introducing a healthy copy of a gene to compensate for a faulty one.
  • Rebisufligene Etisparvovec: The gene therapy being studied, also known as ABO-102 or UX111, designed to treat MPS IIIA by delivering a functional copy of the N-sulfoglucosamine sulfohydrolase gene.
  • Intravenous (IV) Administration: A method of giving medications or fluids directly into a vein.
  • Cerebrospinal Fluid (CSF): A clear, colorless fluid that surrounds the brain and spinal cord, often used to measure biomarkers of disease progression or treatment effectiveness.
  • Heparan Sulfate (HS): A complex sugar that accumulates in the bodies of patients with MPS IIIA, used as a biomarker to assess the effectiveness of treatment.
  • Bayley Scales of Infant and Toddler Development (BSITD-III): A standardized test used to assess cognitive, language, and motor development in young children.
  • Adverse Event (AE): Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.
  • Serious Adverse Event (SAE): An adverse event that results in death, is life-threatening, requires hospitalization, or causes significant disability.
  • Cohort: A group of participants in a study who share a common characteristic or experience within a defined period.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-rebisufligene-etisparvovec-for-patients-with-mucopolysaccharidosis-type-iiia/
  2. http://clinicaltrials.eu/trial/long-term-safety-study-of-rebisufligene-etisparvovec-for-patients-with-mucopolysaccharidosis-type-iiia/