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Study on Long-Term Safety and Efficacy of Cebsulfase Alfa for Children with Late Metachromatic Leukodystrophy Receiving Intrathecal Enzyme Replacement

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What is this trial about?

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

This clinical trial is focused on studying a rare genetic disorder called Metachromatic Leukodystrophy (MLD). MLD affects the nervous system, leading to a gradual loss of physical and mental abilities. The study is investigating the long-term safety and effectiveness of a treatment called HGT-1110, which is administered through an injection into the spinal fluid, a method known as intrathecal injection. The treatment involves an enzyme replacement therapy using a substance called cebsulfase alfa, which is designed to help manage the symptoms of MLD.

The purpose of this study is to gather information on the long-term safety of HGT-1110 in patients who have previously participated in an earlier phase of the study. Participants will receive regular doses of the treatment and will be monitored for any side effects or changes in their health. The study will also look at how the treatment affects motor function and other aspects of daily living over time.

Throughout the study, participants will undergo various health assessments, including laboratory tests and physical examinations, to ensure their well-being. The presence of any antibodies against the treatment in the body will also be checked. This study aims to provide valuable insights into the potential benefits and risks of using HGT-1110 for managing Metachromatic Leukodystrophy.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation in this study requires prior involvement in Study HGT-MLD-070 through Week 40.

    Eligibility is contingent upon the absence of any safety or medical issues that would prevent participation.

    Written informed consent must be provided by the patient, or by the patient's parent or legally authorized representative, before any study-related activities begin.

  2. Step 2

    Treatment administration

    The treatment involves the administration of HGT-1110, also known as cebsulfase alfa, which is delivered through an injection into the space around the spinal cord, a method known as intrathecal use.

    The medication is provided in the form of a solution for injection.

  3. Step 3

    Monitoring safety

    Safety is assessed by monitoring any adverse events that occur during treatment.

    Changes from the initial state in laboratory tests, including blood and urine tests, are evaluated.

    Vital signs and physical examinations are conducted regularly to ensure safety.

    The presence of antibodies against HGT-1110 in the cerebrospinal fluid (CSF) and blood is determined.

  4. Step 4

    Evaluating effectiveness

    The study measures changes in motor function using specific assessments, although some assessments may not be conducted if certain conditions are met.

    Changes in adaptive behavior are evaluated, although some assessments may not be conducted after specific dates.

    Caregiver observations of the patient's functioning are recorded and analyzed.

    The concentration of HGT-1110 in the CSF is measured at selected times after repeated administration.

  5. Step 5

    Study duration

    The study is expected to conclude by December 31, 2024.

Who can join the trial?

5 criteria

  • The patient must have participated in a previous study called Study HGT-MLD-070 up to Week 40.
  • The patient must not have any safety or medical issues that would prevent them from participating in the study.
  • The patient, or the patient's parent or legally authorized representative, must provide written permission to participate in the study. This is called informed consent, which means they understand the study and agree to take part.
  • The study is open to both male and female patients.
  • The study includes patients who are considered part of a vulnerable population, which means they may need extra protection or care.

Who cannot join the trial?

1 criterion

  • Patients who have not participated in Study HGT-MLD-070 through Week 40 cannot join this study.
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Investigated drugs

HGT-1110 is a medication being studied for its long-term safety and effectiveness in patients with Metachromatic Leukodystrophy (MLD). It is administered directly into the spinal fluid through a procedure called intrathecal administration. This method allows the medication to reach the central nervous system more effectively, which is important for treating conditions like MLD that affect the brain and spinal cord. The study aims to gather information on how safe this treatment is for patients over an extended period.

What is already known about the treatment

HGT-1110 – This medication is administered intrathecally, meaning it is delivered directly into the spinal canal. It is currently being studied in clinical trials for its long-term safety and efficacy in treating patients with Late Metachromatic Leukodystrophy (MLD). The main therapeutic indication is to manage and potentially slow the progression of MLD, a rare genetic disorder affecting the nervous system. At the molecular level, HGT-1110 is designed to address the underlying enzyme deficiency that causes the disease. It falls under the pharmacological classification of enzyme replacement therapies.

Investigated diseases

Late Metachromatic Leukodystrophy – This is a rare genetic disorder that affects the nervous system, primarily impacting the white matter of the brain and spinal cord. It is caused by a deficiency in the enzyme arylsulfatase A, leading to the accumulation of sulfatides, which are toxic to the nervous system. As the disease progresses, individuals may experience a decline in motor skills, muscle weakness, and difficulties with coordination and balance. Cognitive functions, such as memory and problem-solving, may also deteriorate over time. The progression of symptoms can vary, but they generally worsen as the disease advances.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase ITrial ID2024-514403-34-00Protocol codeHGT-MLD-071Estimated enrolment16 patientsSponsorShire Human Genetic Therapies Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).