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 study about?

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.

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 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 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 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 study duration

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

Who Can Join the Study?

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 Study?

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

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Marien-Hospital Wesel gGmbH	Wesel	Germany
Centre Hospitalier Universitaire d’Orléans	Orléans	France
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Fakultni Nemocnice Brno	Brno	Czechia
Hospital Femme Mere Enfant	Bron	France
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Region Midtjylland	Aarhus	Denmark
Rigshospitalet	Copenhagen	Denmark
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Ssf Zfhywrnomoyqrvg Sqfw Grbl	Suhl	Germany
Uxcrqbfnatd Wwruhldhiqbatyw &atbpxb Vylxmjpeg Kmbsxek uwf Jsbbdqjyinbv Deldmby	Datteln	Germany
Cnwzsc Hmfrvnwkksp Rdndbxda Dzlivamjuceggy	Angers	France
Cvs Kpaqgip Bwwsily	Le Kremlin-Bicetre	France

Trial status

Country	Status	Recruitment Start
Czechia	Not recruiting	01.04.2013
Denmark	Not recruiting	01.04.2013
France	Not recruiting	01.04.2013
Germany	Not recruiting	01.04.2013
Italy	Not recruiting	01.04.2013

Trial locations

Investigated drugs:

Cebsulfase Alfa

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.

Investigated diseases:

Metachromatic leukodystrophy

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 ID:

2024-514403-34-00

Protocol code:

HGT-MLD-071

NCT ID:

NCT01887938

Trial Phase:

Human Pharmacology (Phase I) – Other

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

What is this study about?

Who Can Join the Study?

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