Study of Weekly Infusions of JR-441 for Patients with Mucopolysaccharidosis Type IIIA

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What is this study about?

This clinical trial is focused on studying a rare genetic disorder called Mucopolysaccharidosis type IIIA (MPS IIIA). MPS IIIA is a condition that affects the body’s ability to break down certain sugar molecules, leading to various health issues. The treatment being tested in this study is called JR-441, which is a special type of medication designed to help manage the symptoms of MPS IIIA. JR-441 is a lyophilized powder, which means it is a freeze-dried substance that is prepared for injection. It works by using a protein that targets specific receptors in the body to help improve the condition.

The purpose of this study is to evaluate the safety and explore the effectiveness of JR-441 in patients with MPS IIIA. Participants in the study will receive weekly infusions of JR-441. The study will monitor the occurrence of any side effects and changes in health indicators such as blood tests, vital signs, and heart function. The study will also look at how the drug is processed in the body and its impact on cognitive function and behavior.

This trial is expected to run until 2030, with the aim of gathering comprehensive data on the safety and potential benefits of JR-441 for individuals with MPS IIIA. Participants will be closely monitored throughout the study to ensure their well-being and to collect valuable information that could lead to improved treatments for this challenging condition.

1 joining the study

Upon joining the study, the patient will receive an initial assessment to confirm eligibility. This includes verifying the diagnosis of Mucopolysaccharidosis type IIIA (MPS IIIA) and ensuring the patient meets the age and weight criteria.

The patient or their legal representative must provide informed consent to participate in the study.

2 treatment initiation

The patient will begin receiving the study medication, JR-441, which is a lyophilized powder prepared for injection.

The medication is administered through an intravenous infusion once a week.

3 monitoring and assessments

Throughout the study, the patient will undergo regular monitoring to evaluate safety and effectiveness. This includes checking for any adverse events, changes in laboratory tests, and vital signs.

The patient will also have periodic assessments of cognitive function and adaptive behavior.

4 completion of treatment

The study is expected to continue until January 11, 2030. The patient will receive weekly infusions of JR-441 until the end of the study or until they choose to withdraw.

After the final administration of the study drug, follow-up assessments will be conducted to ensure the patient’s well-being.

Who Can Join the Study?

The patient must be between 1 year and 18 years old at the time of signing the consent form.
The patient or their legal representative, like parents or guardians, must sign a written consent form approved by an ethics committee. If possible, the patient should also give their agreement to participate.
The patient must have a confirmed diagnosis of Mucopolysaccharidosis type IIIA (MPS IIIA). This includes:
- Low activity of a specific enzyme called N-sulphoglucosamine sulphohydrolase (SGSH) in certain cells.
- Normal activity of at least one other enzyme to rule out other conditions.
- Presence of specific genetic changes in the SGSH gene.
The patient must weigh at least 10 kg.
If the patient is a female who can have children, or if the patient has a female partner who can have children, they must agree to use a reliable method of birth control during the study and for a certain period after the study ends.
If the patient has hearing problems and needs hearing aids, they should use them as much as possible, especially during tests that check development and learning.
The patient must be in stable health and able to follow the study requirements, including travel, without causing too much difficulty for themselves or their family.

Who Cannot Join the Study?

Where you can join this trial?

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Site Name	City	Country	Status
Uaybaafpud Mckjhsx Cyflce Hznxeziicpabtggrr	Hamburg	Germany

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	28.09.2023

Trial locations

Investigated drugs:

N-Sulfoglucosamine Sulfohydrolase Fused To A Humanised Monoclonal Antibody Targeting Human Transferrin Receptor

JR-441 is an investigational medication being studied for its potential to treat patients with mucopolysaccharidosis type IIIA (MPS IIIA). This medication is administered through weekly infusions. The main goal of the trial is to assess the safety of JR-441 and to explore its effectiveness in managing the symptoms of MPS IIIA, a rare genetic disorder that affects the body’s ability to break down certain sugars.

Investigated diseases:

Mucopolysaccharidosis III

Mucopolysaccharidosis type IIIA (MPS IIIA) – This is a rare genetic disorder that affects the body’s ability to break down certain complex carbohydrates called glycosaminoglycans. It is caused by a deficiency of the enzyme heparan N-sulfatase, leading to the accumulation of heparan sulfate in the body. This accumulation primarily affects the brain and central nervous system, resulting in progressive neurological symptoms. Children with MPS IIIA may initially develop normally but then experience a decline in cognitive and motor skills. Over time, they may also develop behavioral issues, sleep disturbances, and loss of speech. The progression of the disease varies, but it typically leads to severe neurological impairment.

Trial ID:

2024-517045-14-00

Protocol code:

JR-441-101

NCT ID:

NCT06095388

Trial Phase:

Human Pharmacology (Phase I) – First administration to humans

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Study of Weekly Infusions of JR-441 for Patients with Mucopolysaccharidosis Type IIIA

What is this study about?

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