Study of Weekly Infusions of JR-441 for Patients with Mucopolysaccharidosis Type IIIA
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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 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.
The research process
The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
9 criteria
- 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.
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