A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy
This study is investigating a potential new treatment named rhASA for children diagnosed with Late Infantile Metachromatic Leukodystrophy (MLD). MLD is a severe genetic condition that affects the brain and nervous system, leading to difficulties with movement and other essential functions. The main goal of this particular study is to see if rhASA can help children maintain their ability to move and prevent the disease from getting worse. Researchers will carefully watch to see how long it takes for a child's movement abilities to decline significantly or for other serious issues to arise, comparing treated children with what is known about how the disease usually progresses. They will also look at other factors like brain and nerve chemical levels and language development.
At a glance
What is this study about?
This study is an important research project looking into a new treatment called rhASA for a serious genetic condition known as Late Infantile Metachromatic Leukodystrophy, often shortened to MLD. MLD is an illness that affects the brain and nervous system, leading to problems with movement, speech, and mental abilities. 'Late Infantile' means it typically appears in very young children, usually before they start school. Currently, there aren't many effective treatments for MLD, so finding new options like rhASA is really important for families.
The main purpose of this study is to find out if rhASA can help children with Late Infantile MLD keep their ability to move and slow down how fast the disease progresses. When children are diagnosed with MLD, their movement abilities often get worse over time. The researchers will be carefully observing children in the study for over two years (about 106 weeks) to see how their movement skills change. They will be checking if the treatment helps them maintain their abilities better than would be expected without it.
Beyond movement, the study will also look at other important aspects of the disease. This includes checking specific chemicals in the fluid around the brain and spine, which can give clues about how the disease is affecting the body. They will also assess changes in how children speak and understand language. All these measurements help researchers understand if rhASA is making a positive difference for children with MLD.
Key takeaways
- This study is testing a new treatment called rhASA for Late Infantile Metachromatic Leukodystrophy (MLD).
- The main aim is to see if rhASA can help children maintain their movement abilities and slow disease progression.
- The treatment is given directly into the spinal fluid.
- Participants will be closely monitored for over two years, with regular hospital visits and assessments.
- This is an exploratory study (Phase II), meaning it's still in the earlier stages of testing.
- Participation involves potential benefits but also possible risks, which will be fully explained.
Who may be eligible?
This study is open to both boys and girls of all ages who have been diagnosed with Late Infantile Metachromatic Leukodystrophy (MLD). The research team will carefully review each potential participant's medical history to make sure the study is a safe and suitable option for them.
Being eligible to take part means that the researchers believe the study drug could potentially help you, and that taking part would be safe given your overall health. It also means your involvement will provide the best possible information to help other children with MLD in the future.
If you are interested, the study doctor will discuss all the requirements with you and your family to determine if this study is the right fit.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Have you or your child been diagnosed with Late Infantile MLD?
- Are you able to travel to a study site for regular appointments?
- Are you comfortable with receiving an injection into the spinal fluid?
- Are you able to commit to assessments and follow-up for over two years?
- Are you aged between any to any years old?
- Are you male or female?
What does participation involve?
If you decide to participate in this study, you will receive the study treatment, rhASA, which is given into the fluid surrounding your spinal cord (an 'intrathecal' injection). This will involve regular visits to a dedicated study clinic or hospital. During these visits, the medical team will carry out various assessments to monitor your health and how you are responding to the treatment.
These assessments will include checks of your physical movement and abilities, such as how you walk, stand, and use your muscles. Your language development will also be assessed. There will also be blood tests and possibly other tests to check specific chemicals related to MLD. The study will last for at least 106 weeks, which is just over two years, with ongoing follow-up to check your progress. All these steps are important to understand if the new treatment is helping.
Potential risks and benefits
Locations (6)
- —UnverifiedGermany
- —UnverifiedFrance
- —UnverifiedBelgium
- —UnverifiedGreece
- —UnverifiedSpain
- —UnverifiedItaly
Common questions
What is Late Infantile MLD?
Late Infantile MLD is a serious inherited condition that affects the brain and nervous system, often causing problems with movement and speech in young children.
What is rhASA?
rhASA is the name of the new treatment being studied. It is designed to help address the underlying cause of MLD.
How is rhASA given?
The treatment is given as an injection directly into the fluid that surrounds the spinal cord, called an intrathecal injection.
How long will the study last?
Participants will be part of the study for at least 106 weeks, which is a little over two years, including follow-up appointments.
Can I stop participating if I want to?
Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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