Active not recruitingPHASE3INTERVENTIONAL

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

This study is looking at a new treatment called JR-141 for a condition called Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. It's a 'Phase III' study, which means it's a large and important step in testing a new medicine. Researchers want to find out how safe JR-141 is and how well it works compared to a treatment that's already available, called idursulfase. Some people in the study will receive JR-141, and others will receive idursulfase. The study is running in many places around the world and involves different types of patients with MPS II, including children and adults. The main goal is to see if JR-141 could be a good new option for treating MPS II.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

JCR Pharmaceuticals Co., Ltd.

Enrolment target

Start

14 Feb 2022

Estimated completion

31 Oct 2027

Mucopolysaccharidosis II

What is this study about?

This study is called STARLIGHT and it's looking into a new treatment for Mucopolysaccharidosis II, or MPS II, which is also known as Hunter syndrome. MPS II is a rare genetic condition that can affect many parts of the body. There are treatments available, but scientists are always working to find new and potentially better options.

The main idea of this study is to compare a new medicine, JR-141, with a treatment that's already used for MPS II, called idursulfase. Researchers want to see if JR-141 is just as safe, or safer, and if it works just as well, or better, than the current treatment. This is an important step in developing new medicines, known as a 'Phase III' study.

Because this is a global study, it means many people from different countries will be taking part. By comparing these treatments, the study aims to gather important information that could help provide more treatment choices for people living with MPS II in the future.

Key takeaways

This study is testing a new medicine (JR-141) for Hunter syndrome (MPS II).
It compares JR-141 to an existing treatment (idursulfase).
It's a large, international study (Phase III).
Both children and adults with MPS II may be able to join.
Participation involves regular clinic visits and health checks.
You can choose to leave the study at any time.

Who may be eligible?

To join this study, you or your child must have a confirmed diagnosis of MPS II. You'll also need to agree to be part of the study by signing a consent form. If the person with MPS II is under 16 years old in the UK (or under 18 elsewhere) or has a learning disability related to MPS II, a parent or guardian will need to sign on their behalf. If possible, the patient will also be asked to agree.

Patients can be new to treatment, or they can already be receiving idursulfase treatment, but they must have been on a steady dose for at least 3 months (12 weeks) before starting the study. For women who could become pregnant, or men with partners who could become pregnant, it's important to agree to use effective contraception, like condoms, during the study.

The study is looking for patients of all ages, but there are different groups, or 'cohorts', based on age and how the MPS II affects them. For younger children, some tests will be done to understand their development. For older children and adults, there are specific IQ score requirements to participate.

Quick self-check

Do you (or your child) have a confirmed diagnosis of MPS II?
Are you willing and able to sign a consent form, or can your parent/guardian sign for you?
Are you new to treatment, or have you been on a stable dose of idursulfase for at least 3 months?
If you are female and able to become pregnant, or male with a partner who might, are you willing to use contraception during the study?
For younger children, can they participate in specific development tests?
For older children/adults, is your IQ above 70?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will either receive the new medicine JR-141 or the existing medicine idursulfase. The medical team will decide which treatment you receive, and for some of the study, neither you nor the doctors will know which one it is (this is called 'assessor-blinded'). You'll have regular visits to the clinic for assessments, which will likely include checking your health, taking blood samples, and performing other tests to see how you're responding to the treatment. The total length of your participation, including follow-up visits, will be explained in detail by the study team.

Potential risks and benefits

Taking part in this study could offer the benefit of receiving a new experimental treatment for MPS II or continuing an existing one, which might improve your condition. You will also be closely monitored by medical professionals, which means any health changes will be carefully watched. However, like all medicines, JR-141 could have side effects, and some tests or procedures might cause discomfort. It's important to remember that you can stop participating in the study at any time, for any reason, without it affecting your usual medical care.

Locations (25)

Phoenix Children's Hospital
Phoenix, United States
UCSF Benioff Children's Hospital Oakland
Oakland, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
University of Minnesota
Minneapolis, United States
Columbia University
New York, United States
University of North Carolina at Chapel Hill Medical School Wing E
Chapel Hill, United States
Children's Hospital of Philadelphia
Philadelphia, United States
Hospital Universitario Austral
Buenos Aires, Argentina
Hospital de Clínicas de Porto Alegre
Porto Alegre, Brazil
Instituto de Genética e Erros Inatos do Metabolismo
São Paulo, Brazil
Fundación Cardio Infantil - Instituto de Cardiología
Bogotá, Colombia
Hôpital Femme Mère Enfant
Lyon, France

+13 more sites — see the official record for the full list.

Common questions

What is Mucopolysaccharidosis II (MPS II)?

MPS II, also known as Hunter syndrome, is a rare genetic condition where the body can't break down certain complex sugars, leading to a build-up that can affect many parts of the body.

What is the new medicine JR-141?

JR-141 is an investigational medicine being tested to see if it can help treat MPS II. It's not yet available for general use.

Will I know if I'm getting the new medicine or the existing one?

For part of the study, neither you nor the doctors will know which treatment you are receiving. This helps make sure the results are fair and accurate.

What does 'Phase III study' mean?

A Phase III study is a large study that compares a new treatment to existing ones to confirm its safety and effectiveness before it can be considered for approval.

Can I stop being part of the study at any time?

Yes, you have the right to withdraw from the study at any point, and it will not affect your medical care.