Summary:
Hunter syndrome clinical research study for pediatric patients
The AIM IT research study is evaluating an investigational therapy called idursulfase IT. Researchers are evaluating whether this investigational therapy, when given with IV-administered idursulfase, may be effective in treating symptoms associated with the brain. The AIM-IT study is being conducted in 10 centers around the world, including locations in North and South America.
What is Hunter syndrome?
Hunter syndrome is typically a severe, progressive, and life-limiting disease. Hunter syndrome is a rare genetic disorder that occurs when an enzyme the body needs is either missing or doesn’t work properly. Because the body doesn’t have enough of the enzyme to break down certain complex molecules, the molecules build up in harmful amounts in certain cells and tissues. The buildup that occurs in Hunter syndrome eventually causes permanent, progressive damage affecting appearance, mental development, organ function, and physical abilities. The multisystem symptoms and effects are extremely debilitating. Hunter syndrome appears in children as young as 18 months, and it nearly always occurs in males. There’s no cure for Hunter syndrome.
What is the purpose of the AIM-IT study?
The standard therapy for Hunter syndrome is IV-administered enzyme replacement therapy (ERT). Although patients treated with IV-administered ERT may have improvements with other symptoms of Hunter syndrome, they are not expected to have improvement in brain or cognitive function because the IV-administered ERT does not enter the brain and spinal cord due to the large size of the enzyme. There is a critical need for a therapy that can help with symptoms associated with the brain, such as the loss of skills that help with learning, understanding, and reasoning.
The AIM-IT research study is evaluating an investigational therapy called idursulfase-IT. Researchers are looking to determine whether this investigational therapy, when given with IV administered ERT, may be effective in treating symptoms associated with the brain.
Who is eligible to participate?
Boys aged 3-17 years, with a documented diagnosis of Hunter syndrome (MPS-II), who display evidence of Hunter syndrome-related cognitive impairment may be eligible to participate if they have received and tolerated at least 4 months of therapy with IV-administered ERT before study participation and have not had a stem cell or bone marrow transplant. Patients will need to meet additional eligibility criteria to participate and meeting all criteria does not guarantee participation.
Patients younger than 3 years of age may be enrolled in a separate sub-study if they meet other criteria.
What is the time commitment involved?
Patients who qualify will be in the study for 12 months (52 weeks) and will participate in a follow-up call 7 days after Week 52. Patients may also be evaluated for an extension study following their participation in the AIM-IT study.
How many patients are currently enrolled? Are there spots left in the study? How long will enrollment last and when might the study be complete?
These are all questions that are best directed to your local study site. A study doctor can discuss with you whether enrollment will be continuing or if your child might be eligible to participate or re-screen for the study. To connect with a study site in your country, please visit our website [URL] and complete our basic pre-screening questions. Study site contact information will be provided at the end of the pre-screening questions.
Is there any cost to participate in the study?
You will not have to pay for the study medication, study visits, or any tests that are part of the study. For those required to travel long distances to the study location, the AIM-IT research study will provide travel assistance at no cost to you.
Why should I consider participating?
Research studies play an important part in improving medical understanding and the development of medical treatments. Clinical trials are an especially important part of the Hunter syndrome community because of the need to develop better ways to treat the disease. What researchers learn in the AIM-IT study could help future Hunter patients and their families.
Participating also allows your child to:
- Contribute to the understanding of how the investigational drug may improve or prevent worsening symptoms in children diagnosed with Hunter syndrome
- Receive study-related medical care from a team of study physicians (in addition to your own family physician)
Like all research studies, the study procedures, as well as the investigational medication, carry a certain amount of risk. The study doctor can talk to you about side effects that people have reported after taking the investigational medication. It is important that you report any and all symptoms or health problems to the study doctor, whether or not you think these problems are related to the investigational medication. Your involvement is completely voluntary and you may choose to leave the study at any time.