AvailableEXPANDED_ACCESS

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

This programme provides ongoing access to a medicine called Idursulfase-IT for children with Hunter syndrome. It's specifically for those who have already participated in two particular past research studies (HGT-HIT-046 or SHP609-302). The main goal is to allow these children to keep receiving the treatment if it's still proving helpful for them, and if their doctors believe the benefits are greater than any possible risks. This isn't a new study testing the drug, but rather a way to continue care for those who found it useful during earlier research. It ensures that valuable treatment can carry on once the original studies have finished.

At a glance

Status

Available

Sponsor

Takeda

Hunter Syndrome

What is this study about?

This programme is for children who have Hunter syndrome, a rare genetic condition. It offers continued access to a medicine called Idursulfase-IT. This particular drug is used to help manage Hunter syndrome. It's important to understand that this isn't a new research study to see if the medicine works; those studies have already happened.

Instead, this programme is a way for children who took part in those earlier studies (specifically HGT-HIT-046 or SHP609-302) to keep getting this medicine. The doctors and researchers involved want to make sure that children who were benefiting from Idursulfase-IT during the studies can continue to receive it, as long as it's still considered helpful and safe for them.

The main reason for this programme is to provide ongoing support and treatment to children who have found this medicine beneficial. It helps bridge the gap after a formal study ends, ensuring that effective care can carry on for those who need it most. It's about maintaining treatment for children who have already shown a positive response.

Key takeaways

This programme offers continued access to an existing Hunter syndrome medicine.
It's only for boys who took part in specific past research studies.
The goal is to extend treatment for those who have already benefited.
Decisions are based on whether benefits outweigh any potential risks.
It's about ongoing care, not testing a new drug.
Parental consent and child agreement (where appropriate) are needed.

Who may be eligible?

This programme is for children who have already taken part in one of two specific past studies: HGT-HIT-046 or SHP609-302. To join, they must have completed the treatment part of one of these studies before starting on this new programme.

Also, a parent or legal guardian will need to understand what this programme involves and agree in writing for their child to take part. If the child is old enough, they will also be asked to agree. Because this condition only affects boys, only boys can participate.

However, some children might not be able to join. If a doctor believes that taking part might not be safe for the child due to another health problem, they won't be able to participate. Also, if a child is known to have a severe allergic reaction to Idursulfase-IT or any of its ingredients, they will not be eligible.

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.

Is your child a boy diagnosed with Hunter syndrome?
Did your child complete the treatment part of the HGT-HIT-046 or SHP609-302 study?
Has your child's doctor confirmed that the benefits of the treatment still outweigh the risks?
Does your child not have any serious allergic reactions to Idursulfase-IT?
Are you, as a parent/guardian, willing to give informed consent for your child to continue treatment?

Answer every question to see your result.

What does participation involve?

This is a programme specifically for male children who have already completed treatment in one of the previous studies (HGT-HIT-046 or SHP609-302). If eligible, the child would continue to receive the Idursulfase-IT medicine. The exact details of how often the medicine is given, what examinations are needed, and how long they will stay on the programme, would be discussed with you by the medical team. This programme aims to provide continued access for as long as the treatment is beneficial and safe for the child.

Potential risks and benefits

The potential benefit of joining this programme is that your child could continue to receive a medicine that has already been shown to be helpful for their Hunter syndrome during previous studies. This means they could keep experiencing the positive effects of the treatment. As with any medicine, there are potential risks, such as side effects or allergic reactions, which would have been discussed during the earlier studies. Your doctor will carefully weigh whether the good effects of the medicine are still much greater than any possible problems. You, as a parent or guardian, always have the right to withdraw your child from the programme at any time.

Locations (22)

Phoenix Childrens Hospital
Verified postcode
Phoenix, United States· Available
The Regents of the University of California
Verified postcode
Oakland, United States· Available
The Nemours Foundation
Verified postcode
Wilmington, United States· Available
Jackson Memorial Hospital University of Miami
Verified postcode
Miami, United States· Available
Ann and Robert H Lurie Childrens Hospital of Chicago
Verified postcode
Chicago, United States· Available
Washington University
Verified postcode
Washington, United States· Available
Board of Regents of the University of Nebraska
Verified postcode
Omaha, United States· Available
Joseph M. Sanzari Children's Hospital
Verified postcode
Hackensack, United States· Available
NYU Langone Medical Center
Verified postcode
New York, United States· Available
The University of North Carolina
Verified postcode
Chapel Hill, United States· Available
The Cleveland Clinic Foundation
Verified postcode
Cleveland, United States· Available
Randall Children's Hospital at Legacy Emanuel
Verified postcode
Portland, United States· Available

Common questions

What is Hunter syndrome?

Hunter syndrome is a rare genetic condition that mainly affects boys, causing various health problems over time.

Is this a new research study?

No, this is not a new study. It's a programme to provide ongoing treatment for children who were already in previous studies and benefited from the medicine.

Who can take part in this programme?

Only boys with Hunter syndrome who have already completed one of two specific earlier studies (HGT-HIT-046 or SHP609-302) are eligible.

What is Idursulfase-IT?

Idursulfase-IT is a medicine used to treat Hunter syndrome, and it was given to participants in the earlier studies.

Can my child stop the treatment at any time?

Yes, you or your child (if old enough) can decide to stop participating in the programme at any time.

How to find out more

Takeda Contact

medinfous@takeda.com +1877-825-3327 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.