AvailableEXPANDED_ACCESS

Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

This program, called an Expanded Access Protocol, was set up to help boys with Duchenne muscular dystrophy in the United States and Canada continue to get a medicine named vamorolone. It's for those who have already taken part in previous vamorolone studies and are familiar with the treatment. The idea is to make sure they can keep receiving the medicine while the company prepares and waits for it to be officially approved for wider use. Doctors and families work together to decide if continued treatment is best for the child. While the US program has closed because vamorolone is now approved there, the Canadian program is still running for existing participants but isn't accepting new ones.

At a glance

Status

Available

Sponsor

Santhera Pharmaceuticals

Duchenne Muscular Dystrophy

What is this study about?

This information is about a special program for boys who have Duchenne muscular dystrophy and have already taken part in one of the previous studies for a medicine called vamorolone. These studies, known as VBP15-LTE, VBP15-004, or VBP15-006, explored how well vamorolone works. Since they can't join another research study, this 'Expanded Access Protocol' allows them to keep receiving vamorolone. This is especially helpful while the medicine is going through the final steps to get approved for wider use in places like Canada.

The main goal is to make sure that boys who have already benefited from vamorolone in earlier studies don't have to stop taking it during the approval process. The medicine is sent directly to the family, and the child also receives their usual care for Duchenne muscular dystrophy. Doctors involved in this program keep a close eye on any side effects or changes, which they report to the company that makes the drug.

Doctors can adjust the dose of vamorolone within a certain range (2 mg, 4 mg, or 6 mg per kilogram of body weight per day), given once daily. It's important to take the medicine with a fatty food like a glass of full-fat milk, just like in the earlier studies. If the doctors and family agree it's helping the child and there are no big safety concerns, the child can continue taking vamorolone as long as the program is active, or until the medicine officially gets approved.

Key takeaways

This program allows boys already using vamorolone in Canada to continue treatment.
It's for existing participants only; no new patients are being recruited.
The goal is continued access to the medicine while it seeks wider approval.
Doctors will monitor your child's health and can adjust the medicine's dose.
Vamorolone must be taken once daily with a high-fat food, like full-fat milk.
Participation can continue indefinitely if beneficial and safe for your child.

Who may be eligible?

This program is for specific boys with Duchenne muscular dystrophy who have already participated in certain earlier studies. To be considered, a parent or legal guardian must give their written permission for their child to take part.

The child must have completed one of three specific previous studies for vamorolone (VBP15-LTE, VBP15-004, or VBP15-006) up to a certain point in those studies. Both the child and their parent or guardian must also be willing and able to follow the instructions for taking the medicine and attend all necessary follow-up appointments with their doctor.

Boys cannot join if they experienced a serious side effect in a previous vamorolone study that their doctor and the drug company believe makes it unsafe for them to continue taking the medication. Also, if the child or parent is unable or unwilling to keep up with regular medical care and appointments, they cannot participate.

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.

My child is a boy diagnosed with Duchenne muscular dystrophy.
My child has already completed one of the following vamorolone studies: VBP15-LTE (up to 24 months), VBP15-004 (up to 48 weeks), or VBP15-006 (up to 12 weeks).
I, as the parent or guardian, am willing to provide written permission.
My child and I can commit to following the medication schedule and attending all necessary doctor's appointments.
My child did not have a serious side effect in a previous study that would make continued use of vamorolone unsafe.

Answer every question to see your result.

What does participation involve?

If your child is in this program, vamorolone will be sent directly to your family by the study site. Your child will continue to receive their usual care for Duchenne muscular dystrophy, just as they would normally. You will give your child vamorolone once a day, making sure to mix it with fatty food like a glass of full-fat milk. The dose might be adjusted by the doctor between 2 mg, 4 mg, or 6 mg per kilogram of body weight per day.

Your child's doctor will carefully record any health issues or side effects reported by you, your child, or themselves. These reports are important for safety monitoring. As long as your child's doctor and your family agree that vamorolone is helping and is safe, your child can continue taking it indefinitely, or until the medicine gets wider approval. There is no set end date for participation, as long as the program is active and beneficial for your child.

Potential risks and benefits

The potential benefit of participating is that your child, having already been in a vamorolone study, can continue to receive the medicine without interruption while it awaits full approval. This ensures ongoing access to a treatment that may have been beneficial. Potential risks include the possibility of experiencing side effects from vamorolone, as with any medication, even if your child tolerated it well in previous studies. Your doctor will monitor your child closely for any issues. You and your child have the right to withdraw from this program at any time if you decide it is no longer in your child's best interest.

Locations (12)

University of California Davis
Verified postcode
Davis, United States· Available
Nemours Children's Hospital
Verified postcode
Orlando, United States· Available
urie Children's Hospital of Chicago
Verified postcode
Chicago, United States· Available
Duke University
Verified postcode
Durham, United States· Available
University of Texas Southwestern Medical Center
Verified postcode
Dallas, United States· Available
Seattle Children's
Verified postcode
Seattle, United States· Available
Alberta's Children Hospital
Verified postcode
Calgary, Canada· Available
British Columbia Children's Hospital
Verified postcode
Vancouver, Canada· Available
Children's Hospital of Eastern Ontario
Verified postcode
Ottawa, Canada· Available
The Hospital for Sick Children
Verified postcode
Toronto, Canada· Available
Montreal Childrens Hospital
Verified postcode
Montreal, Canada· Available
Schneider Chidlren's Medical Center
Verified postcode
Petah Tikva, Israel· Available

Common questions

What is vamorolone?

Vamorolone is a medicine being studied for Duchenne muscular dystrophy. This program allows boys who have already used it in previous studies to keep taking it.

Is this program looking for new participants?

No, the program in Canada is currently active for boys who are already participating but is not looking for new people to join.

What if my child experiences side effects?

Your doctor will closely monitor your child for any side effects and report them to the company. Your child's dose may be adjusted if needed.

How long can my child stay in this program?

If it's safe and helpful, your child can continue taking vamorolone as long as the program is active, or until the medicine is officially approved.

Do I have to pay for the medicine?

The information provided suggests the medicine will be shipped to the family by the study site, which usually implies it's provided as part of the program.

How to find out more

Medical director

ekaterina.gresko@santhera.com +41 61 906 89 51 Official trial record

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