Boys with DMD get continued access to promising steroid alternative
NCT ID NCT03863119
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This program offers ongoing access to vamorolone, a steroid-like drug, for boys with Duchenne muscular dystrophy who finished earlier studies. It aims to keep them on treatment while the drug is being reviewed for approval. Participants take vamorolone daily by mouth at doses chosen by their doctor.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Vamorolone
What this could lead to
If successful, this expanded access program provides continuous treatment for boys with Duchenne muscular dystrophy while the drug is being reviewed for approval.
What could go wrong
This is an expanded access program, not a controlled trial, so results are observational. Vamorolone may not be approved, and long-term risks are still being studied.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for DUCHENNE MUSCULAR DYSTROPHY are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Alberta's Children Hospital
AVAILABLECalgary, Alberta, T3B 6A8, Canada
Contact
Contact Phone: •••-•••-••••
-
British Columbia Children's Hospital
AVAILABLEVancouver, British Columbia, V6H 3N1, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Email: •••••@•••••
-
Children's Hospital of Eastern Ontario
AVAILABLEOttawa, Ontario, K1H 8L1, Canada
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Duke University
AVAILABLEDurham, North Carolina, 27710, United States
Contact
-
Montreal Childrens Hospital
AVAILABLEMontreal, Quebec, H4A 3J1, Canada
Contact Email: •••••@•••••
Contact
-
Nemours Children's Hospital
AVAILABLEOrlando, Florida, 32827, United States
Contact
-
Schneider Chidlren's Medical Center
AVAILABLEPetah Tikva, 4920435, Israel
Contact
-
Seattle Children's
AVAILABLESeattle, Washington, 98105, United States
Contact
-
The Hospital for Sick Children
AVAILABLEToronto, Ontario, M5G 1X8, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
University of California Davis
AVAILABLEDavis, California, 95616, United States
Contact
-
University of Texas Southwestern Medical Center
AVAILABLEDallas, Texas, 75207, United States
Contact
-
urie Children's Hospital of Chicago
AVAILABLEChicago, Illinois, 60611, United States
Contact