Boys with DMD get continued access to promising steroid alternative

NCT ID NCT03863119

AVAILABLE Disease control Sponsor: Santhera Pharmaceuticals Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

Locations

Alberta's Children Hospital
AVAILABLE

Calgary, Alberta, T3B 6A8, Canada
British Columbia Children's Hospital
AVAILABLE

Vancouver, British Columbia, V6H 3N1, Canada
Children's Hospital of Eastern Ontario
AVAILABLE

Ottawa, Ontario, K1H 8L1, Canada
Duke University
AVAILABLE

Durham, North Carolina, 27710, United States
Montreal Childrens Hospital
AVAILABLE

Montreal, Quebec, H4A 3J1, Canada
Nemours Children's Hospital
AVAILABLE

Orlando, Florida, 32827, United States
Schneider Chidlren's Medical Center
AVAILABLE

Petah Tikva, 4920435, Israel
Seattle Children's
AVAILABLE

Seattle, Washington, 98105, United States
The Hospital for Sick Children
AVAILABLE

Toronto, Ontario, M5G 1X8, Canada
University of California Davis
AVAILABLE

Davis, California, 95616, United States
University of Texas Southwestern Medical Center
AVAILABLE

Dallas, Texas, 75207, United States
urie Children's Hospital of Chicago
AVAILABLE

Chicago, Illinois, 60611, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.