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A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

NCT05185622 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 54

Last updated 2025-10-24

Study results available
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Summary

This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in steroid-naïve boys ages 2 to \<4 years, and glucocorticoid-treated and currently untreated boys ages 7 to \<18 years with DMD.

Conditions

Interventions

DRUG

Vamorolone

Oral administration of vamorolone for 12 weeks.

Sponsors & Collaborators

  • Santhera Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Jean K Mah, M.D. · Alberta Children's Hospital Research Institute, University of Calgary

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-21
Primary Completion
2024-07-16
Completion
2024-07-16
FDA Drug
Yes

Countries

  • Canada

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05185622 on ClinicalTrials.gov