Vamorolone for Duchenne Muscular Dystrophy

Recruiting at4 trial locations

Overseen by Jean K Mah, M.D.

Age: < 18

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: ReveraGen BioPharma, Inc.

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Research Team

Jean K Mah, M.D.

Principal Investigator

Alberta Children's Hospital Research Institute, University of Calgary

Eligibility Criteria

This trial is for boys aged 2 to less than 4 years, and 7 to less than 18 years with a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD). They must have no major renal or hepatic impairment, diabetes, immunosuppression, or severe behavioral issues. Participants should not be on certain medications like mineralocorticoid receptor agents or other investigational drugs recently.

Inclusion Criteria

Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not clinically significant, in the opinion of the Investigator

Subject and parent(s)/guardian(s) are willing and able to comply with scheduled visits, study drug administration plan, and study procedures

I am a boy aged 2-3 or 7-17.

Exclusion Criteria

Subject has an allergy or hypersensitivity to the study medication or to any of its constituents

Subject is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to enrollment

My child, aged 2-4, has been treated with steroids or other immune-weakening medicines.

Treatment Details

Interventions

• Vamorolone (Corticosteroid)

Trial Overview The study tests Vamorolone's safety and effectiveness over a period of 12 weeks in steroid-naïve young boys and those previously treated with glucocorticoids for DMD. It aims to assess various aspects including physical functioning and neuropsychological effects.

Participant Groups

Group I: Treatment Group 7 Experimental Treatment 1 Intervention

Patients in Treatment Group 7 must be ages 12-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 7 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study.

Group II: Treatment Group 6 Experimental Treatment 1 Intervention

Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study.

Group III: Treatment Group 5 Experimental Treatment 1 Intervention

Patients in Treatment Group 5 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 5 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study.

Group IV: Treatment Group 4 Experimental Treatment 1 Intervention

Patients in Treatment Group 4 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 4 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study.

Group V: Treatment Group 3 Experimental Treatment 1 Intervention

Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study.

Group VI: Treatment Group 2 Experimental Treatment 1 Intervention

Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1.

Group VII: Treatment Group 1 Experimental Treatment 1 Intervention

Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2.

Who Is Running the Clinical Trial?

ReveraGen BioPharma, Inc.

Lead Sponsor

Trials

9

Recruited

440+

Santhera Pharmaceuticals

Lead Sponsor

Trials

32

Recruited

2,800+