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Vamorolone for Becker Muscular Dystrophy

Phase 2
Recruiting
Research Sponsored by ReveraGen BioPharma, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subject has an NSAA score of 32 or less at screening
Subject is between 18 and 65 years of age at the time of informed consent
Must not have
Subject has evidence of symptomatic cardiomyopathy
Subject has current or history of chronic systemic fungal or viral infections
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1, week 4, week 12, week 24, week 28

Summary

This trial is to study a new drug, vamorolone, to treat boys with Duchenne muscular dystrophy. The trial will last 24 weeks and compare vamorolone to a placebo. The study is funded by the FDA.

Who is the study for?
This trial is for males with Becker Muscular Dystrophy who can walk 10 meters in ≤ 30 seconds, even with a cane or walker. They should have an NSAA score ≤ 32 and not be on oral steroids or immunosuppressants for the past 3 months. Participants must agree to use barrier contraception during the study and be between 18-65 years old.
What is being tested?
The study tests Vamorolone, a potential treatment for BMD, against a placebo over six months. It's double-blind, meaning neither researchers nor participants know who gets the real drug versus placebo. The goal is to assess safety, how well it works (efficacy), and its effects on the body (pharmacodynamics).
What are the potential side effects?
While specific side effects of Vamorolone are not listed here, similar drugs often cause issues like weight gain, bone thinning, mood changes, high blood sugar levels and increased risk of infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My NSAA score is 32 or lower.
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I am between 18 and 65 years old.
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I am a male diagnosed with Becker dystrophy due to a specific mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have heart muscle disease with symptoms.
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I have or had a long-term fungal or viral infection.
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I have had serious kidney, liver issues, uncontrolled diabetes, or a weak immune system.
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I have severe mental or behavioral issues that prevent me from joining the study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1, week 4, week 12, week 24, week 28
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1, week 4, week 12, week 24, week 28 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Safety as measured by 12-lead ECG
Safety as measured by Body Temperature
Safety as measured by Body Weight
+8 more
Secondary study objectives
Efficacy as measured by concentration of serum pharmacodynamic biomarkers
Pharmacokinetics as measured by AUCinf
Safety as measured by concentration of Salivary Cortisol
+4 more
Other study objectives
Efficacy as measured by North Star Ambulatory Assessment (NSAA) score
Efficacy as measured by Time to Run/Walk Test (TTRW)
Tolerability as measured by NeuroQOL score

Side effects data

From 2021 Phase 2 trial • 121 Patients • NCT03439670
36%
Upper respiratory tract infection
25%
Pyrexia
21%
Vomiting
18%
Cough
14%
Cushingoid
11%
Abdominal Pain
11%
Constipation
11%
Diarrhoea
11%
Headache
11%
Gastroenteritis viral/viral gasteroenteritis
7%
Nasopharyngitis
7%
Abnormal behaviour
7%
Vitamin D Deficiency
7%
Rash
7%
Rhinitis
7%
Contusion
7%
Fall
7%
Pain in extremity
7%
Psychomotor hyperactivity
7%
Aggression
7%
Impetigo
4%
Pharyngitis streptococcal
4%
Arthropod bite
4%
Ear Infection
4%
Athralgia
4%
Protein Urine Present
4%
Poor quality sleep
4%
Muscle atrophy
4%
Abdominal Pain upper
4%
Weight increased
4%
Increased appetite
4%
Hypertrichosis
4%
Toothache
4%
Enterobiasis
4%
Influenza
4%
Back pain
4%
Rhinorrhoea
4%
Oropharyngeal pain
4%
Dry skin
4%
Erythema
4%
Cortisol decreased
4%
Blood pressure increased
4%
Fatigue
4%
Proteinuria
4%
Blood uric acid increased
4%
Tonsilitis
4%
Pneumonia
4%
Ligament strain
4%
Muscle strain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Treatment Group 1
Treatment Group 2
Treatment Group 3
Treatment Group 4
Treatment Group 5
Treatment Group 6

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Vamorolone 500mg/day [250mg if <50kg body weight]Experimental Treatment1 Intervention
Subjects will be randomized to one of two treatment groups in a 1:2 ratio (placebo:vamorolone).
Group II: PlaceboPlacebo Group1 Intervention
Subjects will be randomized to one of two treatment groups in a 1:2 ratio (placebo:vamorolone).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vamorolone
2018
Completed Phase 2
~230

Find a Location

Who is running the clinical trial?

ReveraGen BioPharma, Inc.Lead Sponsor
8 Previous Clinical Trials
403 Total Patients Enrolled
Santhera PharmaceuticalsIndustry Sponsor
31 Previous Clinical Trials
2,777 Total Patients Enrolled
Paula Clemens, M.D.Study ChairUniversity of Pittsburgh
1 Previous Clinical Trials
121 Total Patients Enrolled

Media Library

Placebo Clinical Trial Eligibility Overview. Trial Name: NCT05166109 — Phase 2
Becker Muscular Dystrophy Research Study Groups: Vamorolone 500mg/day [250mg if <50kg body weight], Placebo
Becker Muscular Dystrophy Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT05166109 — Phase 2
Placebo 2023 Treatment Timeline for Medical Study. Trial Name: NCT05166109 — Phase 2
~7 spots leftby Jun 2025
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