Vamorolone for Duchenne Muscular Dystrophy

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Duchenne Muscular DystrophyVamorolone - Drug
Eligibility
2 - 17
Male
What conditions do you have?
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Study Summary

This trial tests a new drug for safety, tolerability, and efficacy in treating Duchenne Muscular Dystrophy in young boys.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

16 Primary · 5 Secondary · Reporting Duration: Day 1, Week 2, Week 6, Week 12, Week 16

12 weeks
Change in Body Mass Index (BMI) from baseline to Week 12
Change in Body Mass Index (BMI) z-score from baseline to Week 12
Number of Participants with Adverse Events as Assessed by Common Terminology Criteria for Adverse Events version 4.03 (CTCAE v4.03)
Number of participants with Cataracts
Number of participants with Glaucoma
Day 1, Week 2
Area under the Curve infinity (AUCinf) following oral administration
Week 16
Change in body temperature from baseline to each of the scheduled on-treatment and post-treatment assessment time points
Change in heart rate from baseline to each of the scheduled on-treatment and post-treatment assessment time points
Change in respiratory rate from baseline to each of the scheduled on-treatment and post-treatment assessment time points
Change in sitting blood pressure from baseline to each of the scheduled on-treatment and post-treatment assessment time points
Week 16
Number of participants with abnormal blood laboratory test results
Number of participants with abnormal urine laboratory test results
Week 12
Change in Bayley Scales of Infant and Toddler Development-III (Bayley-III) Gross Motor scale (ages 2 to <4 years only) from baseline to Week 12
Change in Height from baseline to Week 12
Change in Height z score from baseline to Week 12
Change in Pediatric Outcome Data Collection Instrument (PODCI) from baseline to Week 12
Change in Performance of Upper Limb (PUL) test (7 to <18 years only) from baseline to Week 12
Change in Personal Adjustment and Role Skills Scale, ed. 3 (PARS III) questionnaire from baseline to Week 12
Change in concentration of serum aminoterminal propeptide of type I collagen (P1NP) from baseline to Week 12
Change in concentration of serum osteocalcin from baseline to Week 12
Change in fasting serum concentration of glucose from baseline to Week 12
Change in fasting serum concentration of insulin from baseline to Week 12
Change in morning cortisol concentration from baseline to Week 12
Change in serum concentration of C terminal peptide fragment of collagen 1 (CTX) from baseline to Week 12
Change in serum concentration of hemoglobin A1c (HbA1c) from baseline to Week 12
Number of participants with abnormal ECGs
Week 16
Change in Weight from baseline to each of the scheduled on treatment and post-treatment assessment time points
Week 6, Week 12
Ease of Study Medication Administration Assessment (ages 2 to <4 years only) at each of the scheduled study assessment time points
Study Medication Acceptability Assessment (ages 7 to <18 years only) at each of the scheduled study assessment time points
Week 16
Number of participants with Cushingoid features

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Side Effects for

Treatment Group 4
27%Cushingoid
27%Pain in extremity
27%Cough
20%Protein Urine Present
20%Upper respiratory tract infection
20%Fall
13%Fatigue
13%Vitamin D Deficiency
13%Abdominal Pain upper
13%Constipation
13%Nasopharyngitis
13%Blood uric acid increased
13%Aggression
13%Rash
13%Psychomotor hyperactivity
13%Nasal congestion
7%Muscle strain
7%Initial insomnia
7%Hypertriglyceridaemia
7%Asthenia
7%Thirst
7%Emotional disorder
7%Back injury
7%Oppositional defiant disorder
7%Anger
7%Electrocardiogram abnormal
7%Viral rash
7%Overweight
7%Abdominal Pain
7%Weight increased
7%Diarrhoea
7%Pyrexia
7%COVID-19
7%Respiratory track infection viral
7%Increased appetite
7%Hypertrichosis
7%Ear Infection
7%Flatulance
7%Viral upper respiratory tract infection
7%Vertebral wedging
7%Musculoskeletal stiffness
7%Agitation
7%Mood swings
7%Epistaxis
7%Rhinorrhoea
7%Oropharyngeal pain
7%Perioral dermatitis
7%Bacterial test postive
7%Polydipsia
7%Syncope
7%Tympanic membrane perforation
7%Trichotillomania
7%Headache
7%Back pain
7%Dry skin
7%Night sweats
7%Glycosylated haemoglobin increased
7%Lipase decreased
7%Decreased appetite
7%Chromaturia
7%Impaired healing
7%Poor quality sleep
7%Hot flush
7%Peripheral coldness
7%Ear pain
7%Leukopenia
This histogram enumerates side effects from a completed 2021 Phase 2 trial (NCT03439670) in the Treatment Group 4 ARM group. Side effects include: Cushingoid with 27%, Pain in extremity with 27%, Cough with 27%, Protein Urine Present with 20%, Upper respiratory tract infection with 20%.

Trial Design

6 Treatment Groups

Treatment Group 1
1 of 6
Treatment Group 4
1 of 6
Treatment Group 3
1 of 6
Treatment Group 2
1 of 6
Treatment Group 6
1 of 6
Treatment Group 5
1 of 6

Experimental Treatment

44 Total Participants · 6 Treatment Groups

Primary Treatment: Vamorolone · No Placebo Group · Phase 2

Treatment Group 1
Drug
Experimental Group · 1 Intervention: Vamorolone · Intervention Types: Drug
Treatment Group 4
Drug
Experimental Group · 1 Intervention: Vamorolone · Intervention Types: Drug
Treatment Group 3
Drug
Experimental Group · 1 Intervention: Vamorolone · Intervention Types: Drug
Treatment Group 2
Drug
Experimental Group · 1 Intervention: Vamorolone · Intervention Types: Drug
Treatment Group 6
Drug
Experimental Group · 1 Intervention: Vamorolone · Intervention Types: Drug
Treatment Group 5
Drug
Experimental Group · 1 Intervention: Vamorolone · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vamorolone
2018
Completed Phase 2
~130

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: day 1, week 2, week 6, week 12, week 16

Who is running the clinical trial?

ReveraGen BioPharma, Inc.Lead Sponsor
8 Previous Clinical Trials
388 Total Patients Enrolled
Santhera PharmaceuticalsIndustry Sponsor
27 Previous Clinical Trials
2,634 Total Patients Enrolled
Jean K Mah, M.D.Study ChairAlberta Children's Hospital Research Institute, University of Calgary

Eligibility Criteria

Age 2 - 17 · Male Participants · 8 Total Inclusion Criteria

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Who else is applying?

What state do they live in?
Puerto Rico100.0%
How old are they?
< 18100.0%
What site did they apply to?
British Columbia Children's Hospital100.0%
What portion of applicants met pre-screening criteria?
Did not meet criteria50.0%
Met criteria50.0%
References