Vamorolone for Duchenne Muscular Dystrophy

Phase-Based Progress Estimates

Effectiveness

Safety

Duchenne Muscular DystrophyVamorolone - Drug

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Eligibility

2 - 17

Male

What conditions do you have?

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Eligibility

2 - 17

Male

What conditions do you have?

Select

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

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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

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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