← Back to Search

Corticosteroid

Vamorolone for Duchenne Muscular Dystrophy

Q: Does this research endeavor have an age limit of 80 years or lower?

Participants for this research must be aged 2 or above, but no older than 17 years old.

Q: Does this experiment have any available slots for participation?

As per the data documented on clinicaltrials.gov, this medical study is currently looking for volunteers to participate in their research project. The trial was initiated on March 21st 2022 and most recently amended on September 29th 2022.

Q: Are there any eligibility requirements to participate in this research?

The requirements for participating in this study are that applicants must have Becker <a href="https://www.withpower.com/clinical-trials/muscular-dystrophy">Muscular dystrophy</a> and be 2 - 17 years old. This clinical trial is aiming to enrol a total of 44 patients.

Q: In how many locations is the management team overseeing this clinical trial?

This medical trial is presently recruiting from 4 sites, which are situated in Toronto, Calgary and Vancouver amongst other areas. It's advisable to pick the closest centre when enrolling so as to reduce travel demands.

Q: What is the primary objective of this scientific investigation?

Santhera Pharmaceuticals, the study sponsor, has specified that their primary outcome will be changes to heart rate observed over a Day 1-Week 16 timeline. Secondary objectives include assessing AUCinf following oral administration and variations in fasting glucose/insulin concentrations from baseline values at Week 12.

Q: Has the U.S. Food and Drug Administration sanctioned Vamorolone?

Based on the limited data available, our team assigned Vamorolone a safety rating of 2. This is because while there is some evidence indicating its security profile, there are currently no trials that have tested for efficacy.

Q: How many participants has this research accepted thus far?

This clinical trial necessitates the recruitment of 44 individuals that meet certain inclusion criteria. Patients in Toronto, Alberta and Calgary, British Columbia can join this study at The Hospital for Sick Children and Alberta's Children Hospital respectively.

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

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1, week 2, week 6, week 12, week 16

Awards & highlights

Study Summary

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

Who is the study for?

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.Check my eligibility

What is being tested?

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.See study design

What are the potential side effects?

While the specific side effects are not listed here, potential risks may include reactions similar to those seen with steroids since Vamorolone is being tested as an alternative treatment for DMD.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1, week 2, week 6, week 12, week 16

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1, week 2, week 6, week 12, week 16

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1, week 2, week 6, week 12, week 16 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in Body Mass Index (BMI) from baseline to Week 12

Change in Body Mass Index (BMI) z-score from baseline to Week 12

Change in Height from baseline to Week 12

+13 more

Secondary outcome measures

Area under the Curve infinity (AUCinf) following oral administration

Change in fasting serum concentration of glucose from baseline to Week 12

Change in fasting serum concentration of insulin from baseline to Week 12

+2 more

Other outcome measures

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 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

+6 more

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

Nasopharyngitis

Abnormal behaviour

Vitamin D Deficiency

Rash

Rhinitis

Contusion

Fall

Pain in extremity

Psychomotor hyperactivity

Aggression

Impetigo

Arthropod bite

Ear Infection

Pharyngitis streptococcal

Athralgia

Protein Urine Present

Poor quality sleep

Muscle atrophy

Abdominal Pain upper

Weight increased

Increased appetite

Hypertrichosis

Toothache

Enterobiasis

Influenza

Back pain

Rhinorrhoea

Oropharyngeal pain

Dry skin

Erythema

Cortisol decreased

Blood pressure increased

Fatigue

Proteinuria

Blood uric acid increased

Tonsilitis

Pneumonia

Ligament strain

Muscle strain

100%

80%

60%

40%

20%

Study treatment Arm

Treatment Group 1

Treatment Group 2

Treatment Group 3

Treatment Group 4

Treatment Group 5

Treatment Group 6

Trial Design

7Treatment groups

Experimental Treatment

Group I: Treatment Group 7Experimental Treatment1 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 6Experimental Treatment1 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 5Experimental Treatment1 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 4Experimental Treatment1 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 3Experimental Treatment1 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 2Experimental Treatment1 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 1Experimental Treatment1 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vamorolone

2018

Completed Phase 2

~130

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

ReveraGen BioPharma, Inc.Lead Sponsor

8 Previous Clinical Trials

388 Total Patients Enrolled

Santhera PharmaceuticalsLead Sponsor

28 Previous Clinical Trials

2,634 Total Patients Enrolled

Jean K Mah, M.D.Study ChairAlberta Children's Hospital Research Institute, University of Calgary

Media Library

Vamorolone (Corticosteroid) Clinical Trial Eligibility Overview. Trial Name: NCT05185622 — Phase 2

Duchenne Muscular Dystrophy Research Study Groups: Treatment Group 6, Treatment Group 4, Treatment Group 5, Treatment Group 3, Treatment Group 7, Treatment Group 1, Treatment Group 2

Duchenne Muscular Dystrophy Clinical Trial 2023: Vamorolone Highlights & Side Effects. Trial Name: NCT05185622 — Phase 2

Vamorolone (Corticosteroid) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05185622 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research endeavor have an age limit of 80 years or lower?

"Participants for this research must be aged 2 or above, but no older than 17 years old."

Answered by AI

Does this experiment have any available slots for participation?

"As per the data documented on clinicaltrials.gov, this medical study is currently looking for volunteers to participate in their research project. The trial was initiated on March 21st 2022 and most recently amended on September 29th 2022."

Answered by AI

Are there any eligibility requirements to participate in this research?

"The requirements for participating in this study are that applicants must have Becker Muscular dystrophy and be 2 - 17 years old. This clinical trial is aiming to enrol a total of 44 patients."

Answered by AI

In how many locations is the management team overseeing this clinical trial?

"This medical trial is presently recruiting from 4 sites, which are situated in Toronto, Calgary and Vancouver amongst other areas. It's advisable to pick the closest centre when enrolling so as to reduce travel demands."

Answered by AI

What is the primary objective of this scientific investigation?

"Santhera Pharmaceuticals, the study sponsor, has specified that their primary outcome will be changes to heart rate observed over a Day 1-Week 16 timeline. Secondary objectives include assessing AUCinf following oral administration and variations in fasting glucose/insulin concentrations from baseline values at Week 12."

Answered by AI

Has the U.S. Food and Drug Administration sanctioned Vamorolone?

"Based on the limited data available, our team assigned Vamorolone a safety rating of 2. This is because while there is some evidence indicating its security profile, there are currently no trials that have tested for efficacy."

Answered by AI

How many participants has this research accepted thus far?

"This clinical trial necessitates the recruitment of 44 individuals that meet certain inclusion criteria. Patients in Toronto, Alberta and Calgary, British Columbia can join this study at The Hospital for Sick Children and Alberta's Children Hospital respectively."

Answered by AI