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Corticosteroid
Weekly Prednisolone for Duchenne Muscular Dystrophy
Phase 4
Recruiting
Led By Anne Connolly, MD
Research Sponsored by Anne M. Connolly
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Subjects ages 1 month through 30 months
Weakness consistent with Duchenne on exam, creatine kinase ≥ 20 times the upper limit of normal, and genetic mutation known to be causative for DMD.
Must not have
Prior treatment with Glucocorticosteroids
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline visit to 24 month visit
Awards & highlights
All Individual Drugs Already Approved
Approved for 5 Other Conditions
Drug Has Already Been Approved
No Placebo-Only Group
Pivotal Trial
Summary
This trial is testing if a lower dose of steroids given once a week is as effective as a higher dose for infants and young boys with Duchenne muscular dystrophy. The goal is to see if the lower dose can reduce muscle damage while causing fewer side effects, like weight gain. The study will measure improvements in motor skills over time. Prednisolone has been shown to improve strength in Duchenne dystrophy, but high doses are associated with significant side effects.
Who is the study for?
This trial is for young children from 1 month to 30 months old who show signs of Duchenne Muscular Dystrophy (DMD) on physical exams, have high levels of an enzyme called creatine kinase in their blood, and a genetic mutation known to cause DMD. Children previously treated with glucocorticosteroids can't participate.
What is being tested?
The study is testing if a lower weekly dose of the corticosteroid Prednisolone (5mg/kg) is as effective as the standard higher dose (10mg/kg) for treating symptoms of Duchenne Muscular Dystrophy in infants.
What are the potential side effects?
Prednisolone may cause side effects such as increased appetite, weight gain, mood changes, high blood pressure, bone weakening over time, and higher risk of infections.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 1 month and 30 months old.
Select...
I have Duchenne muscular dystrophy with high creatine kinase levels and a confirmed genetic mutation.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have been treated with steroids before.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline visit to 24 month visit
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline visit to 24 month visit
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
The change from baseline to 24 months for the Gross Motor Scaled Score.
Secondary study objectives
Language (expressive and receptive), Social and Fine Motor skills at 24 months as assessed by the Bayley-4 Scales of Infant and Toddler Development
Linear growth
Awards & Highlights
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
1Treatment groups
Experimental Treatment
Group I: ExperimentalExperimental Treatment1 Intervention
This is a one-arm study and the group of subjects are all experimental and will receive drug.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Prednisolone
FDA approved
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Duchenne Muscular Dystrophy (DMD) include oral corticosteroids and genetic therapies. Oral corticosteroids, such as prednisone, work by reducing inflammation and suppressing the immune system, which helps to slow muscle degeneration and improve muscle strength.
This is crucial for DMD patients as it can prolong mobility and improve quality of life. Genetic therapies, like exon skipping drugs (eteplirsen, golodirsen, viltolarsen), aim to increase the production of dystrophin, a protein that is deficient in DMD patients.
By skipping over specific exons in the DMD gene, these therapies allow for the production of a shorter but functional dystrophin protein, which can help stabilize muscle fibers and slow disease progression. Both treatment approaches are vital as they address the underlying mechanisms of muscle deterioration in DMD.
Find a Location
Who is running the clinical trial?
Muscular Dystrophy AssociationOTHER
37 Previous Clinical Trials
60,725 Total Patients Enrolled
University of TexasOTHER
189 Previous Clinical Trials
143,113 Total Patients Enrolled
Virginia Commonwealth UniversityOTHER
714 Previous Clinical Trials
22,888,409 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am between 1 month and 30 months old.I have been treated with steroids before.I have Duchenne muscular dystrophy with high creatine kinase levels and a confirmed genetic mutation.
Research Study Groups:
This trial has the following groups:- Group 1: Experimental
Awards:
This trial has 5 awards, including:- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- Approved for 5 Other Conditions - This treatment demonstrated efficacy for 5 other conditions.
- Drug Has Already Been Approved - The FDA has already approved this drug, and is just seeking more data.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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