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Treatment for Myotonic Dystrophy (ASPIRE-DM1 Trial)
N/A
Recruiting
Led By Nicholas E. Johnson, MD
Research Sponsored by Virginia Commonwealth University
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 baseline, month 12, month 18
Awards & highlights
No Placebo-Only Group
Summary
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and childhood myotonic dystrophy type 1, and develop biomarkers for the condition.
Eligible Conditions
- Myotonic Dystrophy
- Congenital Diaphragmatic Hernia
- Congenital Hyperinsulinism
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, month 12, month 18
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, month 12, month 18
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
To evaluate motor milestone attainment in individuals with CDM and ChDM and compare to typically developing children
Secondary study objectives
AIMS
CCMDHI
Domain Delta
+3 moreOther study objectives
Blood Sampling
Correlate the functional outcome measures with potential biomarkers in CDM.
Muscle mass, DEXA
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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Who is running the clinical trial?
Virginia Commonwealth UniversityLead Sponsor
714 Previous Clinical Trials
22,888,441 Total Patients Enrolled
2 Trials studying Myotonic Dystrophy
800 Patients Enrolled for Myotonic Dystrophy
Nicholas E. Johnson, MDPrincipal InvestigatorVirginia Commonwealth University
2 Previous Clinical Trials
580 Total Patients Enrolled
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