Once Weekly Infant Corticosteroid Trial for DMD
Primary Purpose
Duchenne Muscular Dystrophy
Status
Recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Prednisolone
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne Muscular Dystrophy, DMD, Steroid, Muscular Dystrophy
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- Prior treatment with Glucocorticosteroids
Sites / Locations
- Lurie Children's Hospital of ChicagoRecruiting
- University of CincinnatiRecruiting
- Nationwide Children's HospitalRecruiting
- University of Texas Southwestern
- Virginia Commonwealth University
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Experimental
Arm Description
This is a one-arm study and the group of subjects are all experimental and will receive drug.
Outcomes
Primary Outcome Measures
The change from baseline to 24 months for the Gross Motor Scaled Score.
Neuromuscular Gross Motor Outcome (GRO): The Neuromuscular GRO is a gross motor outcome measure developed to assess whole body strength, motor development, and function for all levels of ability across the lifespan in those diagnosed with neuromuscular disease. Items are administered following the developmental sequence, as appropriate for age and ability. Maximum score is 100 points.
Secondary Outcome Measures
Language (expressive and receptive), Social and Fine Motor skills at 24 months as assessed by the Bayley-4 Scales of Infant and Toddler Development
The Bayley Scales of Infant and Toddler Development (Bayley-4) are recognized internationally as a comprehensive tool to assess children from as young as 15 days old. With Bayley-4, it is possible to obtain detailed information from non-verbal children as to their functioning. Children are assessed in the five key developmental domains of cognition, language (receptive and expressive), & motor (fine and gross).
Linear growth
We have previously shown that all infants and young children treated with 10mg/kg/week did maintain their linear growth. This protocol is designed to determine also if the lower dose will still maintain benefit.
Full Information
NCT ID
NCT05412394
First Posted
February 2, 2022
Last Updated
September 15, 2023
Sponsor
Anne M. Connolly
Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago, Children's Hospital Medical Center, Cincinnati, Muscular Dystrophy Association, University of Texas, Virginia Commonwealth University
1. Study Identification
Unique Protocol Identification Number
NCT05412394
Brief Title
Once Weekly Infant Corticosteroid Trial for DMD
Official Title
Phase-2 Trial of 5mg/kg/Week Prednisolone in Young Boys With DMD
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 30, 2021 (Actual)
Primary Completion Date
August 30, 2026 (Anticipated)
Study Completion Date
December 30, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Anne M. Connolly
Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago, Children's Hospital Medical Center, Cincinnati, Muscular Dystrophy Association, University of Texas, Virginia Commonwealth University
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.
Detailed Description
The investigators know that the muscle destruction from Duchenne muscular dystrophy (DMD) begins in infancy and we previously demonstrated that motor decline in infancy compared to typically developing infants reflects that destruction. Because of the known side effects of daily corticosteroids, most physicians do not begin treatment until age 3-5 years. Most side effects (Cushingoid faces, linear growth arrest, and bone density loss) did not happen in infants and in ambulatory boys in two separate studies. However, in infants and young boys taking (10mg/kg/week), 56% of infants and young boys did have an increase in weight percentile compared to baseline. This study will test this lower dose of prednisolone (5mg/kg/week) in an unblinded study in infants and young DMD boys (ages 1 through 30 months) to determine if equal efficacy can be achieved with fewer side effects. The primary outcome for this study will be gross motor function. The study team will enroll boys from age 1 month through 30 months and follow each for two years. We will assess gross motor function using three outcome measures: the Bayley-4 Scales of Infant and Toddler Development (Bayley-4) and the newly developed Neuromuscular Gross Motor Outcomes (GRO) and the NorthStar Ambulatory Assessment (NSAA). The one-year outcome will be the change in the Bayley-4 Scaled Score and the two-year outcome will be the change in the GRO score. This study will determine if a lower dose is equally effective and if that dose may lessen the weight gain seen in about half of the infants in the first study. Both the Bayley-4 and the GRO allow assessment of gross motor function and are feasible in all boys with DMD under the age of 42 months. In addition, the GRO allows continued assessment of motor function across a wide age span which will allow this cohort to be followed for two full years.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne Muscular Dystrophy, DMD, Steroid, Muscular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
26 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Experimental
Arm Type
Experimental
Arm Description
This is a one-arm study and the group of subjects are all experimental and will receive drug.
Intervention Type
Drug
Intervention Name(s)
Prednisolone
Other Intervention Name(s)
Corticosteroid
Intervention Description
Liquid, 5mg/kg per week, for one year
Primary Outcome Measure Information:
Title
The change from baseline to 24 months for the Gross Motor Scaled Score.
Description
Neuromuscular Gross Motor Outcome (GRO): The Neuromuscular GRO is a gross motor outcome measure developed to assess whole body strength, motor development, and function for all levels of ability across the lifespan in those diagnosed with neuromuscular disease. Items are administered following the developmental sequence, as appropriate for age and ability. Maximum score is 100 points.
Time Frame
Baseline visit to 24 month visit
Secondary Outcome Measure Information:
Title
Language (expressive and receptive), Social and Fine Motor skills at 24 months as assessed by the Bayley-4 Scales of Infant and Toddler Development
Description
The Bayley Scales of Infant and Toddler Development (Bayley-4) are recognized internationally as a comprehensive tool to assess children from as young as 15 days old. With Bayley-4, it is possible to obtain detailed information from non-verbal children as to their functioning. Children are assessed in the five key developmental domains of cognition, language (receptive and expressive), & motor (fine and gross).
Time Frame
Baseline visit to 24 month visit
Title
Linear growth
Description
We have previously shown that all infants and young children treated with 10mg/kg/week did maintain their linear growth. This protocol is designed to determine also if the lower dose will still maintain benefit.
Time Frame
Baseline visit to 24 month visit
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
30 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
Prior treatment with Glucocorticosteroids
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Kevin Warf
Phone
614-355-2765
Email
kevin.warf@nationwidechildrens.org
First Name & Middle Initial & Last Name or Official Title & Degree
Cosob Barre
Phone
(614) 722-4883
Email
cosob.barre@nationwidechildrens.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anne Connolly, MD
Organizational Affiliation
Nationwide Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Cincinnati
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45220
Country
United States
Individual Site Status
Recruiting
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kevin Warf, BS
Phone
614-355-2765
Email
kevin.warf@nationwidechildrens.org
First Name & Middle Initial & Last Name & Degree
Anne Connolly, MD
Facility Name
University of Texas Southwestern
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23284
Country
United States
Individual Site Status
Not yet recruiting
12. IPD Sharing Statement
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Once Weekly Infant Corticosteroid Trial for DMD
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