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Once Weekly Infant Corticosteroid Trial for DMD

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ClinicalTrials.gov Identifier: NCT05412394
Recruitment Status : Recruiting
First Posted : June 9, 2022
Last Update Posted : June 9, 2022
Sponsor:
Collaborators:
Ann & Robert H Lurie Children's Hospital of Chicago
Children's Hospital Medical Center, Cincinnati
Information provided by (Responsible Party):
Anne M. Connolly, Nationwide Children's Hospital

Study Description
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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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Prednisolone Phase 4

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 26 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase-2 Trial of 5mg/kg/Week Prednisolone in Young Boys With DMD
Actual Study Start Date : April 30, 2021
Estimated Primary Completion Date : August 30, 2024
Estimated Study Completion Date : December 30, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Experimental
This is a one-arm study and the group of subjects are all experimental and will receive drug.
 Drug: Prednisolone
Liquid, 5mg/kg per week, for one year
Other Name: Corticosteroid


Outcome Measures
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Primary Outcome Measures :
  1. The change from baseline to 24 months for the Gross Motor Scaled Score. [ Time Frame: Baseline visit to 24 month visit ]
    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 :
  1. Language (expressive and receptive), Social and Fine Motor skills at 24 months as assessed by the Bayley-4 Scales of Infant and Toddler Development [ Time Frame: Baseline visit to 24 month visit ]
    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).

  2. Linear growth [ Time Frame: Baseline visit to 24 month visit ]
    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.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   1 Month to 30 Months   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05412394


Contacts
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Contact: Kevin Warf 614-355-2765 kevin.warf@nationwidechildrens.org
Contact: Kevin Ignasiak 614-355-2606 kevin.ignasiak@nationwidechildrens.org

Locations
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United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Kevin Warf, BS    614-355-2765    kevin.warf@nationwidechildrens.org   
Principal Investigator: Anne Connolly, MD         
Sponsors and Collaborators
Anne M. Connolly
Ann & Robert H Lurie Children's Hospital of Chicago
Children's Hospital Medical Center, Cincinnati
Investigators
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Principal Investigator: Anne Connolly, MD Nationwide Children's Hospital
More Information
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Responsible Party: Anne M. Connolly, Chief of Division of Neurology, Nationwide Children's Hospital
ClinicalTrials.gov Identifier: NCT05412394    
Other Study ID Numbers: Infant Steroid Phase II
First Posted: June 9, 2022    Key Record Dates
Last Update Posted: June 9, 2022
Last Verified: June 2022

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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
Keywords provided by Anne M. Connolly, Nationwide Children's Hospital:
Duchenne Muscular Dystrophy
DMD
Steroid
Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
 Prednisolone
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents