Once Weekly Infant Corticosteroid Trial for DMD

NCT05412394 | Recruiting Phase 4 DMC FDA Drug

• 1 other identifier: Infant Steroid Phase II
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 4

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; DMD; Steroid; Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline visit to 24 month visit

Secondary Outcomes (2)

• Language (expressive and receptive), Social and Fine Motor skills at 24 months as assessed by the Bayley-4 Scales of Infant and Toddler Development

  Baseline visit to 24 month visit

• Linear growth

  Baseline visit to 24 month visit

Study Arms (1)

Experimental

EXPERIMENTAL

This is a one-arm study and the group of subjects are all experimental and will receive drug.

Drug: Prednisolone

Interventions

Prednisolone DRUG

Liquid, 5mg/kg per week, for one year

Also known as: Corticosteroid

Experimental

Eligibility Criteria

• Age: 1 Month - 30 Months
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• Prior treatment with Glucocorticosteroids

Sponsors & Collaborators

• Anne M. Connolly: lead
• Children's Hospital Medical Center, Cincinnati: collaborator
• Muscular Dystrophy Association: collaborator
• University of Texas: collaborator
• Virginia Commonwealth University: collaborator
• Ann & Robert H Lurie Children's Hospital of Chicago: collaborator

Study Sites (4)

Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

RECRUITING

University of Cincinnati

Cincinnati, Ohio, 45220, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

University of Texas Southwestern

Dallas, Texas, 75235, United States

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Interventions

Prednisolone; Adrenal Cortex Hormones

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

Study Officials

• Anne Connolly, MD

  Nationwide Children's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sara Marshall

CONTACT

614-355-3508; sara.adamczak@nationwidechildrens.org

Kevin Warf

CONTACT

614-355-2765; kevin.warf@nationwidechildrens.org

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief of Division of Neurology

Study Record Dates

• First Submitted: February 2, 2022
• First Posted: June 9, 2022
• Study Start: April 30, 2021
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: August 13, 2025

Record last verified: 2025-02

Locations

US (4)