Once Weekly Infant Corticosteroid Trial for DMD

September 15, 2023 updated by: Anne M. Connolly

Phase-2 Trial of 5mg/kg/Week Prednisolone in Young Boys With DMD

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

26

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Lurie Children's Hospital of Chicago
    • Ohio
      • Cincinnati, Ohio, United States, 45220
        • Recruiting
        • University of Cincinnati
      • Columbus, Ohio, United States, 43205
        • Recruiting
        • Nationwide Children's Hospital
        • Contact:
        • Principal Investigator:
          • Anne Connolly, MD
    • Texas
      • Dallas, Texas, United States, 75235
        • Not yet recruiting
        • University of Texas Southwestern
    • Virginia
      • Richmond, Virginia, United States, 23284
        • Not yet recruiting
        • Virginia Commonwealth University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 1 year (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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 Names:
  • Corticosteroid

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline visit to 24 month visit

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
Baseline visit to 24 month visit
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.
Baseline visit to 24 month visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Anne M. Connolly

Collaborators

Ann & Robert H Lurie Children's Hospital of Chicago
Children's Hospital Medical Center, Cincinnati
University of Texas
Virginia Commonwealth University
Muscular Dystrophy Association

Investigators

  • Principal Investigator: Anne Connolly, MD, Nationwide Children's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 30, 2021

Primary Completion (Estimated)

August 30, 2026

Study Completion (Estimated)

December 30, 2026

Study Registration Dates

First Submitted

February 2, 2022

First Submitted That Met QC Criteria

June 7, 2022

First Posted (Actual)

June 9, 2022

Study Record Updates

Last Update Posted (Actual)

September 18, 2023

Last Update Submitted That Met QC Criteria

September 15, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Infant Steroid Phase II

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Duchenne Muscular Dystrophy

Clinical Trials on Prednisolone

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Georgia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe