Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD)
Primary Purpose
Duchenne Muscular Dystrophy
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Prednisone
Prednisone
Deflazacort
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring prednisone, deflazacort, muscular dystrophy, neuromuscular disease, multi-center
Eligibility Criteria
Inclusion Criteria:
- Evidence of signed and dated informed consent form.
- Confirmed diagnosis of Duchenne muscular dystrophy
- Age greater than or equal to 4 years and less than 8 years old
- Ability to rise independently from floor, from supine to standing
- Willingness and ability to comply with scheduled visits, drug administration plan and study procedures
- Ability to maintain reproducible FVC measurements.
Exclusion Criteria:
- History of major renal or hepatic impairment, immunosuppression or other contraindications to corticosteroid therapy.
- History of chronic systemic fungal or viral infections. Acute bacterial infection(including TB) would exclude from enrolment until the infection had been appropriately treated and resolved.
- Diabetes mellitus.
- Idiopathic hypercalcuria.
- Lack of chicken pox immunity and refusal to undergo immunization.
- Evidence of symptomatic cardiomyopathy at screening assessment (one to three months prior to the baseline visit). Asymptomatic cardiac abnormality on investigation would not be an exclusion.
- Current or previous treatment (greater than four consecutive weeks of oral therapy) with corticosteroids or other immunosuppressive treatments for DMD or other recurrent indications (e.g., asthma), unless approved by FOR-DMD Team (i.e., concurrent participation in another allowed DMD trial).
- Inability to take tablets, as assessed by the site investigator by the end of the screening period (the screening period ranges from one to three months prior to the baseline visit).
- Allergy/sensitivity to study drugs or their formulations including lactose and/or sucrose intolerance.
- Severe behavioral problems, including severe autism.
- Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow up will be correctly completed or impair the assessment of study results, in the judgment of the site investigator.
- Weight of less than 13 kilograms.
- Exposure to any investigational drug currently or within 3 months prior to start of study treatment.
Sites / Locations
- University of California Los Angeles (UCLA) Medical Center
- University of California Davis Medical Center
- Children's National Medical Center
- Nemours Children's Hospital
- Ann and Robert H. Lurie Children's Hospital of Chicago
- University of Kansas Medical Center
- Boston Children's Hospital
- University of New Mexico Health Science Center
- University of Rochester Medical Center
- University of North Carolina at Chapel Hill
- Nationwide Children's Hospital
- Penn State Hershey Medical Center
- Vanderbilt Children's Hospital
- University of Utah Medical Center
- Alberta Children's Hospital
- Children's Hospital London Health Sciences Centre
- Children's Hospital of Eastern Ontario (CHEO)
- Children's Hospital, Technical University Dresden
- University Hospital of Essen
- University Medical Center Freiburg
- Children's University Hospital, Göttingen
- University of Messina AOU Policlinico Gaetano Martino
- C. Besta Neurological Institute Foundation
- University of Padova School of Medicine
- Neuromuscular Center University of Turin
- The General Infirmary at Leeds
- Greater Glasgow and Clyde NHS Yorkhill Hospital
- Heart of England NHS Foundation Trust Birmingham Heartland's Hospital
- Alder Hey Children's Hospital NHS Trust
- Great Ormond Street Hospital for Children NHS Trust
- Royal Manchester Children's Hospital
- Institute of Human Genetics, International Centre for Life
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
Daily prednisone
Intermittent prednisone
Daily deflazacort
Arm Description
daily prednisone (0.75 mg/kg/day)
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
daily deflazacort (0.9 mg/kg/day
Outcomes
Primary Outcome Measures
Forced Vital Capacity
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Rise From the Floor Velocity
Reciprocal of time to rise from the floor
Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes.
Secondary Outcome Measures
North Star Ambulatory Assessment (NSAA) Score
The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects.
The activities are graded as follows:
2 - "Normal" - no obvious modification of activity
1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function.
6 Minute Walk Test
Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36.
Range of Motion (Goniometry) of Left Ankle
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Range of Motion (Goniometry) of Right Ankle
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Number of Participants Who Tolerated the Regimen
The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication.
Heart Rate
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Quality of Life - Parent
Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child.
Quality of Life- Child
Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life.
Left Ventricular Ejection Fraction Percent
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Fractional Shortening Percent
Measured by trans-thoracic echocardiogram and 12-lead ECG.
PR Interval
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Participant Weight
Participant Height
Participant Body Mass Index
Full Information
NCT ID
NCT01603407
First Posted
April 3, 2012
Last Updated
August 11, 2022
Sponsor
University of Rochester
Collaborators
Newcastle University, University Medical Center Freiburg, National Institute of Neurological Disorders and Stroke (NINDS)
1. Study Identification
Unique Protocol Identification Number
NCT01603407
Brief Title
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
Acronym
FOR-DMD
Official Title
Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
Study Type
Interventional
2. Study Status
Record Verification Date
August 2022
Overall Recruitment Status
Completed
Study Start Date
January 2013 (undefined)
Primary Completion Date
November 2019 (Actual)
Study Completion Date
November 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Rochester
Collaborators
Newcastle University, University Medical Center Freiburg, National Institute of Neurological Disorders and Stroke (NINDS)
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.
Detailed Description
Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up. Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems.
Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug. No controlled, long-term study has ever looked at the effects of different corticosteroids to see which one improves strength the most and which one causes the fewest side effects, over a period of time. Different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all.
The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of taking the following corticosteroids by the mouth to determine which increases muscle strength the most, and which causes the fewest side effects:
Prednisone 0.75mg/kg/day
Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment
Deflazacort 0.9mg/kg/day.
The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany and Italy.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
prednisone, deflazacort, muscular dystrophy, neuromuscular disease, multi-center
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
196 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Daily prednisone
Arm Type
Experimental
Arm Description
daily prednisone (0.75 mg/kg/day)
Arm Title
Intermittent prednisone
Arm Type
Experimental
Arm Description
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Arm Title
Daily deflazacort
Arm Type
Experimental
Arm Description
daily deflazacort (0.9 mg/kg/day
Intervention Type
Drug
Intervention Name(s)
Prednisone
Intervention Description
daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
Intervention Type
Drug
Intervention Name(s)
Prednisone
Intervention Description
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
Intervention Type
Drug
Intervention Name(s)
Deflazacort
Intervention Description
daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
Primary Outcome Measure Information:
Title
Forced Vital Capacity
Description
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Time Frame
Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Title
Rise From the Floor Velocity
Description
Reciprocal of time to rise from the floor
Time Frame
Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Title
Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
Description
The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes.
Time Frame
Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Secondary Outcome Measure Information:
Title
North Star Ambulatory Assessment (NSAA) Score
Description
The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects.
The activities are graded as follows:
2 - "Normal" - no obvious modification of activity
1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function.
Time Frame
Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Title
6 Minute Walk Test
Description
Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36.
Time Frame
Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Title
Range of Motion (Goniometry) of Left Ankle
Description
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Time Frame
Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Title
Range of Motion (Goniometry) of Right Ankle
Description
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Time Frame
Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Title
Number of Participants Who Tolerated the Regimen
Description
The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication.
Time Frame
3 years
Title
Heart Rate
Description
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time Frame
36 months
Title
Quality of Life - Parent
Description
Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child.
Time Frame
Average of Months 12, 24, and 36 visits
Title
Quality of Life- Child
Description
Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life.
Time Frame
Average of Months 12, 24, and 36 visits
Title
Left Ventricular Ejection Fraction Percent
Description
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time Frame
36 months
Title
Fractional Shortening Percent
Description
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time Frame
36 months
Title
PR Interval
Description
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time Frame
36 months
Title
Participant Weight
Time Frame
36 months
Title
Participant Height
Time Frame
36 months
Title
Participant Body Mass Index
Time Frame
36 months
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Evidence of signed and dated informed consent form.
Confirmed diagnosis of Duchenne muscular dystrophy
Age greater than or equal to 4 years and less than 8 years old
Ability to rise independently from floor, from supine to standing
Willingness and ability to comply with scheduled visits, drug administration plan and study procedures
Ability to maintain reproducible FVC measurements.
Exclusion Criteria:
History of major renal or hepatic impairment, immunosuppression or other contraindications to corticosteroid therapy.
History of chronic systemic fungal or viral infections. Acute bacterial infection(including TB) would exclude from enrolment until the infection had been appropriately treated and resolved.
Diabetes mellitus.
Idiopathic hypercalcuria.
Lack of chicken pox immunity and refusal to undergo immunization.
Evidence of symptomatic cardiomyopathy at screening assessment (one to three months prior to the baseline visit). Asymptomatic cardiac abnormality on investigation would not be an exclusion.
Current or previous treatment (greater than four consecutive weeks of oral therapy) with corticosteroids or other immunosuppressive treatments for DMD or other recurrent indications (e.g., asthma), unless approved by FOR-DMD Team (i.e., concurrent participation in another allowed DMD trial).
Inability to take tablets, as assessed by the site investigator by the end of the screening period (the screening period ranges from one to three months prior to the baseline visit).
Allergy/sensitivity to study drugs or their formulations including lactose and/or sucrose intolerance.
Severe behavioral problems, including severe autism.
Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow up will be correctly completed or impair the assessment of study results, in the judgment of the site investigator.
Weight of less than 13 kilograms.
Exposure to any investigational drug currently or within 3 months prior to start of study treatment.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert C. Griggs, MD
Organizational Affiliation
University of Rochester
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Kate Bushby, MD
Organizational Affiliation
Newcastle University
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of California Los Angeles (UCLA) Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
University of California Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Nemours Children's Hospital
City
Orlando
State/Province
Florida
ZIP/Postal Code
32827
Country
United States
Facility Name
Ann and Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of New Mexico Health Science Center
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87131
Country
United States
Facility Name
University of Rochester Medical Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
University of North Carolina at Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Penn State Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Vanderbilt Children's Hospital
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
University of Utah Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Alberta Children's Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3B 6A8
Country
Canada
Facility Name
Children's Hospital London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 4G5
Country
Canada
Facility Name
Children's Hospital of Eastern Ontario (CHEO)
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Facility Name
Children's Hospital, Technical University Dresden
City
Dresden
ZIP/Postal Code
01307
Country
Germany
Facility Name
University Hospital of Essen
City
Essen
ZIP/Postal Code
45122
Country
Germany
Facility Name
University Medical Center Freiburg
City
Freiburg
ZIP/Postal Code
79110
Country
Germany
Facility Name
Children's University Hospital, Göttingen
City
Göttingen
ZIP/Postal Code
37075
Country
Germany
Facility Name
University of Messina AOU Policlinico Gaetano Martino
City
Messina
ZIP/Postal Code
98125
Country
Italy
Facility Name
C. Besta Neurological Institute Foundation
City
Milan
ZIP/Postal Code
20133
Country
Italy
Facility Name
University of Padova School of Medicine
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Neuromuscular Center University of Turin
City
Torino
ZIP/Postal Code
10126
Country
Italy
Facility Name
The General Infirmary at Leeds
City
Leeds
State/Province
England
ZIP/Postal Code
LS2 9NS
Country
United Kingdom
Facility Name
Greater Glasgow and Clyde NHS Yorkhill Hospital
City
Glasgow
State/Province
Scotland
ZIP/Postal Code
G3 8SJ
Country
United Kingdom
Facility Name
Heart of England NHS Foundation Trust Birmingham Heartland's Hospital
City
Birmingham
ZIP/Postal Code
B9 5SS
Country
United Kingdom
Facility Name
Alder Hey Children's Hospital NHS Trust
City
Liverpool
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Facility Name
Great Ormond Street Hospital for Children NHS Trust
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Name
Royal Manchester Children's Hospital
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Facility Name
Institute of Human Genetics, International Centre for Life
City
Newcastle Upon Tyne
ZIP/Postal Code
NE1 3BZ
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
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Finding the Optimum Regimen for Duchenne Muscular Dystrophy
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