Finding the Optimum Regimen for Duchenne Muscular Dystrophy

NCT ID: NCT01603407

Last Updated: 2022-08-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 196 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-01-31
• Study Completion Date: 2019-11-30

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:

1. Prednisone 0.75mg/kg/day

2. Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment

3. 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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Daily prednisone

daily prednisone (0.75 mg/kg/day)

Group Type: EXPERIMENTAL

Prednisone

Intervention Type: DRUG

daily prednisone (0.75 mg/kg/day) tablets for 36-60 months

Intermittent prednisone

intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)

Group Type: EXPERIMENTAL

Prednisone

Intervention Type: DRUG

intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months

Daily deflazacort

daily deflazacort (0.9 mg/kg/day

Group Type: EXPERIMENTAL

Deflazacort

Intervention Type: DRUG

daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months

Interventions

Prednisone

daily prednisone (0.75 mg/kg/day) tablets for 36-60 months

Intervention Type: DRUG

Prednisone

intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months

Intervention Type: DRUG

Deflazacort

daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months

Intervention Type: DRUG

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.

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 7 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Newcastle University

OTHER

Sponsor Role: collaborator

University Medical Center Freiburg

OTHER

Sponsor Role: collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

University of Rochester

OTHER

Sponsor Role: lead

Responsible Party

Robert Griggs, MD

Professor of Neurology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Robert C. Griggs, MD

Role: PRINCIPAL_INVESTIGATOR

University of Rochester

Kate Bushby, MD

Role: PRINCIPAL_INVESTIGATOR

Newcastle University

Locations

University of California Los Angeles (UCLA) Medical Center

Los Angeles, California, United States

University of California Davis Medical Center

Sacramento, California, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Nemours Children's Hospital

Orlando, Florida, United States

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Boston Children's Hospital

Boston, Massachusetts, United States

University of New Mexico Health Science Center

Albuquerque, New Mexico, United States

University of Rochester Medical Center

Rochester, New York, United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Penn State Hershey Medical Center

Hershey, Pennsylvania, United States

Vanderbilt Children's Hospital

Nashville, Tennessee, United States

University of Utah Medical Center

Salt Lake City, Utah, United States

Alberta Children's Hospital

Calgary, Alberta, Canada

Children's Hospital London Health Sciences Centre

London, Ontario, Canada

Children's Hospital of Eastern Ontario (CHEO)

Ottawa, Ontario, Canada

Children's Hospital, Technical University Dresden

Dresden, , Germany

University Hospital of Essen

Essen, , Germany

University Medical Center Freiburg

Freiburg im Breisgau, , Germany

Children's University Hospital, Göttingen

Göttingen, , Germany

University of Messina AOU Policlinico Gaetano Martino

Messina, , Italy

C. Besta Neurological Institute Foundation

Milan, , Italy

University of Padova School of Medicine

Padua, , Italy

Neuromuscular Center University of Turin

Torino, , Italy

The General Infirmary at Leeds

Leeds, England, United Kingdom

Greater Glasgow and Clyde NHS Yorkhill Hospital

Glasgow, Scotland, United Kingdom

Heart of England NHS Foundation Trust Birmingham Heartland's Hospital

Birmingham, , United Kingdom

Alder Hey Children's Hospital NHS Trust

Liverpool, , United Kingdom

Great Ormond Street Hospital for Children NHS Trust

London, , United Kingdom

Royal Manchester Children's Hospital

Manchester, , United Kingdom

Institute of Human Genetics, International Centre for Life

Newcastle upon Tyne, , United Kingdom

Countries

United States; Canada; Germany; Italy; United Kingdom

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Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Speed C, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Griggs RC; FOR-DMD Investigators of the Muscle Study Group; Straub V, van Ruiten H, Childs AM, Ciafaloni E, Shieh PB, Spinty S, Maggi L, Baranello G, Butterfield RJ, Horrocks IA, Roper H, Alhaswani Z, Flanigan KM, Kuntz NL, Manzur A, Darras BT, Kang PB, Morrison L, Krzesniak-Swinarska M, Mah JK, Mongini TE, Ricci F, von der Hagen M, Finkel RS, O'Reardon K, Wicklund M, Kumar A, McDonald CM, Han JJ, Joyce N, Henricson EK, Schara-Schmidt U, Gangfuss A, Wilichowski E, Barohn RJ, Statland JM, Campbell C, Vita G, Vita GL, Howard JF Jr, Hughes I, McMillan HJ, Pegoraro E, Bello L, Burnette WB, Thangarajh M, Chang T. Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA. 2022 Apr 19;327(15):1456-1468. doi: 10.1001/jama.2022.4315.

Reference Type: DERIVED

PMID: 35381069 (View on PubMed)

Schiava M, Amos R, VanRuiten H, McDermott MP, Martens WB, Gregory S, Mayhew A, McColl E, Tawil R, Willis T, Bushby K, Griggs RC, Guglieri M; FOR-DMD Investigators of the Muscle Study Group. Clinical and Genetic Characteristics in Young, Glucocorticoid-Naive Boys With Duchenne Muscular Dystrophy. Neurology. 2022 Jan 24;98(4):e390-e401. doi: 10.1212/WNL.0000000000013122.

Reference Type: DERIVED

PMID: 34857536 (View on PubMed)

Provided Documents

Document Type: Study Protocol

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Document Type: Statistical Analysis Plan

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Other Identifiers

2010-023744-33

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U01NS061799

Identifier Type: NIH

Identifier Source: secondary_id

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46102316

Identifier Type: REGISTRY

Identifier Source: secondary_id

U01NS061799

Identifier Type: NIH

Identifier Source: org_study_id

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