Finding the Optimum Regimen for Duchenne Muscular Dystrophy

NCT01603407 | Completed Phase 3 Results DMC

• 3 other identifiers: U01NS0617992010-023744-3346102316
• Study Type: interventional
• Target: 196
• Locations: 5 countries
• Sites: 32

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

prednisone; deflazacort; muscular dystrophy; neuromuscular disease; multi-center

Outcome Measures

Primary Outcomes (3)

• 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.

  Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

• Rise From the Floor Velocity

  Reciprocal of time to rise from the floor

  Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

• 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.

  Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Secondary Outcomes (14)

• North Star Ambulatory Assessment (NSAA) Score

  Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

• 6 Minute Walk Test

  Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

• Range of Motion (Goniometry) of Left Ankle

  Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

• Range of Motion (Goniometry) of Right Ankle

  Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

• Number of Participants Who Tolerated the Regimen

  3 years

Study Arms (3)

Daily prednisone

EXPERIMENTAL

daily prednisone (0.75 mg/kg/day)

Drug: Prednisone

Intermittent prednisone

EXPERIMENTAL

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

Drug: Prednisone

Daily deflazacort

EXPERIMENTAL

daily deflazacort (0.9 mg/kg/day

Drug: Deflazacort

Interventions

Prednisone DRUG

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

Daily prednisone

Deflazacort DRUG

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

Daily deflazacort

Eligibility Criteria

• Age: 4 Years - 7 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• University of Rochester: lead
• Newcastle University: collaborator
• University Medical Center Freiburg: collaborator
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (32)

University of California Los Angeles (UCLA) Medical Center

Los Angeles, California, 90095, United States

Location

University of California Davis Medical Center

Sacramento, California, 95817, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Nemours Children's Hospital

Orlando, Florida, 32827, United States

Location

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

Chicago, Illinois, 60614, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

University of New Mexico Health Science Center

Albuquerque, New Mexico, 87131, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Vanderbilt Children's Hospital

Nashville, Tennessee, 37232, United States

Location

University of Utah Medical Center

Salt Lake City, Utah, 84132, United States

Location

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

Location

Children's Hospital London Health Sciences Centre

London, Ontario, N6A 4G5, Canada

Location

Children's Hospital of Eastern Ontario (CHEO)

Ottawa, Ontario, K1H 8L1, Canada

Location

Children's Hospital, Technical University Dresden

Dresden, 01307, Germany

Location

University Hospital of Essen

Essen, 45122, Germany

Location

University Medical Center Freiburg

Freiburg im Breisgau, 79110, Germany

Location

Children's University Hospital, Göttingen

Göttingen, 37075, Germany

Location

University of Messina AOU Policlinico Gaetano Martino

Messina, 98125, Italy

Location

C. Besta Neurological Institute Foundation

Milan, 20133, Italy

Location

University of Padova School of Medicine

Padua, 35128, Italy

Location

Neuromuscular Center University of Turin

Torino, 10126, Italy

Location

The General Infirmary at Leeds

Leeds, England, LS2 9NS, United Kingdom

Location

Greater Glasgow and Clyde NHS Yorkhill Hospital

Glasgow, Scotland, G3 8SJ, United Kingdom

Location

Heart of England NHS Foundation Trust Birmingham Heartland's Hospital

Birmingham, B9 5SS, United Kingdom

Location

Alder Hey Children's Hospital NHS Trust

Liverpool, L12 2AP, United Kingdom

Location

Great Ormond Street Hospital for Children NHS Trust

London, WC1N 3JH, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

Institute of Human Genetics, International Centre for Life

Newcastle upon Tyne, NE1 3BZ, United Kingdom

Location

Related Publications (58)

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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.

  PMID: 35381069 DERIVED

• 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.

  PMID: 34857536 DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies; Neuromuscular Diseases

Interventions

Prednisone; deflazacort

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Results Point of Contact

• Title: Robert Griggs
• Organization: University of Rochester

robert_griggs@urmc.rochester.edu

585-275-6072

Study Officials

• Robert C. Griggs, MD

  University of Rochester

  PRINCIPAL INVESTIGATOR

• Kate Bushby, MD

  Newcastle University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: April 3, 2012
• First Posted: May 23, 2012
• Study Start: January 1, 2013
• Primary Completion: November 1, 2019
• Study Completion: November 1, 2019
• Last Updated: August 12, 2022
• Results First Posted: August 12, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3); IT (4); GB (7); US (14); DE (4)