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FOR-DMD: Corticosteroid Regimens in Duchenne Muscular Dystrophy
NCT01603407Phase 3
Key facts
- Status
- Completed
- Start
- Jan 2013
- Primary completion
- Nov 2019
- Countries
- 5
- Sponsor
- University of Rochester
- Modality
- Orthosteric Ligands
- Conditions
- Duchenne muscular dystrophy (DMD)
01
Overview
Phase 3, randomised, double-blind trial of prednisone and deflazacort in Duchenne muscular dystrophy (DMD) (3 arms), sponsored by University of Rochester. Primary endpoints: Forced Vital Capacity at 36 months, Rise From the Floor Velocity at 36 months and 1 more. Started Jan 2013; status: completed.
02
Design
3 arms- Study design
- RandomizedDouble-blind
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
03
Who it enrols
Boys aged 4-7 with confirmed Duchenne muscular dystrophy who are able to rise independently from the floor and have not received long-term corticosteroid treatment.
04
Endpoints
Primary
Forced Vital Capacity
Rise From the Floor Velocity
Treatment Satisfaction Questionnaire for Medication Global Satisfaction With Treatment Score
Secondary
North Star Ambulatory Assessment
6 Minute Walk Test
Range of Motion of Left/Right Ankle
Number of Participants Who Tolerated the Regimen
Heart Rate
Quality of Life (Parent and Child)
Left Ventricular Ejection Fraction
Fractional Shortening Percent
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