To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to \<12 years).
This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in participants with DMD, aged 6 to \<12 years (ambulatory participants only). Approximately 70 participants will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic deflazacort or equivalent potency of corticosteroids administered orally) or Arm B (placebo+ systemic deflazacort or equivalent potency of corticosteroids administered orally), respectively. Randomization will be stratified by exon 44 deletion for analysis. Stratification has no impact upon treatment assignment nor dosage. Participants must be fully informed of the potential benefits of approved products and make an informed decision when participating in a clinical trial in which they could be randomized to placebo. The main study has 3 study periods: * Screening period: Up to 4 weeks * Treatment period: 52 weeks * Safety Follow-up period/final assessment: A visit 28 days (+/- 3 Days) and a final safety follow-up phone call 60 days (+ 3 Days) after the last dose Each participant will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks. Participants who complete 52 weeks of treatment may be eligible for an open-label extension (OLE), offering extended treatment with pamrevlumab. Participants who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
73
Pamrevlumab will be administered per dose and schedule specified in the arm description.
Placebo will be administered per schedule specified in the arm description.
Systemic deflazacort or equivalent potency of corticosteroids administered orally
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score ranging from 0 (worst) to 34 (fully independent function). If fewer than 15 of the 17 activities were performed, the total score was considered missing. If 15 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. Higher scores indicated better functioning. Least square (LS) mean and standard error (SE) were analyzed using a mixed model for repeated measure (MMRM).
Time frame: Baseline, Week 52
Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
The 4SCV (centimeters \[cm\]/second \[sec\]) was calculated as the ratio of the total height (cm) of stairs climbed divided by the number of seconds taken to complete the 4-stair climb.
Time frame: Baseline, Week 52
Change From Baseline in the 10-Meter Walk/Run Test at Week 52
The time (in sec) required for a participant to run or walk a distance of 10 meters as quickly as possible was calculated as velocity (meters/sec).
Time frame: Baseline, Week 52
Change From Baseline in Time to Stand (TTSTAND) at Week 52
The time (in sec) required for a participant to stand from supine position has been reported. A longer time taken reflected a worse outcome.
Time frame: Baseline, Week 52
Time to Loss of Ambulation (LoA) From Baseline to Week 52
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Arkansas Children's Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of California Davis Children's Hospital
Sacramento, California, United States
University of California San Diego Health
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
University of Florida Health Shands Hospital
Gainesville, Florida, United States
Rare Disease Research - Tampa
Tampa, Florida, United States
Rare Disease Research Center
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
...and 42 more locations
Time (days) to LoA was defined as the number of days from randomization to the date of LoA, or all-cause death based on observed data, whichever occurred earlier during the on-study period. Median time (days) to LoA was calculated using Kaplan Meier Survival Estimates.
Time frame: Baseline to Week 52