Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy: Neuromuscular Disorders

K.R. Wagner, H.Z. Abdel-Hamid, J.K. Mah, C. Campbell, M. Guglieri, F. Muntoni, Y. Takeshima, C.M. McDonald, A. Kostera-Pruszczyk, P. Karachunski, R.J. Butterfield, E. Mercuri, C. Fiorillo, E.S. Bertini, C. Tian, J. Statland, A.B. Sadosky, V.S. Purohit, S.P. Sherlock, J.P. PalmerM. Binks, L. Charnas, S. Marraffino, B.L. Wong

Research output: Contribution to journalArticlepeer-review


We report results from a phase 2, randomized, double-blind, 2-period trial (48 weeks each) of domagrozumab and its open-label extension in patients with Duchenne muscular dystrophy (DMD). Of 120 ambulatory boys (aged 6 to <16 years) with DMD, 80 were treated with multiple ascending doses (5, 20, and 40 mg/kg) of domagrozumab and 40 treated with placebo. The primary endpoints were safety and mean change in 4-stair climb (4SC) time at week 49. Secondary endpoints included other functional tests, pharmacokinetics, and pharmacodynamics. Mean (SD) age was 8.4 (1.7) and 9.3 (2.3) years in domagrozumab- and placebo-treated patients, respectively. Difference in mean (95% CI) change from baseline in 4SC at week 49 for domagrozumab vs placebo was 0.27 (–7.4 to 7.9) seconds (p = 0.94). There were no significant between-group differences in any secondary clinical endpoints. Most patients had ≥1 adverse event in the first 48 weeks; most were mild and not treatment-related. Median serum concentrations of domagrozumab increased with administered dose within each dose level. Non-significant increases in muscle volume were observed in domagrozumab- vs placebo-treated patients. Domagrozumab was generally safe and well tolerated in patients with DMD. Efficacy measures did not support a significant treatment effect. Clinicaltrials.gov identifiers: NCT02310763 and NCT02907619

Original languageEnglish
Pages (from-to)492-502
Number of pages11
JournalNeuromuscular Disord.
Issue number6
Publication statusPublished - Jun 2020


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