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Wagner, Kathryn R.; Abdel-Hamid, Hoda Z.; Mah, Jean K.; Campbell, Craig; Guglieri, Michela; Muntoni, Francesco; Takeshima, Yasuhiro; McDonald, Craig M.; Kostera-Pruszczyk, Anna; Karachunski, Peter; Butterfield, Russell J.; Mercuri, Eugenio; Fiorillo, Chiara; Bertini, Enrico S.; Tian, Cuixia; Statland, Jeffery; Sadosky, Alesia B.; Purohit, Vivek S.; Sherlock, Sarah P.; Palmer, Jeffrey P.; Binks, Michael; Charnas, Lawrence; Marraffino, Shannon; Wong, Brenda L.
Neuromuscular disorders : NMD, June 2020, 2020-06-00, Letnik: 30, Številka: 6Journal Article
•Domagrozumab at 5, 20, and 40 mg/kg was generally safe and well tolerated.•Primary endpoint of mean change from baseline in 4SC time at week 49 was not met.•Efficacy measures did not support a significant treatment effect with domagrozumab.•Nonsignificant increase in muscle volume was observed with domagrozumab vs placebo. 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
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