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Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

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العنوان: Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
المؤلفون: Guglieri, M, Clemens, PR, Perlman, SJ, Smith, EC, Horrocks, I, Finkel, RS, Mah, JK, Deconinck, N, Goemans, N, Haberlova, J, Straub, V, Mengle-Gaw, LJ, Schwartz, BD, Harper, AD, Shieh, PB, De Waele, L, Castro, D, Yang, ML, Ryan, MM, McDonald, CM, Tulinius, M, Webster, R, McMillan, HJ, Kuntz, NL, Rao, VK, Baranello, G, Spinty, S, Childs, A-M, Sbrocchi, AM, Selby, KA, Monduy, M, Nevo, Y, Vilchez-Padilla, JJ, Nascimento-Osorio, A, Niks, EH, de Groot, IJM, Katsalouli, M, James, MK, van den Anker, J, Damsker, JM, Ahmet, A, Ward, LM, Jaros, M, Shale, P, Dang, UJ, Hoffman, EP
بيانات النشر: American Medical Association (AMA)
سنة النشر: 2022
المجموعة: The University of Melbourne: Digital Repository
الوصف: IMPORTANCE: Corticosteroidal anti-inflammatory drugs are widely prescribed but long-term use shows adverse effects that detract from patient quality of life. OBJECTIVE: To determine if vamorolone, a structurally unique dissociative steroidal anti-inflammatory drug, is able to retain efficacy while reducing safety concerns with use in Duchenne muscular dystrophy (DMD). DESIGN, SETTING, AND PARTICIPANTS: Randomized, double-blind, placebo- and prednisone-controlled 24-week clinical trial, conducted from June 29, 2018, to February 24, 2021, with 24 weeks of follow-up. This was a multicenter study (33 referral centers in 11 countries) and included boys 4 to younger than 7 years of age with genetically confirmed DMD not previously treated with corticosteroids. INTERVENTIONS: The study included 4 groups: placebo; prednisone, 0.75 mg/kg per day; vamorolone, 2 mg/kg per day; and vamorolone, 6 mg/kg per day. MAIN OUTCOMES AND MEASURES: Study outcomes monitored (1) efficacy, which included motor outcomes (primary: time to stand from supine velocity in the vamorolone, 6 mg/kg per day, group vs placebo; secondary: time to stand from supine velocity [vamorolone, 2 mg/kg per day], 6-minute walk distance, time to run/walk 10 m [vamorolone, 2 and 6 mg/kg per day]; exploratory: NorthStar Ambulatory Assessment, time to climb 4 stairs) and (2) safety, which included growth, bone biomarkers, and a corticotropin (ACTH)-challenge test. RESULTS: Among the 133 boys with DMD enrolled in the study (mean [SD] age, 5.4 [0.9] years), 121 were randomly assigned to treatment groups, and 114 completed the 24-week treatment period. The trial met the primary end point for change from baseline to week 24 time to stand velocity for vamorolone, 6 mg/kg per day (least-squares mean [SE] velocity, 0.05 [0.01] m/s vs placebo -0.01 [0.01] m/s; 95% CI, 0.02-0.10; P = .002) and the first 4 sequential secondary end points: time to stand velocity, vamorolone, 2 mg/kg per day, vs placebo; 6-minute walk test, vamorolone, 6 mg/kg per day, vs placebo; 6-minute ...
نوع الوثيقة: article in journal/newspaper
اللغة: English
تدمد: 2168-6149
2168-6157
Relation: pii: 2795868; Guglieri, M., Clemens, P. R., Perlman, S. J., Smith, E. C., Horrocks, I., Finkel, R. S., Mah, J. K., Deconinck, N., Goemans, N., Haberlova, J., Straub, V., Mengle-Gaw, L. J., Schwartz, B. D., Harper, A. D., Shieh, P. B., De Waele, L., Castro, D., Yang, M. L., Ryan, M. M. ,. Hoffman, E. P. (2022). Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Neurol, 79 (10), pp.1005-1014. https://doi.org/10.1001/jamaneurol.2022.2480.; http://hdl.handle.net/11343/320255
الاتاحة: http://hdl.handle.net/11343/320255
Rights: CC BY ; https://creativecommons.org/licenses/by/4.0
رقم الانضمام: edsbas.D1655328
قاعدة البيانات: BASE
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