Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

被引：65

作者：

Paganoni, Sabrina ^{[1
,2
,3
]}

Berry, James D. ^{[1
,2
]}

Quintana, Melanie ^{[4
]}

Macklin, Eric ^{[5
]}

Saville, Benjamin R. ^{[4
,6
]}

Detry, Michelle A. ^{[4
]}

Chase, Marianne ^{[1
,2
]}

Sherman, Alexander, V ^{[1
,2
]}

Yu, Hong ^{[1
,2
]}

Drake, Kristin ^{[1
,2
]}

Andrews, Jinsy ^{[7
]}

Shefner, Jeremy ^{[8
]}

Chibnik, Lori B. ^{[5
]}

Vestrucci, Matteo ^{[4
]}

Cudkowicz, Merit E. ^{[1
,2
]}

机构：

[1] Harvard Med Sch, Sean M Healey & AMG Ctr ALS, Massachusetts Gen Hosp, 165 Cambridge St,Suite 600, Boston, MA 02114 USA

[2] Harvard Med Sch, Neurol Clin Res Inst, Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USA

[3] Harvard Med Sch, Spaulding Rehabil Hosp, Boston, MA 02114 USA

[4] Berry Consultants, Austin, TX USA

[5] Harvard Med Sch, Massachusetts Gen Hosp, Biostat Ctr, Boston, MA 02114 USA

[6] Vanderbilt Univ, Sch Med, Dept Biostat, Nashville, TN 37212 USA

[7] Columbia Univ, New York, NY USA

[8] Barrow Neurol Inst, Phoenix, AZ 85013 USA

来源：

ANNALS OF NEUROLOGY | 2022年 / 91卷 / 02期

关键词：

CLINICAL-TRIALS; ALS; METAANALYSIS; DESIGN;

D O I：

10.1002/ana.26285

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints.

引用

页码：165 / 175

页数：11

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