Ruben P A van Eijk1,2, Tessa Kliest1, Leonard H van den Berg1. 1. Department of Neurology, UMC Utrecht Brain Centre. 2. Biostatistics & Research Support, Julius Centre for Health Sciences and Primary Care, University Medical Centre Utrecht, Utrecht, The Netherlands.
Abstract
PURPOSE OF REVIEW: To review the current developments in the design and conduct of clinical trials for amyotrophic lateral sclerosis (ALS), illustrated by a critical appraisal of ClinicalTrials.gov. RECENT FINDINGS: In total, 63 clinical trials were included in the analysis, of which 13 phase 1, 35 phase 2 and 15 phase 3. Virtually all phase 3 clinical trials can be classified as randomized, placebo controlled, whereas this is only true for 57% of the phase 2 clinical trials. There are promising developments in the routes of drug administration, eligibility criteria, efficacy endpoints and overall trial design. Some of these innovative approaches may, however, not fulfil clinical trial guidelines or regulatory requirements. This could delay the development of effective therapy or hamper our ability to determine whether a treatment is truly (in)effective. The initiation of trial consortia comprising patient organizations, academia, industry and funding bodies may significantly strengthen the future clinical trial landscape for ALS. SUMMARY: The ALS clinical trial landscape is currently highly active with several promising innovative developments and therapeutic options. By further refinement of evidence-based guidelines, and alignment of our current endeavours, we may soon be able to positively impact the lives of people living with ALS.
PURPOSE OF REVIEW: To review the current developments in the design and conduct of clinical trials for amyotrophic lateral sclerosis (ALS), illustrated by a critical appraisal of ClinicalTrials.gov. RECENT FINDINGS: In total, 63 clinical trials were included in the analysis, of which 13 phase 1, 35 phase 2 and 15 phase 3. Virtually all phase 3 clinical trials can be classified as randomized, placebo controlled, whereas this is only true for 57% of the phase 2 clinical trials. There are promising developments in the routes of drug administration, eligibility criteria, efficacy endpoints and overall trial design. Some of these innovative approaches may, however, not fulfil clinical trial guidelines or regulatory requirements. This could delay the development of effective therapy or hamper our ability to determine whether a treatment is truly (in)effective. The initiation of trial consortia comprising patient organizations, academia, industry and funding bodies may significantly strengthen the future clinical trial landscape for ALS. SUMMARY: The ALS clinical trial landscape is currently highly active with several promising innovative developments and therapeutic options. By further refinement of evidence-based guidelines, and alignment of our current endeavours, we may soon be able to positively impact the lives of people living with ALS.
Authors: Ruben P A van Eijk; Kit C B Roes; Inez de Greef-van der Sandt; Leonard H van den Berg; Ying Lu Journal: Clin Pharmacol Ther Date: 2022-02-17 Impact factor: 6.903