scholarly journals ALS (Amyotrophic Lateral Sclerosis) Patient Recruitment for Clinical Trials and Epidemiological Studies: National ALS Registry’s Research Notification Mechanism (Preprint)

10.2196/28021 ◽  
2021 ◽  
Author(s):  
Paul Mehta ◽  
Jaime Raymond ◽  
Moon Kwon Han ◽  
Theodore Larson ◽  
James D Berry ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Amyotrophic Lateral Sclerosis Patient ◽  
Epidemiological Studies ◽  
Patient Recruitment ◽  
Lateral Sclerosis

scholarly journals A systematic review of non-motor symptom evaluation in clinical trials for amyotrophic lateral sclerosis

2021 ◽  
Author(s):  
Emily Beswick ◽  
Deborah Forbes ◽  
Zack Hassan ◽  
Charis Wong ◽  
Judith Newton ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Sleep Disturbances ◽  
Neuropsychiatric Symptoms ◽  
Behavioural Change ◽  
Motor Symptom ◽  
Motor Symptoms ◽  
Behavioural Changes ◽  
Non Motor Symptoms ◽  
Lateral Sclerosis

Abstract Background Amyotrophic lateral sclerosis (ALS) is increasingly recognised as a multi-system disorder, presenting with common and impactful non-motor symptoms, such as neuropsychiatric symtpoms, cognitive and behavioural changes, pain, disordered sleep, fatigue and problematic saliva. Aim/hypothesis We aimed to systematically review 25 years of ALS clinical trials data to identify if non-motor features were evaluated, in addition to the traditional measures of motor functioning and survival, and where evaluated to describe the instruments used to assess. We hypothesised that assessment of non-motor symptoms has been largely neglected in trial design and not evaluated with ALS-suitable instruments. Methods We reviewed clinical trials of investigative medicinal products in ALS, since the licensing of riluzole in 1994. Trial registry databases including WHO International Trials Registry, European Clinical Trials Register, clinicaltrials.gov, and PubMed were systematically searched for Phase II, III or IV trials registered, completed or published between 01/01/1994 and 16/09/2020. No language restrictions were applied. Results 237 clinical trials, including over 29,222 participants, were investigated for their use of non-motor outcome measures. These trials evaluated neuropsychiatric symptoms (75, 32%), cognitive impairment (16, 6.8%), behavioural change (34, 14%), pain (55, 23%), sleep disturbances (12, 5%) and fatigue (18, 8%). Problematic saliva was assessed as part of composite ALS-FRS(R) scores in 184 trials (78%) but with no focus on this as an isolated symptom. 31 (13%) trials including 3585 participants did not include any assessment of non-motor symptoms. Conclusions Non-motor symptoms such as neuropsychiatric, cognitive and behavioural changes, pain, disordered sleep, fatigue, and problematic saliva have not been consistently evaluated in trials for people with ALS. Where evaluated, non-symptoms were primarily assessed using instruments and impairment thresholds that are not adapted for people with ALS. Future trials should include non-motor symptom assessments to evaluate the additional potential therapeutic benefit of candidate drugs. PROPSERO registration CRD42020223648.

scholarly journals Japanese amyotrophic lateral sclerosis patient with learning disabilities with a deletion mutation in the C-terminal of theFUS/TLSgene

2015 ◽  
Vol 3 (5) ◽  
pp. 192-193 ◽  
Author(s):  
Akiko Onohara ◽  
Kishin Koh ◽  
Takamura Nagasaka ◽  
Kazumasa Shindo ◽  
Masaaki Kato ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Learning Disabilities ◽  
Amyotrophic Lateral Sclerosis Patient ◽  
Deletion Mutation ◽  
Lateral Sclerosis

A novel SOD1 mutation in a young amyotrophic lateral sclerosis patient with a very slowly progressive clinical course

2010 ◽  
Vol 42 (4) ◽  
pp. 596-597 ◽  
Author(s):  
Eleni Georgoulopoulou ◽  
Cinzia Gellera ◽  
Cinzia Bragato ◽  
Patrizia Sola ◽  
Annalisa Chiari ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Clinical Course ◽  
Amyotrophic Lateral Sclerosis Patient ◽  
Sod1 Mutation ◽  
Lateral Sclerosis

Current clinical trials in amyotrophic lateral sclerosis

2007 ◽  
Vol 16 (8) ◽  
pp. 1197-1207 ◽  
Author(s):  
Jaydeep M Bhatt ◽  
Paul H Gordon
Keyword(s):  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Lateral Sclerosis

scholarly journals Urate levels predict survival in amyotrophic lateral sclerosis: Analysis of the expanded Pooled Resource Open-Access ALS clinical trials database

2017 ◽  
Vol 57 (3) ◽  
pp. 430-434 ◽  
Author(s):  
Sabrina Paganoni ◽  
Katharine Nicholson ◽  
James Chan ◽  
Amy Shui ◽  
David Schoenfeld ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Open Access ◽  
Lateral Sclerosis

scholarly journals Amyotrophic lateral sclerosis patient iPSC-derived astrocytes impair autophagy via non-cell autonomous mechanisms

2017 ◽  
Vol 10 (1) ◽  
Author(s):  
Martin Madill ◽  
Katya McDonagh ◽  
Jun Ma ◽  
Alice Vajda ◽  
Paul McLoughlin ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Amyotrophic Lateral Sclerosis Patient ◽  
Lateral Sclerosis

scholarly journals Edaravone in Amyotrophic Lateral Sclerosis’Lessons from the Clinical Development Program and the Importance of a Strategic Clinical Trial Design

US Neurology ◽  
2018 ◽  
Vol 14 (1) ◽  
pp. 47 ◽  
Author(s):  
Said R Beydoun ◽  
Jeffrey Rosenfeld
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Trial Design ◽  
Development Program ◽  
Clinical Trial Design ◽  
Clinical Development ◽  
Disease Heterogeneity ◽  
Clinical Development Program ◽  
Lateral Sclerosis

Edaravone significantly slows progression of amyotrophic lateral sclerosis (ALS), and is the first therapy to receive approval by the Food and Drug Administration (FDA) for the disease in 22 years. Approval of edaravone has marked a new chapter in pharmaceutical development since the key trial included a novel strategic clinical design involving cohort enrichment. In addition, approval was based on clinical trials that had a relatively small patient number and were performed outside of the US. Edaravone was developed through a series of clinical trials in Japan where it was determined that a well-defined subgroup of patients was required to reveal a treatment effect within the study period. Amyotrophic lateral sclerosis is associated with wide-ranging disease heterogeneity (both within the spectrum of ALS phenotypes as well as in the rate of progression). The patient cohort enrichment strategy aimed to address this heterogeneity and should now be considered as a viable, and perhaps preferred, trial design for future studies. Future research incorporating relevant biomarkers may help to better elucidate edaravone’s mechanism of action, pharmacodynamics, and subsequently ALS phenotypes that may preferentially benefit from treatment. In this review, we discuss the edaravone clinical development program, outline the strategic clinical trial design, and highlight important lessons for future trials.

Plant-Derived Natural Compounds for the treatment of Amyotrophic Lateral Sclerosis: An Update

2021 ◽  
Vol 19 ◽  
Author(s):  
Roohi Mohi-ud-din ◽  
Reyaz Hassan Mir ◽  
Abdul Jalil Shah ◽  
Saba Sabreen ◽  
Taha Umair Wani ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Adverse Effects ◽  
Therapeutic Strategies ◽  
Structure Activity Relationships ◽  
Complex Genetics ◽  
Natural Agents ◽  
Structure Activity ◽  
Preclinical And Clinical Studies ◽  
Lateral Sclerosis

Background: Amyotrophic lateral sclerosis (ALS) is a motor neuron disease (MND) that typically causes death within 3-5 years after diagnosis. Regardless of the substantial scientific knowledge accrued from more than a century ago, truly effective therapeutic strategies remain distant. Various conventional drugs are being used but are having several adverse effects. Objective/Aim: The current study aims to thoroughly review plant-derived compounds with well-defined ALS activities and their structure-activity relationships. Moreover, the review also focuses on complex genetics, clinical trials, and the use of natural products that might decrypt the future and novel therapeutics in ALS. Methods: The collection of data for the compilation of this review work was searched in PubMed Scopus, Google Scholar, and Science Direct. Results: Results showed that phytochemicals like-Ginkgolides, Protopanaxatriol, Genistein, epigallocatechingallate, resveratrol, cassoside, and others possess Amyotrophic lateral sclerosis (ALS) activity by various mechanisms. Conclusion: These plant-derived compounds may be considered as supplements to conventional (ALS). Moreover, further preclinical and clinical studies are required to understand the structure-activity relationships, metabolism, absorption, and mechanisms of plant-derived natural agents.

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