Lead Investigator: Masahiro Takeuchi, The University of Tokyo
Title of Proposal Research: Repositioning of neurological drug Aripiprazole for Amyotrophic lateral sclerosis (ALS) patients
Vivli Data Request: 8581
Funding Source: None
Potential Conflicts of Interest: None
Summary of the Proposed Research:
Amyotrophic lateral sclerosis (ALS) is a serious disease affecting the nerve cells in the spinal cord that control muscle movement, resulting in a reduction in size and strength of muscles. ALS is designated as an intractable disease by Ministry of Health, Labor and Welfare (MHLW). “The criteria for designated intractable diseases are as follows: (1) rarity (affecting less than 0.1% of the population in Japan), (2) unknown etiology (cause), (3) lack of effective treatment, (4) necessity of long-term treatment, and (5) existence of objective diagnostic criteria (i.e., there are specific measures for recognizing and monitoring the disease)”. Globally, it is reported that there are 400,000 ALS patients and 5600 patients are newly diagnosed each year.
ALS is an incurable disease and patients have limited medical therapies. Currently there are only two medical therapies available, Riluzole and Radicavatm.
Phase III clinical trials indicated that survival, time to death and time to progression was longer for Riluzole compared to placebo. For Radicavatm, quality of life was shown to be better using the ALS Functional Rating scale revised (ALSFRS-R) compared to placebo.
Aripiprazole is a medication used to treat some mental health conditions. Phase III trials testing whether Aripiprazole could be a potential medication for treating Alzheimer’s disease did not show statistically significant results.
The primary endpoints in the ALS trials using Radicavatm and the Alzheimer’s trials using Aripiprazole measured similar quality of life endpoints.
The aim of the research is to analyze the quality of life endpoints in both Radicavatm and Aripiprazole clinical trials and to postulate that the results are similar in Alzheimer and ALS patients. This may further indicate that Aripiprazole has an extended indication to ALS patients although it did not show efficacy in Alzheimer patients.
The purpose of this research is to re-analyze the data using all the patient data without imputation. In addition, analysis of the data using appropriate models such as logistic regression for each of the endpoints is necessary. If the analyses of the two endpoints prompts positive results, repositioning of Aripiprazole as a medical therapy for ALS may be considered.
Requested Studies:
CN138-006 Aripiprazole in the Treatment of Patients With Psychosis Associated With Dementia of Alzheimer’s Type
Data Contributor: Otsuka Pharmaceuticals
Study ID: NCT01438060
CN138-004 – Study of Aripiprazole in the Treatment of Patients With Psychosis Associated With Dementia of the Alzheimer’s Type
Data Contributor: Otsuka Pharmaceuticals
Study ID: NCT00036114
CN138-005 – Study of Aripiprazole in the Treatment of Patients With Psychosis Associated With Dementia of the Alzheimer’s Type
Data Contributor: Otsuka Pharmaceuticals
Study ID: NCT00041678