Amyotrophic Lateral Sclerosis (ALS) Clinical Trial
Official title:
A Registry-Based Randomized-Controlled, Double-Blinded Clinical Trial of Pimozide in Patients With Neuromuscular Junction Transmission Dysfunction Due to Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a neuromuscular disease that results in rapid decline
in normal muscle function and tone leading to difficulties with mobility, eating, drinking,
breathing, sleeping, and communicating. The disease is progressive and no cure currently
exists. Most people diagnosed with ALS succumb within 3 to 5 years. The only approved
treatment to slow the progression of ALS is called Rilutek® (riluzole) which has only a
modest effect and has been shown to increase survival by a few months.
Muscular dysfunction present in people with ALS is caused by nerve breakdown and a
dysfunction in the communication between the muscles and the nerves. The area where these
communications occur is called the neuromuscular junction. Some recent studies have focused
on using different medications to enhance communication at the neuromuscular junction with
the goal of improving muscle function as a result. This approach is unproven but may help to
slow the progression of the disease.
Pimozide is a medication that has been demonstrated to enhance communication at the
neuromuscular junction in fish and mice. This study will look at whether Pimozide may help
to slow the progression of ALS and how much medication needs to be taken to have an effect.
This clinical trial has two components: an acute therapy component consisting of a Phase II
placebo-controlled, double-blinded, randomized-controlled pilot study of pimozide for the
treatment of ALS; and a second component featuring a longitudinal follow-up study on ALS
progression and outcomes. This clinical trial is registry-based including subject
recruitment facilitated by the Canadian Neuromuscular Disease Registry (CNDR; National
Principal Investigator: L. Korngut), and longitudinal follow-up data collection will occur
during the second component of this clinical trial through the CNDR.
The acute therapy study duration for each subject is around 11 weeks. The follow up study
duration through the CNDR is up to 5 years.
Number of study participants:25
Randomization: Subjects will be block randomized with a block size of five subjects. Within
each block one subject will be randomly assigned to placebo with the remaining four subjects
randomized to the treatment groups. Study physicians will be blinded to patient
randomization status. Randomization will occur with a 4:1 ratio of study drug (20 subjects)
to placebo (5 subjects). After administration of maximum dose for 45-50 days, subjects will
taper the allocated treatment or placebo. Randomization will occur via permuted block
randomization and study personnel will be blinded to the randomization at all times allowing
full concealment.
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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