Ignite Creation Date:
2025-12-24 @ 7:23 PM
Ignite Modification Date:
2025-12-29 @ 10:57 PM
Study NCT ID:
NCT03272503
Status:
UNKNOWN
Last Update Posted:
2020-05-28
First Post:
2017-08-31
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS)
Sponsor:
University of Calgary
Organization:
Study Overview
Official Title:
A Phase II Randomized, Placebo-Controlled, Double Blinded, Multi-Centre Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis
Status:
UNKNOWN
Status Verified Date:
2020-05
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
Pimozide2
Brief Summary:
This study will look at whether Pimozide may help to slow the progression of Amyotrophic Lateral Sclerosis.
100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo.
Participants will be on study medication up to 22 weeks, and on study up to 26 weeks. There are 8 clinic visits and 1 phone visit over the course of the Treatment Phase of the study. The second phase which is Observational, is optional with follow-up for up to 5 years from the end of the Treatment Phase.
100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo.
Participants will be on study medication up to 22 weeks, and on study up to 26 weeks. There are 8 clinic visits and 1 phone visit over the course of the Treatment Phase of the study. The second phase which is Observational, is optional with follow-up for up to 5 years from the end of the Treatment Phase.
Detailed Description:
Amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease 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. Rilutek® (riluzole) has been approved as a treatment to slow progression of ALS, but is minimally effective with mean increase in survival of only a few months. Radicava® or Radicut® (edaravone) has recently been approved in Canada, USA, Japan and South Korea.
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.
There are two parts to this study.
Treatment Phase: In the first part of this study, 100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo.
Each Pimozide tablet contains 2 mg of Pimozide. The matching placebo tablets for this study will look exactly like the Pimozide tablets. Placebos are used in clinical trials to find out if the results observed in the study are due the drug being tested, or for other reasons.
Neither the subject nor their doctor will know which group a patient belongs to. However, if an emergency should arise, information about a treatment group will be shared with their doctor to ensure appropriate medical care. Participants will take their treatment once a day, every day for about 22 weeks. The total time in the study from the screen visit up until the last phone call communication is about 26 weeks.
Observational Phase: The second part of this study is optional. It is each subject's decision whether to participate only in the first part of this study, or in both parts of the study, or not at all. In the second part of this study, the Canadian Neuromuscular Disease Registry (CNDR) will collect data on overall ALS progression using the Revised ALS Functional Rating Scale (ALSFRS-R) and breathing using Vital Capacity data collected during breathing tests. This information will be collected from a subject medical record following each routine clinical appointment. Data will be collected at each routine clinic visit for up to 5 years from the end of the first part of the study. There will be no extra visits for this part of the study beyond routine ALS clinic visits.
The information collected during this part of the study will be used to compare the progression of ALS, after the clinical trial is completed, among the two treatment types (Placebo or 2 mg per day). By analyzing this information, the researchers conducting this study hope to determine if Pimozide may help to slow the progression of ALS. To participate in this part of the study consent must be provided to join the CNDR. A subject who hasn't already provided their CNDR consent, and wishes to participate in this part of the study will be given a CNDR consent form to review and sign in addition to this consent form. A subject already participating in the CNDR will just need to sign the main study consent form.
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.
There are two parts to this study.
Treatment Phase: In the first part of this study, 100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo.
Each Pimozide tablet contains 2 mg of Pimozide. The matching placebo tablets for this study will look exactly like the Pimozide tablets. Placebos are used in clinical trials to find out if the results observed in the study are due the drug being tested, or for other reasons.
Neither the subject nor their doctor will know which group a patient belongs to. However, if an emergency should arise, information about a treatment group will be shared with their doctor to ensure appropriate medical care. Participants will take their treatment once a day, every day for about 22 weeks. The total time in the study from the screen visit up until the last phone call communication is about 26 weeks.
Observational Phase: The second part of this study is optional. It is each subject's decision whether to participate only in the first part of this study, or in both parts of the study, or not at all. In the second part of this study, the Canadian Neuromuscular Disease Registry (CNDR) will collect data on overall ALS progression using the Revised ALS Functional Rating Scale (ALSFRS-R) and breathing using Vital Capacity data collected during breathing tests. This information will be collected from a subject medical record following each routine clinical appointment. Data will be collected at each routine clinic visit for up to 5 years from the end of the first part of the study. There will be no extra visits for this part of the study beyond routine ALS clinic visits.
The information collected during this part of the study will be used to compare the progression of ALS, after the clinical trial is completed, among the two treatment types (Placebo or 2 mg per day). By analyzing this information, the researchers conducting this study hope to determine if Pimozide may help to slow the progression of ALS. To participate in this part of the study consent must be provided to join the CNDR. A subject who hasn't already provided their CNDR consent, and wishes to participate in this part of the study will be given a CNDR consent form to review and sign in addition to this consent form. A subject already participating in the CNDR will just need to sign the main study consent form.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: