Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease (PEGASUS)
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ClinicalTrials.gov Identifier: NCT03533257 |
Recruitment Status :
Active, not recruiting
First Posted : May 23, 2018
Last Update Posted : April 20, 2020
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Sponsor:
Amylyx Pharmaceuticals Inc.
Collaborators:
Alzheimer's Drug Discovery Foundation
Alzheimer's Association
Information provided by (Responsible Party):
Amylyx Pharmaceuticals Inc.
Tracking Information | |||||
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First Submitted Date ICMJE | April 30, 2018 | ||||
First Posted Date ICMJE | May 23, 2018 | ||||
Last Update Posted Date | April 20, 2020 | ||||
Actual Study Start Date ICMJE | August 27, 2018 | ||||
Estimated Primary Completion Date | October 1, 2020 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Quantity of Adverse Events Observed in the Study [ Time Frame: 6 Months ] Rate of Adverse Events between placebo and Active Groups
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures | Same as current | ||||
Descriptive Information | |||||
Brief Title ICMJE | Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease | ||||
Official Title ICMJE | Phase II Study to Assess the Safety, Tolerability, and Target Engagement of AMX0035, a Fixed Combination of Sodium Phenylbutyrate and Tauroursodeoxycholic Acid for the Treatment of Alzheimer's Disease | ||||
Brief Summary | The proposed study will be a 24-week, randomized, double-blind, multi-site, placebo-controlled study in volunteers with late mild cognitive impairment (MCI) or early dementia due to Alzheimer's disease (AD). | ||||
Detailed Description | The study is a 24-week, randomized, double-blind, multi-site, placebo-controlled study in volunteers with late mild cognitive impairment (MCI) or early dementia due to Alzheimer's disease (AD). The study is designed to evaluate the safety, tolerability, drug target engagement and neurobiological effects of treatment with AMX0035 over 24 weeks. The study is designed to yield deep phenotyping insight for the purposes of demonstrating the effects of AMX0035 on mechanistic targets of engagement and disease biology. The study will evaluate diverse disease-relevant markers and produce an informative dataset that will allow for evaluation and correlation of imaging-based markers, neurobiological changes, functional measures, and cognitive outcomes. | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 2 | ||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Intervention Model Description: Placebo-Controlled, Double-Blind, Parallel-Group Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Primary Purpose: Treatment |
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Condition ICMJE | Alzheimer Disease | ||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Estimated Enrollment ICMJE |
100 | ||||
Original Estimated Enrollment ICMJE |
50 | ||||
Estimated Study Completion Date ICMJE | December 1, 2020 | ||||
Estimated Primary Completion Date | October 1, 2020 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 55 Years to 89 Years (Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT03533257 | ||||
Other Study ID Numbers ICMJE | AMX-8000 | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Amylyx Pharmaceuticals Inc. | ||||
Study Sponsor ICMJE | Amylyx Pharmaceuticals Inc. | ||||
Collaborators ICMJE |
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Investigators ICMJE | Not Provided | ||||
PRS Account | Amylyx Pharmaceuticals Inc. | ||||
Verification Date | January 2020 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |