Extension Study Evaluating The Safety And Tolerability of AMX0035
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ClinicalTrials.gov Identifier: NCT05619783 |
Recruitment Status :
Enrolling by invitation
First Posted : November 17, 2022
Last Update Posted : January 5, 2024
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Sponsor:
Amylyx Pharmaceuticals Inc.
Information provided by (Responsible Party):
Amylyx Pharmaceuticals Inc.
Tracking Information | |||||
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First Submitted Date ICMJE | October 27, 2022 | ||||
First Posted Date ICMJE | November 17, 2022 | ||||
Last Update Posted Date | January 5, 2024 | ||||
Actual Study Start Date ICMJE | December 29, 2022 | ||||
Estimated Primary Completion Date | March 2026 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
To assess the Incidence of Treatment-Emergent Adverse Events during treatment with AMX0035 [ Time Frame: 108 weeks ] Incidence of all adverse events (AE)s; AEs leading to treatment discontinuation or study withdrawal, and all serious adverse events (SAE)s in participants treated with AMX0035
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
To assess the impact of long-term treatment with AMX0035 on survival [ Time Frame: 108 weeks ]
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Original Secondary Outcome Measures ICMJE | Same as current | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Extension Study Evaluating The Safety And Tolerability of AMX0035 | ||||
Official Title ICMJE | A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants With Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX) | ||||
Brief Summary | The primary objective is to evaluate the safety and tolerability of AMX0035 over 108 weeks of open label treatment for participants previously enrolled in Study A35-004 (PHOENIX). | ||||
Detailed Description | All participants will receive open-label treatment with AMX0035, starting on Day 1 with twice a day oral dosing (once in the morning and once in the evening) for the duration of the study. After the Baseline Visit (Day 1), enrolled participants will complete visits approximately every 12 weeks (± 2 weeks), until Week 108 or the end of treatment (EOT) visit, followed by a safety follow-up approximately 28 days after the last dose. A survival follow-up assessment will be completed every 12 weeks following the EOT visit until time of death or end of study (EOS). | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 3 | ||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Amyotrophic Lateral Sclerosis | ||||
Intervention ICMJE | Drug: AMX0035
Combination of 3 g phenylbutyrate and 1 g taurursodiol
Other Name: RELYVRIO
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Study Arms ICMJE | Experimental: Active
All participants will be treated with oral (or feeding tube) AMX0035 (a fixed-dose combination of Sodium Phenylbutyrate (PB) and taurursodiol). All participants will take 2 sachets daily (one morning dose and one evening dose) starting on Day 1, for the duration of the study (if twice a day dosing is poorly tolerated, dosing interruptions and reductions are further discussed in section 6.3) AMX0035 will be supplied by Amylyx as a carton box containing approximately 1 month supply of single use sachets. Each AMX0035 sachet contains active ingredients in a powder formulation with 3 g PB and 1 g taurursodiol. AMX0035 powder is mixed with water and taken orally (or via feeding tube).
Intervention: Drug: AMX0035
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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 | Enrolling by invitation | ||||
Estimated Enrollment ICMJE |
600 | ||||
Original Estimated Enrollment ICMJE | Same as current | ||||
Estimated Study Completion Date ICMJE | August 2026 | ||||
Estimated Primary Completion Date | March 2026 (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 | 18 Years and older (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 | Belgium, France, Germany, Ireland, Italy, Netherlands, Poland, Portugal, Spain, Sweden, United Kingdom | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT05619783 | ||||
Other Study ID Numbers ICMJE | A35-011 | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | Amylyx Pharmaceuticals Inc. | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Amylyx Pharmaceuticals Inc. | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Amylyx Pharmaceuticals Inc. | ||||
Verification Date | January 2024 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |