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A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04516096
Expanded Access Status : Temporarily not available
First Posted : August 18, 2020
Last Update Posted : August 18, 2020
Information provided by (Responsible Party):
Amylyx Pharmaceuticals Inc.

Brief Summary:
The protocol is intended is to provide extended access to AMX0035 for the treatment of patients who previously completed evaluation in a study of AMX0035 for ALS.

Condition or disease Intervention/treatment
Amyotrophic Lateral Sclerosis Drug: AMX0035

Detailed Description:
In this open-label, compassionate extended use study, patients who completed monitoring in an Amylyx sponsored protocol for ALS may be offered compassionate use extension of treatment with AMX0035 under this protocol. Patient currently treated with AMX0035 will continue to receive treatment at their current dose/regimen. Patients who have interrupted AMX0035 for more than 28 days will resume AMX0035 treatment with 1 sachet (oral [or feeding tube]) once a day in the morning to be escalated to twice a day (morning and evening) after approximately 2 weeks on the basis of GI tolerance. Enrolled patients will complete regular outpatient visit approximately every 16 weeks (± 2 weeks). Completion of the ALS functional rating scale (ALSFRS-R) will be optional.

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: AMX0035 for Amyotrophic Lateral Sclerosis - Compassionate Use Protocol

Intervention Details:
  • Drug: AMX0035
    Tauroursodeoxycholic Acid and Sodium Phenylbutyrate orally given twice a day

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

  • Any patient who has completed follow-up in an Amylyx sponsored trial of AMX0035 for the treatment of ALS will be eligible to enroll into this Protocol
  • Capable of providing informed consent
  • Capable and willing to follow trial procedures
  • Capable and willing of travelling to the site at regular intervals for interim site visits and to return and collect study drug or able to attend telemedicine remote site visits if such are currently in use at the site
  • Participants who have established care with a neurologist at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the protocol.
  • Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug.
  • Women must not be nursing, be pregnant or planning to become pregnant for the duration of the study and 3 months after last dose of study drug
  • Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide sperm for donation for the duration of the study and 3 months after last dose of study drug

Exclusion Criteria:

  • Ongoing severe adverse events that in the opinion of the Site Investigator are contraindication to the study drug, including severe renal or liver insufficiency or Class III/IV heart failure (per New York Heart Association)
  • Ongoing serious adverse event that was assessed as related to study drug per the Site Investigator
  • Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the patient to provide informed consent, according to Site Investigator judgment;
  • Current severe biliary disease which may result in the investigator medical judgement in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder;
  • Clinically significant unstable medical condition (other than ALS) that would pose a risk to the patient if they were to participate in the study, according to Site Investigator judgment;
  • Treatment, current or within 90 days from screening with any cell therapies or gene therapies;
  • Implantation of Diaphragm Pacing System (DPS);
  • Current or planned exposure to any prohibited medications listed in Section

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04516096

Sponsors and Collaborators
Amylyx Pharmaceuticals Inc.
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Study Director: Machelle Manual, PhD Amylyx Pharmaceuticals Inc.
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Responsible Party: Amylyx Pharmaceuticals Inc. Identifier: NCT04516096    
Other Study ID Numbers: A35-003
First Posted: August 18, 2020    Key Record Dates
Last Update Posted: August 18, 2020
Last Verified: August 2020
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases