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

NCT04516096 | Completed Phase 2 FDA Drug

• 1 other identifier: A35-003
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 5

Brief Summary

The protocol is intended to provide extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for ALS.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Treatment emergent Adverse Events

  Rate of treatment emergent adverse events during AMX0035 therapy

  Through study completion an average of 1 year

Study Arms (1)

AMX-0035 long term treatment extension

EXPERIMENTAL

AMX0035 administered twice daily p.o.

Drug: AMX0035

Interventions

AMX0035 DRUG

Tauroursodeoxycholic Acid and Sodium Phenylbutyrate orally given twice a day

AMX-0035 long term treatment extension

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amylyx Pharmaceuticals Inc.: lead

Study Sites (5)

Forbes Norris MDA/ALS Research Center - California Pacific Medical Center

San Francisco, California, 94114, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Massachusetts Memorial Medical Center

Worcester, Massachusetts, 01655, United States

Location

University of Michigan Medical Center

Ann Arbor, Michigan, 48109, United States

Location

Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

sodium phenylbutyrate and taurursodiol

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Machelle Manual, PhD

  Amylyx Pharmaceuticals Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open label long term extension of treatment

Study Record Dates

• First Submitted: August 9, 2020
• First Posted: August 18, 2020
• Study Start: November 22, 2020
• Primary Completion: December 30, 2022
• Study Completion: January 31, 2023
• Last Updated: April 7, 2023

Record last verified: 2022-10

Locations

US (5)