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

• Register: ClinicalTrials.gov
• Last refreshed on: 17 April 2023
• Main ID: NCT04516096
• Date of registration: 09/08/2020
• Prospective Registration: Yes
• Primary sponsor: Amylyx Pharmaceuticals Inc.
• Public title: A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: AMX0035 for Amyotrophic Lateral Sclerosis - Compassionate Use Protocol
• Date of first enrolment: November 22, 2020
• Target sample size: 28
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04516096
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

United States

Contacts

• Name: Machelle Manual, PhD
• Affiliation: Amylyx Pharmaceuticals Inc.

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: AMX0035

Primary Outcome(s)

Treatment emergent Adverse Events [Time Frame: Through study completion an average of 1 year]

Secondary ID(s)

A35-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors