Clinical Research Alert: Phase 1 Study of AMX0114 in Adults with ALS

Researchers at Amylyx Pharmaceuticals, Inc. are seeking adults with amyotrophic lateral sclerosis (ALS) to participate in a phase 1 clinical trial (LUMINA) to evaluate the safety and efficacy of the investigational therapy AMX0114 to treat ALS. AMX0114 is designed to reduce the levels of calpain-2, an enzyme linked to the degeneration and death of neurons in people living with sporadic ALS, with the goal of potentially slowing progression of the disease. This study will examine how safe and tolerable AMX0114 is as a treatment for people with ALS.

The study

This is a placebo-controlled study that will examine at least four dose levels of AMX0114, sequentially, in approximately 48 participants. The participants will be divided into four cohorts made up of 12 participants. In each cohort, nine participants will receive active AMX0114 and three participants will receive an inactive placebo control. For all participants, the study will consist of three periods including a Screening Period, a Treatment Period, and a Safety Follow-up Period. During the Screening Period, participants can expect to come to the clinic between one and three times. During the Treatment Period, participants can expect to come to the clinic at least 10 times and be called on the phone at least eight times. During the Safety Follow-up Period, participants can expect to come to the clinic at least once and be called at least once. The total duration of the study will be 25 weeks.

AMX0014 will be administered intrathecally through a lumbar puncture. Participants will undergo a total of five lumbar punctures throughout the study. The effects will be evaluated using a number of tests and procedures including but not limited to: examination of adverse events, lumbar puncture to collect cerebrospinal fluid (CSF) samples, blood tests to collect blood biomarker and pharmacokinetics samples, collection of blood and urine samples, assessments of ALS progression using the ALS Functional Rating Scale – Revised (ALSFRS-R) and the slow vital capacity test (SVC), and additional physiological and neurological exams, assessments, and questionnaires.

Study criteria

To be eligible for this research study, individuals must meet the following inclusion criteria:

• Ability to understand the purpose and risks of this study and willingness to comply with the study and to provide informed consent in accordance with local laws and regulations
• Male or female, at least 18 years of age.
• Diagnosis of clinically definite or clinically probable ALS
• Time since onset of first symptom of ALS should be <24 months prior to beginning the study
• If treated with riluzole and/or edaravone before or during the trial, must be on a stable regimen for at least 30 days prior to starting the study and through the end of the study
• No plans for pregnancy or sperm donation during the trial for up to 90 days after the last dose

Individuals may not be eligible to participate if they are affected by another condition or receiving another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

Travel support will be available for eligible participants.

To learn more about the study or inquire about participation, contact the Amylyx Medical Director at clinicaltrials@amylyx.com or visit our trial listing at https://clinicaltrials.gov/study/NCT06665165.