RECRUITING

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

Conditions

ALS Amyotrophic Lateral Sclerosis Sporadic ALS Motor Neuron Disease Antisense oligonucleotide ASO Calpain-2

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).

Official Title

Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Evaluate Safety, Tolerability, PK and PD of Antisense Oligonucleotide AMX0114 Administered to Adult Participants With Amyotrophic Lateral Sclerosis

Quick Facts

Study Start:2025-04-07

Study Completion:2027-10

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06665165

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Ability to understand the purpose and risks of this study, willingness to comply with the study and to provide informed consent in accordance with local laws and regulations. 2. Male or female, at least 18 years of age. 3. Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS. 4. Time since onset of first symptom of ALS should be \<24 months prior to beginning the study. Date of ALS symptom onset is defined as the onset of weakness (in the limbs, bulbar region, or trunk). 5. If the participant is to be treated with riluzole and/or edaravone before or during the trial, then treatment must be previously started and maintained at a stable regimen for at least 30 days prior to starting the study and through the end of the study. 6. Women of childbearing potential (e.g., not post-menopausal for at least one year or surgically sterile) must agree to use an acceptable birth control method for the duration of the trial and 60 days after the last dose of Study Drug or be of non-childbearing potential. 7. Female participants or female partners of male participants must not be pregnant or plan to become pregnant for the duration of the trial and for up to 90 days after the last dose of Study Drug. 8. Male participants must agree to abstain from sperm donation for the duration of the trial and practice contraception with a female partner, for at least 90 days after last dose of Study Drug.	1. Presence of tracheostomy or permanent assisted ventilation. 2. SVC less than 75%. 3. Abnormal liver function defined as aspartate aminotransferase and/or alanine aminotransferase \> 3 times the upper limit of normal (ULN) and/or total bilirubin \> 1.5 times the ULN (obtained within 4 weeks of first dose) except when a result of Gilbert syndrome. 4. Abnormal renal function defined as estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73m2. 5. Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time. 6. Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding. 7. Current or previous clinically significant, unstable medical condition (other than ALS), that in the opinion of the Investigator could affect a participant's safety or ability to comply with the study. 8. Significant abnormalities in physical/neurological examination, vital signs, or electrocardiogram (ECG), which in the opinion of the Investigator could affect the safety of the participant. 9. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that could affect the participant's ability to provide informed consent or comply with study procedures. 10. Current or previous enrollment in another trial involving use of an investigational therapy, in most cases within 30 days after the last dose of the study drug, prior to starting this study. 11. Current or previous treatment with small interfering ribonucleic acid, stem cell therapy, any ASO or gene therapy. 12. Any contraindications for lumbar puncture or repeated intrathecal injection and/or underlying disorders that could be affected by intrathecal injections. 13. Prior severe reaction or known hypersensitivity to any part of the Study Drug.

Inclusion Criteria

Exclusion Criteria

1. Ability to understand the purpose and risks of this study, willingness to comply with the study and to provide informed consent in accordance with local laws and regulations.
2. Male or female, at least 18 years of age.
3. Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS.
4. Time since onset of first symptom of ALS should be \<24 months prior to beginning the study. Date of ALS symptom onset is defined as the onset of weakness (in the limbs, bulbar region, or trunk).
5. If the participant is to be treated with riluzole and/or edaravone before or during the trial, then treatment must be previously started and maintained at a stable regimen for at least 30 days prior to starting the study and through the end of the study.
6. Women of childbearing potential (e.g., not post-menopausal for at least one year or surgically sterile) must agree to use an acceptable birth control method for the duration of the trial and 60 days after the last dose of Study Drug or be of non-childbearing potential.
7. Female participants or female partners of male participants must not be pregnant or plan to become pregnant for the duration of the trial and for up to 90 days after the last dose of Study Drug.
8. Male participants must agree to abstain from sperm donation for the duration of the trial and practice contraception with a female partner, for at least 90 days after last dose of Study Drug.

1. Presence of tracheostomy or permanent assisted ventilation.
2. SVC less than 75%.
3. Abnormal liver function defined as aspartate aminotransferase and/or alanine aminotransferase \> 3 times the upper limit of normal (ULN) and/or total bilirubin \> 1.5 times the ULN (obtained within 4 weeks of first dose) except when a result of Gilbert syndrome.
4. Abnormal renal function defined as estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73m2.
5. Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time.
6. Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding.
7. Current or previous clinically significant, unstable medical condition (other than ALS), that in the opinion of the Investigator could affect a participant's safety or ability to comply with the study.
8. Significant abnormalities in physical/neurological examination, vital signs, or electrocardiogram (ECG), which in the opinion of the Investigator could affect the safety of the participant.
9. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that could affect the participant's ability to provide informed consent or comply with study procedures.
10. Current or previous enrollment in another trial involving use of an investigational therapy, in most cases within 30 days after the last dose of the study drug, prior to starting this study.
11. Current or previous treatment with small interfering ribonucleic acid, stem cell therapy, any ASO or gene therapy.
12. Any contraindications for lumbar puncture or repeated intrathecal injection and/or underlying disorders that could be affected by intrathecal injections.
13. Prior severe reaction or known hypersensitivity to any part of the Study Drug.

Contacts and Locations

Study Contact

Medical Director, Amylyx

CONTACT

857-320-6200

clinicaltrials@amylyx.com

Principal Investigator

Medical Director, Amylyx

STUDY_DIRECTOR

Medical Monitor

Study Locations (Sites)

Georgetown University Hospital Pasquerilla Healthcare Center

Washington, District of Columbia, 20007

United States

Massachusetts General Hospital, Healey & AMG Center for ALS

Boston, Massachusetts, 02128

United States

Alliance for Multispecialty Research, LLC

Knoxville, Tennessee, 37909

United States

Collaborators and Investigators

Sponsor: Amylyx Pharmaceuticals Inc.

Medical Director, Amylyx, STUDY_DIRECTOR, Medical Monitor

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-04-07

Study Completion Date2027-10

Study Record Updates

Study Start Date2025-04-07

Study Completion Date2027-10

Terms related to this study

Keywords Provided by Researchers

Amyotrophic Lateral Sclerosis
Sporadic ALS
Motor Neuron Disease
Antisense oligonucleotide
ASO
Calpain-2

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

Conditions

Quick Navigation

Study Overview

Description

Official Title

Quick Facts

Study ID

Participation Criteria

Contacts and Locations

Study Contact

Principal Investigator

Study Locations (Sites)

Collaborators and Investigators

Study Record Dates

Study Registration Dates

Study Record Updates

Terms related to this study

Keywords Provided by Researchers

Additional Relevant MeSH Terms