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New treatment option for ALS
Official title AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
ClinicalTrials.gov ID: NCT06665165
What this study is testing
What is AMX0114?
AMX0114 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for als.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 1: an early, usually small safety study
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Ability to understand the purpose and risks of this study, willingness to comply with the study and to provide informed consent in accordance with...
- Male or female, at least 18 years of age.
- Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS.
- 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...
- If the participant is to be treated with riluzole and/or edaravone before or during the trial, then treatment must be previously started and...
You likely can't join if
- Presence of tracheostomy or permanent assisted ventilation.
- SVC less than 65%.
- Abnormal liver function defined as aspartate aminotransferase and/or alanine aminotransferase \> 3 times the upper limit of normal (ULN) and/or total...
- Abnormal renal function defined as estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73m2.
- Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial...
- Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding.
See the full eligibility criteria
- 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.
- Male or female, at least 18 years of age.
- Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS.
- 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).
- 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...
- 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...
- 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.
- 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.
- Presence of tracheostomy or permanent assisted ventilation.
- SVC less than 65%.
- 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...
- Abnormal renal function defined as estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73m2.
- Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time.
- Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding.
- 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.
- 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.
- 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.
- 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.
- Current or previous treatment with small interfering ribonucleic acid, stem cell therapy, any ASO or gene therapy.
- Any contraindications for lumbar puncture or repeated intrathecal injection and/or underlying disorders that could be affected by intrathecal injections.
- Prior severe reaction or known hypersensitivity to any part of the Study Drug.
The study team makes the final eligibility decision.
Where it's taking place
- La Jolla, California, United States
- Washington D.C., District of Columbia, United States
- Gainesville, Florida, United States
- Jacksonville, Florida, United States
- Orlando, Florida, United States
- Boston, Massachusetts, United States
- Rochester, Minnesota, United States
- Philadelphia, Pennsylvania, United States
- Knoxville, Tennessee, United States
- Houston, Texas, United States
- Calgary, Alberta, Canada
- Hamilton, Ontario, Canada
- London, Ontario, Canada
- Montreal, Quebec, Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include La Jolla, California, United States; Washington D.C., District of Columbia, United States; Gainesville, Florida, United States; Jacksonville, Florida, United States; Orlando, Florida, United States; Boston, Massachusetts, United States and 8 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.