[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100567206":3},{"organization":4,"armGroups":7,"interventions":20,"overallOfficials":30,"centralContacts":35,"locations":40,"responsibleParty":301,"collaborators":26,"id":303,"slug":304,"hasResults":305,"nctId":306,"briefTitle":307,"officialTitle":308,"acronym":309,"eligibilityCriteria":310,"healthyVolunteers":305,"sex":311,"minAge":312,"maxAge":26,"enrollmentInfo":313,"targetDuration":26,"studyType":316,"phases":317,"briefSummary":319,"conditions":320,"keywords":322,"overallStatus":43,"whyStopped":26,"lastUpdateSubmitDate":329,"lastUpdatePostDateStruct":330,"startDateStruct":333,"completionDateStruct":335,"leadSponsor":337,"locationsCount":338},{"fullName":5,"class":6},"Amylyx Pharmaceuticals Inc.","INDUSTRY",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Active Treatment: AMX0114","EXPERIMENTAL","AMX0114 will be administered once every 4 weeks by intrathecal bolus injection for a total of up to 4 doses. Treatment will be administered on Day 1, followed by repeat dosing every 4 weeks at approximately Day 29, Day 57 and Day 85.",[13],"Drug: AMX0114",{"label":15,"type":16,"description":17,"interventionNames":18},"Placebo","PLACEBO_COMPARATOR","Placebo drug will be administered once every 4 weeks by intrathecal bolus injection for a total of up to 4 doses. Treatment will be administered on Day 1, followed by repeat dosing every 4 weeks at approximately Day 29, Day 57 and Day 85.",[19],"Other: Placebo",[21,27],{"type":22,"name":23,"description":24,"armGroupLabels":25,"otherNames":26},"DRUG","AMX0114","Antisense oligonucleotides (ASOs) are a type of medicine that treats diseases by intercepting the mRNA messages sent within the cell, resulting in fewer specific proteins being made. AMX0114 is an ASO that targets the mRNA messenger that instructs the body to create a protein called calpain-2. Calpain-2 has been linked to the degeneration and death of neurons in many neurological diseases, including people living with sporadic ALS. AMX0114 is designed to reduce the levels of calpain-2, with the goal of slowing down the process that leads to neuron injury and death.",[9],null,{"type":28,"name":15,"description":15,"armGroupLabels":29,"otherNames":26},"OTHER",[15],[31],{"name":32,"affiliation":33,"role":34},"Medical Director, Amylyx","Medical Monitor","STUDY_DIRECTOR",[36],{"name":32,"role":37,"phone":38,"phoneExt":26,"email":39},"CONTACT","857-320-6200","clinicaltrials@amylyx.com",[41,63,80,97,113,129,149,170,190,210,231,250,268,285],{"facility":42,"status":43,"city":44,"state":45,"zip":46,"country":47,"countryCode":48,"cosmosGeoPoint":49,"geoPoint":54,"contacts":55},"University of California, San Diego","RECRUITING","La Jolla","California","92093","United States","US",{"type":50,"coordinates":51},"Point",[52,53],-117.2742,32.84727,{"lat":53,"lon":52},[56,60],{"name":57,"role":37,"phone":58,"phoneExt":26,"email":59},"Rose Previte","858-246-1319","rprevite@ucsd.edu",{"name":61,"role":62,"phone":26,"phoneExt":26,"email":26},"John Ravits, MD","PRINCIPAL_INVESTIGATOR",{"facility":64,"status":43,"city":65,"state":66,"zip":67,"country":47,"countryCode":48,"cosmosGeoPoint":68,"geoPoint":72,"contacts":73},"Georgetown University Hospital Pasquerilla Healthcare Center","Washington D.C.","District of Columbia","20007",{"type":50,"coordinates":69},[70,71],-77.03637,38.89511,{"lat":71,"lon":70},[74,78],{"name":75,"role":37,"phone":76,"phoneExt":26,"email":77},"Cate Ledoux","240-681-9556","csl83@georgetown.edu",{"name":79,"role":62,"phone":26,"phoneExt":26,"email":26},"Shakti Nayar, MD",{"facility":81,"status":43,"city":82,"state":83,"zip":84,"country":47,"countryCode":48,"cosmosGeoPoint":85,"geoPoint":89,"contacts":90},"University of Florida","Gainesville","Florida","32611",{"type":50,"coordinates":86},[87,88],-82.32483,29.65163,{"lat":88,"lon":87},[91,95],{"name":92,"role":37,"phone":93,"phoneExt":26,"email":94},"Jennifer Steshyn","518-262-5226","jennifer.steshyn@neurology.ufl.edu",{"name":96,"role":62,"phone":26,"phoneExt":26,"email":26},"James Wymer, MD, FAAN",{"facility":98,"status":43,"city":99,"state":83,"zip":100,"country":47,"countryCode":48,"cosmosGeoPoint":101,"geoPoint":105,"contacts":106},"Mayo Clinic in Florida","Jacksonville","32224",{"type":50,"coordinates":102},[103,104],-81.65565,30.33218,{"lat":104,"lon":103},[107,111],{"name":108,"role":37,"phone":109,"phoneExt":26,"email":110},"Jeffery Gainer","904-244-4693","jeffery.gainer@jax.ufl.edu",{"name":112,"role":62,"phone":26,"phoneExt":26,"email":26},"Bjorn Oskarsson, MD",{"facility":114,"status":43,"city":115,"state":83,"zip":116,"country":47,"countryCode":48,"cosmosGeoPoint":117,"geoPoint":121,"contacts":122},"Orlando Regional Medical Center, Orlando Health Neuroscience Institute","Orlando","32806",{"type":50,"coordinates":118},[119,120],-81.37924,28.53834,{"lat":120,"lon":119},[123,127],{"name":124,"role":37,"phone":125,"phoneExt":26,"email":126},"Charlene Carlo","321-841-1324","Charlene.carlo@orlandohealth.com",{"name":128,"role":62,"phone":26,"phoneExt":26,"email":26},"Vahid Tohidi, MD, PhD",{"facility":130,"status":43,"city":131,"state":132,"zip":133,"country":47,"countryCode":48,"cosmosGeoPoint":134,"geoPoint":138,"contacts":139},"Massachusetts General Hospital, Healey & AMG Center for ALS","Boston","Massachusetts","02114",{"type":50,"coordinates":135},[136,137],-71.05977,42.35843,{"lat":137,"lon":136},[140,144,147],{"name":141,"role":37,"phone":142,"phoneExt":26,"email":143},"Lucy Lee","617-643-7434","amx0114healey@mgb.org",{"name":145,"role":37,"phone":146,"phoneExt":26,"email":143},"Anika Allen","617-724-9196",{"name":148,"role":62,"phone":26,"phoneExt":26,"email":26},"Sabrina Paganoni, MD, PhD",{"facility":150,"status":43,"city":151,"state":152,"zip":153,"country":47,"countryCode":48,"cosmosGeoPoint":154,"geoPoint":158,"contacts":159},"Mayo Clinic in Rochester","Rochester","Minnesota","55905",{"type":50,"coordinates":155},[156,157],-92.4699,44.02163,{"lat":157,"lon":156},[160,164,168],{"name":161,"role":37,"phone":162,"phoneExt":26,"email":163},"Brenda Nelson","507-293-9237","nelson.brenda6@mayo.edu",{"name":165,"role":37,"phone":166,"phoneExt":26,"email":167},"Jeff Laivell","507-538-8095","laivell.jeffrey@mayo.com",{"name":169,"role":62,"phone":26,"phoneExt":26,"email":26},"Eric Sorenson, MD",{"facility":171,"status":43,"city":172,"state":173,"zip":174,"country":47,"countryCode":48,"cosmosGeoPoint":175,"geoPoint":179,"contacts":180},"Temple University of the Commonwealth System of Higher Education","Philadelphia","Pennsylvania","19140",{"type":50,"coordinates":176},[177,178],-75.16362,39.95238,{"lat":178,"lon":177},[181,184,188],{"name":182,"role":37,"phone":26,"phoneExt":26,"email":183},"Kathleen Hatala, 215-707-4171","kathleen.hatala@tuhs.temple.edu",{"name":185,"role":37,"phone":186,"phoneExt":26,"email":187},"Christine Barr","215-707-5440","christine.barr@tuhs.temple.edu",{"name":189,"role":62,"phone":26,"phoneExt":26,"email":26},"Terry Heiman - Patterson, MD",{"facility":191,"status":43,"city":192,"state":193,"zip":194,"country":47,"countryCode":48,"cosmosGeoPoint":195,"geoPoint":199,"contacts":200},"Alliance for Multispecialty Research, LLC","Knoxville","Tennessee","37909",{"type":50,"coordinates":196},[197,198],-83.92074,35.96064,{"lat":198,"lon":197},[201,205,208],{"name":202,"role":37,"phone":203,"phoneExt":26,"email":204},"Patricia Pryor","865-305-9100","patricia.pryor@amr-clinical.com",{"name":206,"role":37,"phone":26,"phoneExt":26,"email":207},"Felicia Hiatt","Felicia.Hiatt@amr-clinical.com",{"name":209,"role":62,"phone":26,"phoneExt":26,"email":26},"Randall Trudell, MD",{"facility":211,"status":43,"city":212,"state":213,"zip":214,"country":47,"countryCode":48,"cosmosGeoPoint":215,"geoPoint":219,"contacts":220},"Houston Methodist Neurological Institute","Houston","Texas","77030",{"type":50,"coordinates":216},[217,218],-95.36327,29.76328,{"lat":218,"lon":217},[221,225,229],{"name":222,"role":37,"phone":223,"phoneExt":26,"email":224},"Jennifer Garrett","634-238-4516","jmgarrett@houstonmethodist.org",{"name":226,"role":37,"phone":227,"phoneExt":26,"email":228},"Madison Taylor","346-356-3678","mtaylor5@houstonmethodist.org",{"name":230,"role":62,"phone":26,"phoneExt":26,"email":26},"Sheetal Shroff, MD",{"facility":232,"status":43,"city":233,"state":234,"zip":235,"country":236,"countryCode":237,"cosmosGeoPoint":238,"geoPoint":242,"contacts":243},"University of Calgary","Calgary","Alberta","T2N 4Z6","Canada","CA",{"type":50,"coordinates":239},[240,241],-114.08529,51.05011,{"lat":241,"lon":240},[244,248],{"name":245,"role":37,"phone":246,"phoneExt":26,"email":247},"Janet Petrillo Ballantyne","403-210-7006","japetril@ucalgary.ca",{"name":249,"role":62,"phone":26,"phoneExt":26,"email":26},"Collin Luk, MD, PhD",{"facility":251,"status":43,"city":252,"state":253,"zip":254,"country":236,"countryCode":237,"cosmosGeoPoint":255,"geoPoint":259,"contacts":260},"McMaster University","Hamilton","Ontario","L8N 3Z5",{"type":50,"coordinates":256},[257,258],-79.84963,43.25011,{"lat":258,"lon":257},[261,266],{"name":262,"role":37,"phone":263,"phoneExt":264,"email":265},"Daniela Trapsa","905-521-2100","76368","trapsd@mcmaster.ca",{"name":267,"role":62,"phone":26,"phoneExt":26,"email":26},"Marvin Chum, MD",{"facility":269,"status":43,"city":270,"state":253,"zip":271,"country":236,"countryCode":237,"cosmosGeoPoint":272,"geoPoint":276,"contacts":277},"London Health Sciences Centre","London","N6G 2M3",{"type":50,"coordinates":273},[274,275],-81.23304,42.98339,{"lat":275,"lon":274},[278,283],{"name":279,"role":37,"phone":280,"phoneExt":281,"email":282},"Denise Hulley","5196858500","34345","denise.hulley@lhsc.on.ca",{"name":284,"role":62,"phone":26,"phoneExt":26,"email":26},"Christen Shoesmith, MD",{"facility":286,"status":43,"city":287,"state":288,"zip":289,"country":236,"countryCode":237,"cosmosGeoPoint":290,"geoPoint":294,"contacts":295},"McGill University Health Centre - Centre for Innovative Medicine","Montreal","Quebec","H4A 3J1",{"type":50,"coordinates":291},[292,293],-73.58781,45.50884,{"lat":293,"lon":292},[296,299],{"name":297,"role":37,"phone":26,"phoneExt":26,"email":298},"Maria Gobbo","maria.gobbo@muhc.mcgill.ca",{"name":300,"role":62,"phone":26,"phoneExt":26,"email":26},"Angela Genge, MD",{"type":302,"investigatorFullName":26,"investigatorTitle":26,"investigatorAffiliation":26,"oldNameTitle":26,"oldOrganization":26},"SPONSOR","100567206","phase-1-amx0114-in-adult-participants-with-amyotrophic-lateral-sclerosis-100567206",false,"NCT06665165","AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis","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","LUMINA","Inclusion Criteria:\n\n1. 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.\n2. Male or female, at least 18 years of age.\n3. Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS.\n4. Time since onset of first symptom of ALS should be \\\u003C24 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).\n5. If the participant is to be treated with riluzole and\u002For 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.\n6. 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.\n7. 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.\n8. 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.\n\nExclusion Criteria:\n\n1. Presence of tracheostomy or permanent assisted ventilation.\n2. SVC less than 65%.\n3. Abnormal liver function defined as aspartate aminotransferase and\u002For alanine aminotransferase \\> 3 times the upper limit of normal (ULN) and\u002For total bilirubin \\> 1.5 times the ULN (obtained within 4 weeks of first dose) except when a result of Gilbert syndrome.\n4. Abnormal renal function defined as estimated glomerular filtration rate (eGFR) \\\u003C 60 mL\u002Fmin\u002F1.73m2.\n5. Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time.\n6. Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding.\n7. 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.\n8. Significant abnormalities in physical\u002Fneurological examination, vital signs, or electrocardiogram (ECG), which in the opinion of the Investigator could affect the safety of the participant.\n9. 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.\n10. 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.\n11. Current or previous treatment with small interfering ribonucleic acid, stem cell therapy, any ASO or gene therapy.\n12. Any contraindications for lumbar puncture or repeated intrathecal injection and\u002For underlying disorders that could be affected by intrathecal injections.\n13. Prior severe reaction or known hypersensitivity to any part of the Study Drug.","ALL","18 Years",{"count":314,"type":315},48,"ESTIMATED","INTERVENTIONAL",[318],"PHASE1","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).",[321],"ALS",[323,324,325,326,327,328],"Amyotrophic Lateral Sclerosis","Sporadic ALS","Motor Neuron Disease","Antisense oligonucleotide","ASO","Calpain-2","2026-04-09",{"date":331,"type":332},"2026-04-14","ACTUAL",{"date":334,"type":332},"2025-04-07",{"date":336,"type":315},"2027-10",{"name":5,"class":6},14]