{"id":"acetylleucine","rwe":[],"tags":[{"label":"acetylleucine","category":"class"},{"label":"Small Molecule","category":"modality"},{"label":"N07CA04","category":"atc"},{"label":"Active","category":"status"}],"phase":"phase_2","safety":{"safetySignals":[{"llr":77.839,"date":"","count":20,"signal":"Hepatitis acute","source":"DrugCentral FAERS","actionTaken":"Reported 20 times (LLR=78)"},{"llr":74.716,"date":"","count":15,"signal":"Anticoagulation drug level above therapeutic","source":"DrugCentral FAERS","actionTaken":"Reported 15 times (LLR=75)"},{"llr":67.439,"date":"","count":24,"signal":"Haematoma","source":"DrugCentral FAERS","actionTaken":"Reported 24 times (LLR=67)"},{"llr":50.29,"date":"","count":16,"signal":"Inappropriate antidiuretic hormone secretion","source":"DrugCentral FAERS","actionTaken":"Reported 16 times (LLR=50)"}],"commonSideEffects":[{"effect":"Hepatitis acute","drugRate":"LLR 78","severity":"common"},{"effect":"Anticoagulation drug level above therapeutic","drugRate":"LLR 75","severity":"common"},{"effect":"Haematoma","drugRate":"LLR 67","severity":"common"},{"effect":"Inappropriate antidiuretic hormone secretion","drugRate":"LLR 50","severity":"common"}]},"trials":[],"aliases":[],"patents":[],"pricing":[],"allNames":"n-acetylleucine","offLabel":[],"synonyms":["acetylleucine","(S)-2-Acetamido-4-methylpentanoic acid","Acetyl-L-leucine","N-Acetylleucine"],"timeline":[],"brandName":"N-Acetylleucine","ecosystem":[],"mechanism":{"modality":"Small Molecule","drugClass":"acetylleucine","explanation":"Think of it like a traffic cop: N-Acetylleucine helps direct the flow of amino acids in the body by blocking the enzyme that breaks them down. This can help increase the levels of these amino acids, which are important for various bodily functions. By doing so, it may help alleviate certain conditions, although its exact effects are still unknown.","oneSentence":"N-Acetylleucine works by inhibiting the enzyme leucine aminopeptidase, which breaks down certain amino acids.","technicalDetail":"N-Acetylleucine acts as a competitive inhibitor of leucine aminopeptidase, a metalloprotease enzyme involved in the degradation of leucine and other amino acids. This inhibition leads to an accumulation of these amino acids in the body, potentially influencing various physiological processes."},"_wikipedia":{"url":"https://en.wikipedia.org/wiki/Acetylleucine","title":"Acetylleucine","extract":"Acetylleucine (N-acetyl-leucine) is a modified leucine amino acid."},"commercial":{},"references":[{"id":1,"url":"https://drugcentral.org/drugcard/71","fields":["approvals","synonyms","ATC","PK","indications","contraindications","DDIs","targets","patents","FAERS"],"source":"DrugCentral"},{"id":2,"url":"https://clinicaltrials.gov/search?intr=ACETYLLEUCINE","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":3,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=ACETYLLEUCINE","fields":["publications"],"source":"PubMed/NCBI"},{"id":4,"url":"https://en.wikipedia.org/wiki/Acetylleucine","fields":["history","overview"],"source":"Wikipedia"}],"_enrichedAt":"2026-03-30T08:24:10.990024","biosimilars":[],"competitors":[],"genericName":"acetylleucine","indications":{"approved":[],"offLabel":[],"pipeline":[]},"drugCategory":"active","labelChanges":[],"relatedDrugs":[],"trialDetails":[{"nctId":"NCT07221292","phase":"PHASE3","title":"Pivotal Study of N-acetyl-L-leucine for CACNA1A","status":"NOT_YET_RECRUITING","sponsor":"IntraBio Inc","startDate":"2026-09-01","conditions":["CACNA1A","Spinocerebellar Ataxia Type 6","Episodic Ataxia Type 2","Familial Hemiplegic Migraine-1"],"enrollment":60,"completionDate":"2028-11-01"},{"nctId":"NCT03759678","phase":"PHASE2","title":"N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)","status":"TERMINATED","sponsor":"IntraBio Inc","startDate":"2020-01-08","conditions":["Ataxia Telangiectasia"],"enrollment":17,"completionDate":"2025-10-27"},{"nctId":"NCT07380165","phase":"","title":"Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)","status":"AVAILABLE","sponsor":"IntraBio Inc","startDate":"","conditions":["Ataxia-Telangiectasia (A-T)"],"enrollment":0,"completionDate":""},{"nctId":"NCT06673056","phase":"PHASE3","title":"A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)","status":"ACTIVE_NOT_RECRUITING","sponsor":"IntraBio Inc","startDate":"2025-03-18","conditions":["Ataxia-Telangiectasia","Ataxia-Telangiectasia (A-T)"],"enrollment":60,"completionDate":"2028-06-01"},{"nctId":"NCT05163288","phase":"PHASE3","title":"A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C","status":"RECRUITING","sponsor":"IntraBio Inc","startDate":"2022-06-30","conditions":["Niemann-Pick Disease, Type C"],"enrollment":53,"completionDate":"2028-12"},{"nctId":"NCT03759665","phase":"PHASE2","title":"N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)","status":"COMPLETED","sponsor":"IntraBio Inc","startDate":"2019-06-07","conditions":["GM2 Gangliosidosis","Tay-Sachs Disease","Sandhoff Disease"],"enrollment":30,"completionDate":"2023-01-09"},{"nctId":"NCT03759639","phase":"PHASE2","title":"N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)","status":"COMPLETED","sponsor":"IntraBio Inc","startDate":"2019-09-04","conditions":["Niemann-Pick Disease, Type C"],"enrollment":33,"completionDate":"2022-11-07"}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"crossReferences":{"NDDF":"005881","UNII":"K76S41V71X","CHEBI":"CHEBI:166830","INN_ID":"5209","RXNORM":"2375522","UMLSCUI":"C0256552","ChEMBL_ID":"CHEMBL1875805","KEGG_DRUG":"D07350","DRUGBANK_ID":"DB13226","PUBCHEM_CID":"70912","SNOMEDCT_US":"735136009","MESH_SUPPLEMENTAL_RECORD_UI":"C088117"},"formularyStatus":[],"_enricherVersion":"v2","developmentCodes":[],"ownershipHistory":[],"publicationCount":49,"therapeuticAreas":["Other"],"atcClassification":{"source":"DrugCentral","atcCode":"N07CA04","allCodes":["N07CA04"]},"biosimilarFilings":[],"recentPublications":[],"companionDiagnostics":[],"genericManufacturerList":[],"status":"active","companyName":"","companyId":"","modality":"Small molecule","firstApprovalDate":"","aiSummary":"N-Acetylleucine, also known as N-Acetyl-L-Leucine, is being studied as a potential treatment for several rare genetic disorders, including Ataxia-Telangiectasia, Niemann-Pick Disease, Type C, and GM2 Gangliosidosis. A pivotal study, IB1001-303, is currently investigating the effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia in a Phase III, randomized, placebo-controlled, double-blind, crossover study.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":false,"safety":true,"trials":true,"score":3}}