THERAPY DOSSIER · th-a43da9c76ba8

N-acetyl-L-leucine (compared with N-acetyl-DL-leucine and N-acetyl-D-leucine)

Drug · ChEMBL holds a curated mechanism entry with mechanism_of_action 'Unknown' and no assigned target (FDA label 219132s000, 2024). Racemic acetylleucine is the separate entry CHEMBL1875805 (Tanganil, ATC N07CA04, max_phase 2).

No trauma translation Suggest a correction

This record is classified as “NO TRAUMA TRANSLATION”. Read the indication and notes below for the specific context.

Evidence & regulatory context

Mechanism: ChEMBL holds a curated mechanism entry with mechanism_of_action 'Unknown' and no assigned target (FDA label 219132s000, 2024). Racemic acetylleucine is the separate entry CHEMBL1875805 (Tanganil, ATC N07CA04, max_phase 2).
Target: Not recorded
Injury models: Chemical/toxin
Reported indication: Not recorded
First approval: 2024
Development stage: Not recorded
ChEMBL record: CHEMBL56021 ↗
ChEMBL max phase: 4 / 4 (development scale, not regulatory approval)
Synonyms: N-acetyl-L-leucine; Acetyl-L-leucine; Levacetylleucine; IB1001; Aqneursa; NSC-206316
Note: Not recorded

Clinical trials 8

0 linked trials carry an injury-indication flag. These flags support record-level exploration and sit alongside the stringent aggregate counts described in Methods.

NCT05163288 ↗PHASE3recruiting

Effects of N-acetyl-L-leucine on Niemann-Pick disease type C: Phase III randomised crossover

Niemann-Pick Disease, Type C

Enrolment: 53 · Results not recorded

NCT06673056 ↗PHASE3active not recruiting

Effects of N-acetyl-L-leucine on ataxia-telangiectasia: Phase III randomised crossover

Ataxia-Telangiectasia

Enrolment: 60 · Results not recorded

NCT07221292 ↗PHASE3not yet recruiting

Effects of N-acetyl-L-leucine on CACNA1A disorders: Phase III randomised crossover

CACNA1A · Spinocerebellar Ataxia Type 6 · Episodic Ataxia Type 2

Enrolment: 60 · Results not recorded

NCT07275749 ↗PHASE3recruiting

Safety and efficacy of acetyl-leucine in post-stroke ataxia: randomised placebo-controlled trial

Ischemic Stroke

Enrolment: 200 · Results not recorded

NCT03759639 ↗PHASE2completed

Effects of N-acetyl-L-leucine on Niemann-Pick type C disease: open-label rater-blinded Phase II

Niemann-Pick Disease, Type C

Enrolment: 33 · Results not recorded

NCT03759665 ↗PHASE2completed

Effects of N-acetyl-L-leucine on GM2 gangliosidosis (Tay-Sachs and Sandhoff): Phase II

GM2 Gangliosidosis · Tay-Sachs Disease · Sandhoff Disease

Enrolment: 30 · Results not recorded

NCT03759678 ↗PHASE2terminated

Effects of N-acetyl-L-leucine on ataxia-telangiectasia: Phase II

Ataxia Telangiectasia

Enrolment: 17 · Results not recorded

NCT07380165 ↗Phase not recordedavailable

Expanded access programme of levacetylleucine for ataxia-telangiectasia

Ataxia-Telangiectasia

Enrolment not recorded · Results not recorded

Preclinical evidence 1

1 studies reported at therapy level; 1 evidence entries itemised.

PMID 25803613 ↗2015Mixed findings

N-acetyl-L-leucine accelerates vestibular compensation after unilateral labyrinthectomy by action in the cerebellum and thalamus.

RatChemical/toxinSprague-DawleyMaleIntravenous24 mg intravenously per rat, equivalent to 60 mg/kg body we…After unilateral labyrinthectomy, with assessment before and on days 1, 3, 7 an…

Model: Unilateral chemical labyrinthectomy

Postural imbalance improved at day 7 with N-acetyl-DL-leucine (p<0.03) and N-acetyl-L-leucine (p<0.01) at 60 mg/kg, accelerating postural compensation by about 6 days, but nystagmus and head roll tilt were unchanged and lower doses had no significant effect.

Extraction: extract · Supplied extraction confidence: 0.8. Machine-extracted fields require source verification.

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