Clinical Trials

Numerous clinical trials evaluate L-leucine and its derivatives for conditions including acute lymphoblastic leukemia, pyruvate dehydrogenase complex deficiency, fragility fractures, muscle disuse atrophy, major depression, and ataxia-telangiectasia. Ranging from Phase 1 to Phase 3 alongside non-interventional or dietary supplement evaluations, these studies encompass various recruitment statuses from active and completed to expanded access and upcoming. Sponsorship spans government agencies, industry leaders, and academic medical centers.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05163288 RECRUITING
Niemann-Pick Disease, Type C
IntraBio Inc
 2022-06-30 PHASE3
NCT07438158 NOT_YET_RECRUITING
Muscle Damage
Tel Aviv University
 2026-03
NCT06580145 RECRUITING
Major Depression
Emory University
 2025-02-01 PHASE2
NCT06673056 ACTIVE_NOT_RECRUITING
Ataxia-Telangiectasia; Ataxia-Telangiectasia (A-T)
IntraBio Inc
 2025-03-18 PHASE3
NCT07221292 RECRUITING
CACNA1A; Spinocerebellar Ataxia Type 6; Episodic Ataxia Type 2; Familial Hemiplegic Migraine-1
IntraBio Inc
 2026-08-17 PHASE3
NCT05235464 RECRUITING
Atherosclerosis
University of Missouri-Columbia
 2023-03-13
NCT07275749 RECRUITING
Ischemic Stroke
Kafrelsheikh University
 2025-12-01 PHASE3
NCT05251207 COMPLETED
Insulin Resistance
Poznan University of Physical Education
 2022-02-07
NCT03759678 TERMINATED
Ataxia Telangiectasia
IntraBio Inc
 2020-01-08 PHASE2
NCT05621291 Recruiting
B-All|Acute Lymphoblastic Leukemia
National Cancer Institute (NCI)|National Institutes of Health Clinical Center (CC)
 2024-05-15 Not Applicable
NCT06340685 Recruiting
Pyruvate Dehydrogenase Complex Deficiency
Jirair Krikor Bedoyan|Ultragenyx Pharmaceutical Inc|University of Pittsburgh
 2024-05 Phase 1
NCT05720390 COMPLETED
Healthy
University of Adelaide
 2023-02-02
NCT06256744 Not yet recruiting
Dietary Supplementation|Exercise
Brock University|University of Toronto
 2024-03 Not Applicable
NCT06050668 Not yet recruiting
Femoral Fracture|Fragility Fracture|Muscle Atrophy
Michael C Willey|MEND|University of Iowa
 2024-02 Not Applicable
NCT03759665 COMPLETED
GM2 Gangliosidosis; Tay-Sachs Disease; Sandhoff Disease
IntraBio Inc
 2019-06-07 PHASE2
NCT03759639 COMPLETED
Niemann-Pick Disease, Type C
IntraBio Inc
 2019-09-04 PHASE2
NCT05175768 TERMINATED
COVID-19 Infection
Vedic Lifesciences Pvt. Ltd.
 2021-12-27
NCT04247048 COMPLETED
Cholesterol; Metabolic Disorder; Lipoproteinemia
Nantes University Hospital
 2020-07-31
NCT06028607 COMPLETED
Primary Ciliary Dyskinesia
University of Leeds
 2021-05-17
NCT03079297 TERMINATED
Major Depressive Disorder
University of Texas Southwestern Medical Center
 2017-03-09 PHASE2
NCT03762278 COMPLETED
Muscle Disuse Atrophy
University of Birmingham
 2017-03-01
NCT05120011 COMPLETED
Osteoporosis
Poznan University of Physical Education
 2017-09-01
NCT02332434 UNKNOWN
Obesity; Diabetes
Assistance Publique Hopitaux De Marseille
 2015-03
NCT03501628 COMPLETED
Skeletal Muscle
Auburn University
 2016-08-17
NCT05009654 COMPLETED
Muscle Loss
Gdansk University of Physical Education and Sport
 2016-10-01
NCT02629627 COMPLETED
Metabolic Syndrome
Centro Universitario de Ciencias de la Salud, Mexico
 2016-03 PHASE2
NCT01326416 COMPLETED
Obesity; Metabolic Syndrome
University Hospital, Rouen
 2011-03
NCT02823249 COMPLETED
Endocrine System Diseases
University Hospital, Basel, Switzerland
 2014-05
NCT02563847 COMPLETED
Endocrine System Diseases
University Hospital, Basel, Switzerland
 2014-05
NCT02386267 UNKNOWN
Diamond Blackfan Anemia
Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache
 2014-09 PHASE2
NCT01593605 COMPLETED
Impaired Glucose Tolerance
Vanderbilt University
 2012-02
NCT00929929 COMPLETED
Ageing
Hospital Clinic of Barcelona
 2009-12
NCT00683826 COMPLETED
Overweight; Obesity
Columbia University
 2007-05
NCT00429416 COMPLETED
Hematologic Malignancies
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
 2004-03 PHASE1; PHASE2
NCT00429039 TERMINATED
ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
 2000-09 PHASE2

(data from https://clinicaltrials.gov, updated on 2026-04-03)

Check the L-Leucine product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

L-Leucine directly interacts with intracellular nutrient sensors to activate the mammalian target of rapamycin complex 1 (mTORC1) pathway, which phosphorylates downstream effectors such as S6 kinase 1 to drive cellular protein synthesis and ribosomal biogenesis. This activation enhances protein accretion and suppresses proteolysis, providing a mechanistic foundation for clinical studies evaluating its efficacy in treating muscle disuse atrophy, fragility fractures, and metabolic or neurological conditions.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.