Clinical Trials

Multiple clinical trials sponsored by academic medical institutions, including the Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University, have evaluated L-Leucyl-L-Leucine methyl ester hydrobromide for allogeneic hematopoietic stem cell transplantation and various hematologic malignancies. Spanning Phase 1 and Phase 2 designs, this research includes a completed Phase 1/2 investigation into non-myeloablative stem cell transplantation as well as a terminated Phase 2 study assessing cellular immunotherapy following T-cell depletion.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
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 2016-11-29)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

L-Leucyl-L-Leucine methyl ester hydrobromide selectively accumulates within the endolysosomal compartment, where it undergoes intracellular conversion into membranolytic polymers that disrupt organelle integrity. This localized pathway disruption induces targeted apoptosis in cytotoxic lymphocytes and natural killer cells, thereby providing a mechanism to mitigate immune-mediated graft reactions during allogeneic hematopoietic stem cell transplantation.

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.