Clinical Trials

Tosedostat has been evaluated in multiple clinical trials across Phase 1, Phase 2, and Phase 1/2 protocols for hematologic malignancies, such as acute myeloid leukemia and myelodysplastic syndromes, and solid tumors including non-small-cell lung carcinoma. Sponsored by biotechnology firms like Chroma Therapeutics alongside major academic medical centers such as M.D. Anderson and Fred Hutchinson, these studies investigated monotherapy as well as combination regimens with erlotinib or paclitaxel. While several trials are completed, others have been terminated or maintain an unknown recruitment status.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT01636609 TERMINATED
Acute Myeloid Leukemia; High Risk Myelodysplastic Syndrome
M.D. Anderson Cancer Center
 2012-11-20 PHASE1
NCT02452346 COMPLETED
Myelodysplastic Syndrome
Weill Medical College of Cornell University
 2015-03-20 PHASE2
NCT02352831 TERMINATED
Pancreatic Cancer; Cancer of Pancreas; Cancer of the Pancreas; Pancreas Cancer
Washington University School of Medicine
 2015-08-31 PHASE1; PHASE2
NCT01567059 COMPLETED
Acute Myeloid Leukemia With Multilineage Dysplasia; Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; Adult Acute Myeloid Leukemia With Del(5q); Adult Acute Myeloid Leukemia With t(15;17)(q22;q12); de Novo Myelodysplastic Syndromes; Previously Treated Myelodysplastic Syndromes; Secondary Acute Myeloid Leukemia; Secondary Myelodysplastic Syndromes; Untreated Adult Acute Myeloid Leukemia
Fred Hutchinson Cancer Center
 2012-05 PHASE2
NCT01180426 UNKNOWN
Acute Myeloid Leukemia
Chroma Therapeutics
 2010-06 PHASE2
NCT01638442 COMPLETED
Healthy
CTI BioPharma
 2012-06 PHASE1
NCT00780598 COMPLETED
Acute Myeloid Leukemia; AML
Chroma Therapeutics
 2009-10 PHASE2
NCT01180426 Unknown status
Acute Myeloid Leukemia
Chroma Therapeutics
 2010-06 Phase 2
NCT00780598 Completed
Acute Myeloid Leukemia|AML
Chroma Therapeutics|Quintiles Inc.
 2009-10 Phase 2
NCT00737555 COMPLETED
Solid Tumor
Chroma Therapeutics
 2006-08 PHASE1
NCT00522938 TERMINATED
Carcinoma, Non-Small-Cell Lung
Chroma Therapeutics
 2007-12 PHASE1; PHASE2
NCT00689000 COMPLETED
Acute Myeloid Leukemia; Myelodysplastic Syndrome; Multiple Myeloma
Chroma Therapeutics
 2006-05 PHASE1; PHASE2
NCT00522938 Terminated
Carcinoma Non-Small-Cell Lung
Chroma Therapeutics
 2007-12 Phase 1|Phase 2
NCT00692354 COMPLETED
Advanced Solid Tumors
Chroma Therapeutics
 2004-10 PHASE1
NCT00737555 Completed
Solid Tumor
Chroma Therapeutics
 2006-08 Phase 1
NCT00689000 Completed
Acute Myeloid Leukemia|Myelodysplastic Syndrome|Multiple Myeloma
Chroma Therapeutics
 2006-05 Phase 1|Phase 2

(data from https://clinicaltrials.gov, updated on 2020-04-13)

Check the Tosedostat (CHR2797) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tosedostat selectively binds to and inhibits intracellular aminopeptidases, including leucine aminopeptidase, puromycin-sensitive aminopeptidase, and aminopeptidase N, which disrupts protein recycling and depletes essential intracellular amino acid pools. This metabolic disruption triggers an amino acid deprivation response that selectively induces apoptosis in malignant cells, providing the biological rational for its clinical evaluation in acute myeloid leukemia, myelodysplastic syndromes, and non-small-cell lung carcinoma.

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.