Clinical Trials

CB-5339 has been evaluated in several clinical trials focusing on safety, pharmacokinetics, and initial efficacy in Phase 1 studies for hematological malignancies and solid tumors. Industry sponsor Cleave Therapeutics, Inc. reached completed status for a clinical trial in patients with relapsed acute myeloid leukemia and myelodysplastic syndromes. Meanwhile, a clinical trial sponsored by the National Cancer Institute targeting advanced solid tumors and aggressive or indolent non-Hodgkin lymphomas was subsequently withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT04402541 COMPLETED
Acute Myeloid Leukemia, in Relapse; Myelodysplastic Syndromes
Cleave Therapeutics, Inc.
 2020-06-08 PHASE1
NCT04372641 WITHDRAWN
Aggressive Non-Hodgkin Lymphoma; Indolent Non-Hodgkin Lymphoma; Locally Advanced Malignant Solid Neoplasm; Metastatic Malignant Solid Neoplasm
National Cancer Institute (NCI)
 2020-06-18 PHASE1
NCT04402541 Completed
Acute Myeloid Leukemia in Relapse|Myelodysplastic Syndromes
Cleave Therapeutics Inc.
 2020-06-08 Phase 1

(data from https://clinicaltrials.gov, updated on 2023-08-31)

Check the CB-5339 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

CB-5339 selectively binds to and inhibits p97/valosin-containing protein with a half-maximal inhibitory concentration of 9 nM, thereby disrupting essential protein homeostasis pathways including endoplasmic reticulum-associated degradation. This functional blockade leads to severe proteotoxic stress, accumulation of misfolded proteins, and apoptotic cell death, establishing its clinical relevance in trial settings for relapsed acute myeloid leukemia, myelodysplastic syndromes, advanced solid tumors, and non-Hodgkin lymphomas.

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.