Clinical Trials

Multiple clinical trials spanning Early Phase 1 through Phase 4 investigate this compound across conditions including acute myeloid leukemia, multiple myeloma, skin care, postpartum anemia, and infant malnutrition. Conducted by sponsors such as Wayne State University, Karyopharm Therapeutics Inc., DongE E Jiao Corporation Limited, and Baxter Healthcare Corporation, these evaluations encompass recruitment statuses ranging from completed protocols to withdrawn trials.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06175117 COMPLETED
Postpartum Anemia
DongE E Jiao Coporation Limited
 2023-01-07 PHASE4
NCT02649790 COMPLETED
Relapsed/Refractory Multiple Myeloma (RRMM); Metastatic Colorectal Cancer (mCRC); Metastatic Castration-Resistant Prostate Cancer (mCRPC); Higher-Risk Myelodysplastic Syndrome (HR-MDS); Acute Myeloid Leukemia (AML); Newly Diagnosed Intermediate/High-Risk MDS
Karyopharm Therapeutics Inc
 2016-01 PHASE1; PHASE2
NCT03878381 COMPLETED
Skin Care
Wayne State University
 2019-04-01 EARLY_PHASE1
NCT06894446 WITHDRAWN
Malnutrition, Infant; Enteral Feeding Intolerance
Baxter Healthcare Corporation
 2021-08-20 PHASE3

(data from https://clinicaltrials.gov, updated on 2025-12-15)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Compound E potently binds to and inhibits γ-secretase, thereby preventing γ-secretase-mediated cleavage of Notch and suppressing the generation of β-amyloid(40) and β-amyloid(42) peptides. By blocking downstream Notch signaling and amyloidogenic processing, this cellular inhibition halts abnormal proliferation, demonstrating therapeutic relevance across oncological clinical conditions such as acute myeloid leukemia and multiple myeloma.

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.