Clinical Trials

Eprenetapopt (APR-246) has been evaluated across numerous Phase 1, Phase 2, and Phase 3 clinical trials as both a single agent and in combination regimens to restore tumor-suppressor activity. These studies target solid tumors—including non-small cell lung, bladder, gastric, melanoma, and esophageal carcinomas—alongside hematologic malignancies such as myelodysplastic syndrome and acute myeloid leukemia. Supported by commercial sponsors like Aprea Therapeutics and academic institutions including M.D. Anderson Cancer Center, trial recruitment statuses range from completed to terminated or withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT04990778 WITHDRAWN
Recurrent Mantle Cell Lymphoma; Refractory Mantle Cell Lymphoma
M.D. Anderson Cancer Center
 2021-11-30 PHASE2
NCT04383938 COMPLETED
Bladder Cancer; Gastric Cancer; Non Small Cell Lung Cancer; NSCLC; Urothelial Carcinoma; Advanced Solid Tumor
Aprea Therapeutics
 2020-06-25 PHASE1; PHASE2
NCT04214860 COMPLETED
Myeloid Malignancy
Aprea Therapeutics
 2019-12-13 PHASE1
NCT03931291 COMPLETED
Acute Myeloid Leukemia or Myelodysplastic Syndromes
Aprea Therapeutics
 2019-09-16 PHASE2
NCT04419389 TERMINATED
Non Hodgkin Lymphoma; Chronic Lymphocytic Leukemia; Mantle Cell Lymphoma
Aprea Therapeutics
 2021-03-02 PHASE1; PHASE2
NCT03745716 COMPLETED
MDS
Aprea Therapeutics
 2019-01-11 PHASE3
NCT02999893 TERMINATED
Oesophageal Carcinoma
Peter MacCallum Cancer Centre, Australia
 2017-04-11 PHASE1; PHASE2
NCT04383938 Completed
Bladder Cancer|Gastric Cancer|Non Small Cell Lung Cancer|NSCLC|Urothelial Carcinoma|Advanced Solid Tumor
Aprea Therapeutics
 2020-06-25 Phase 1|Phase 2
NCT03588078 UNKNOWN
Myelodysplastic Syndrome With Gene Mutation; Acute Myeloid Leukemia With Gene Mutations; Myeloproliferative Neoplasm; Chronic Myelomonocytic Leukemia
Groupe Francophone des Myelodysplasies
 2018-09-15 PHASE1; PHASE2
NCT04214860 Completed
Myeloid Malignancy
Aprea Therapeutics
 2019-12-13 Phase 1
NCT03072043 COMPLETED
Myelodysplastic Syndrome; Acute Myeloid Leukemia; Myeloproliferative Neoplasm; Chronic Myelomonocytic Leukemia
H. Lee Moffitt Cancer Center and Research Institute
 2017-05-18 PHASE1; PHASE2
NCT03268382 COMPLETED
High-grade Serous Ovarian Cancer
Aprea Therapeutics
 2017-07-31 PHASE2
NCT02098343 COMPLETED
Platinum Sensitive Recurrent High-grade Serous Ovarian Cancer With Mutated p53
Aprea Therapeutics
 2014-03 PHASE1; PHASE2
NCT03391050 TERMINATED
Melanoma
Aprea Therapeutics
 2018-01-18 PHASE1; PHASE2
NCT03391050 Terminated
Melanoma
Aprea Therapeutics|Jules Bordet Institute
 2018-01-18 Phase 1|Phase 2
NCT02999893 Terminated
Oesophageal Carcinoma
Peter MacCallum Cancer Centre Australia
 2017-04-11 Phase 1|Phase 2
NCT00900614 COMPLETED
Hematologic Neoplasms; Prostatic Neoplasms
Aprea Therapeutics
 2009-05 PHASE1

(data from https://clinicaltrials.gov, updated on 2022-03-10)

Check the Eprenetapopt (APR-246) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Eprenetapopt selectively binds to mutant p53 proteins to restore their wild-type tumor-suppressor conformation and functional activity, thereby reactivating downstream signaling pathways that induce apoptosis and autophagy. This targeted restoration of apoptotic signaling inhibits malignant proliferation and underlies its clinical evaluation in TP53-mutated conditions, including myelodysplastic syndrome, acute myeloid leukemia, and advanced solid tumors.

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.