Clinical Trials

Multiple Phase I and Phase II clinical trials are currently evaluating Siremadlin (HDM201) across oncology indications, including hematologic malignancies—such as acute myeloid leukemia, high-risk myelodysplastic syndrome, and myelofibrosis—and solid tumors such as advanced soft-tissue sarcoma. Sponsored by Novartis Pharmaceuticals alongside academic and institutional partners including Centre Leon Berard and Insel Gruppe AG, these studies encompass various recruitment statuses, including actively recruiting, active not recruiting, completed, and terminated protocols.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05180695 ACTIVE_NOT_RECRUITING
Advanced Soft-tissue Sarcoma; Metastatic Soft-tissue Sarcoma
Centre Leon Berard
 2022-04-15 PHASE1; PHASE2
NCT04116541 RECRUITING
Malignant Solid Tumor
Centre Leon Berard
 2020-01-28 PHASE2
NCT03940352 TERMINATED
Acute Myeloid Leukemia (AML); High-risk Myelodysplastic Syndrome (MDS)
Novartis Pharmaceuticals
 2019-06-24 PHASE1
NCT05155709 COMPLETED
Acute Myeloid Leukemia
Novartis Pharmaceuticals
 2022-05-17 PHASE1
NCT05447663 TERMINATED
Acute Myeloid Leukemia; Allogeneic Stem Cell Transplantation
Novartis Pharmaceuticals
 2023-02-23 PHASE1; PHASE2
NCT05599932 COMPLETED
Hepatic Impairment
Novartis Pharmaceuticals
 2022-12-02 PHASE1
NCT03714958 COMPLETED
Colorectal Cancer; Advanced Cancer; Metastatic Cancer
Centre Leon Berard
 2018-12-20 PHASE1
NCT04097821 TERMINATED
Myelofibrosis
Novartis Pharmaceuticals
 2019-09-26 PHASE1; PHASE2
NCT05447663 Terminated
Acute Myeloid Leukemia|Allogeneic Stem Cell Transplantation
Novartis Pharmaceuticals|Novartis
 2023-02-23 Phase 1|Phase 2
NCT04496999 TERMINATED
AML, Adult
Insel Gruppe AG, University Hospital Bern
 2020-07-13 PHASE1
NCT05155709 Active not recruiting
Acute Myeloid Leukemia
Novartis Pharmaceuticals|Novartis
 2022-05-17 Phase 1
NCT05180695 Recruiting
Advanced Soft-tissue Sarcoma|Metastatic Soft-tissue Sarcoma
Centre Leon Berard|Novartis|National Cancer Institute France
 2022-04-15 Phase 1|Phase 2
NCT02890069 COMPLETED
Colorectal Cancer, Non-small Cell Lung Carcinoma (Adenocarcinoma), Triple Negative Breast Cancer, Renal Cell Carcinoma
Novartis Pharmaceuticals
 2016-10-14 PHASE1
NCT02601378 TERMINATED
Uveal Melanoma
Novartis Pharmaceuticals
 2016-02-01 PHASE1
NCT03760445 WITHDRAWN
Leukemia, Myeloid, Acute
Novartis Pharmaceuticals
 2019-11-15 PHASE1; PHASE2
NCT02343172 COMPLETED
Liposarcoma
Novartis Pharmaceuticals
 2015-03-13 PHASE1
NCT03940352 Active not recruiting
Acute Myeloid Leukemia (AML)|High-risk Myelodysplastic Syndrome (MDS)
Novartis Pharmaceuticals|Novartis
 2019-06-24 Phase 1
NCT02143635 COMPLETED
Advanced Solid and Hematological TP53wt Tumors
Novartis Pharmaceuticals
 2014-07-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-06-22)

Check the Siremadlin (HDM201) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Siremadlin (HDM201) acts as a highly potent and selective inhibitor of the p53-Mdm2 interaction with picomolar Mdm2 binding affinity and high selectivity over Mdm4, preventing Mdm2-mediated degradation of p53. This inhibition stabilizes functional p53 and reactivates downstream p53-dependent transcriptional programs to induce cell cycle arrest and apoptosis, driving its therapeutic utility in clinical trials for p53 wild-type malignancies including acute myeloid leukemia and soft-tissue sarcoma.

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.