Clinical Trials

Multiple clinical trials, spanning Early Phase 1 through Phase 2, evaluate the therapeutic potential, safety, and pharmacokinetics of tolinapant across diverse oncology indications, including advanced solid tumors, lymphomas, triple-negative breast, head and neck, and recurrent platinum-resistant ovarian cancers. Supported by pharmaceutical companies, academic centers, and public institutions, these studies investigate monotherapies as well as promising combination strategies, such as tolinapant with pembrolizumab. Recruitment statuses across these trials range from actively recruiting to completed, terminated, or withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06393751 WITHDRAWN
Platinum-Refractory Fallopian Tube Carcinoma; Platinum-Refractory Ovarian Carcinoma; Platinum-Refractory Primary Peritoneal Carcinoma; Recurrent Fallopian Tube Adenocarcinoma; Recurrent Fallopian Tube Carcinosarcoma; Recurrent Fallopian Tube Clear Cell Adenocarcinoma; Recurrent Fallopian Tube High Grade Serous Adenocarcinoma; Recurrent Fallopian Tube Undifferentiated Carcinoma; Recurrent High Grade Endometrioid Adenocarcinoma; Recurrent Ovarian Adenocarcinoma; Recurrent Ovarian Carcinosarcoma; Recurrent Ovarian Clear Cell Adenocarcinoma; Recurrent Ovarian High Grade Serous Adenocarcinoma; Recurrent Ovarian Seromucinous Carcinoma; Recurrent Ovarian Undifferentiated Carcinoma; Recurrent Platinum-Resistant Fallopian Tube Carcinoma; Recurrent Platinum-Resistant Ovarian Carcinoma; Recurrent Platinum-Resistant Primary Peritoneal Carcinoma; Recurrent Primary Peritoneal Adenocarcinoma; Recurrent Primary Peritoneal Carcinosarcoma; Recurrent Primary Peritoneal Clear Cell Adenocarcinoma; Recurrent Primary Peritoneal High Grade Serous Adenocarcinoma; Recurrent Primary Peritoneal Undifferentiated Carcinoma
National Cancer Institute (NCI)
 2025-10-31 PHASE1; PHASE2
NCT05912075 RECRUITING
Locally-advanced Rectal Cancer
Gustave Roussy, Cancer Campus, Grand Paris
 2023-12-19 PHASE1
NCT06590558 WITHDRAWN
Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IV Breast Cancer AJCC v8; Invasive Breast Carcinoma; Locally Advanced HER2-Negative Breast Carcinoma; Locally Advanced Hormone Receptor-Positive Breast Carcinoma; Locally Advanced Triple-Negative Breast Carcinoma; Metastatic HER2-Negative Breast Carcinoma; Metastatic Hormone Receptor-Positive Breast Carcinoma; Metastatic Triple-Negative Breast Carcinoma; Unresectable HER2-Negative Breast Carcinoma; Unresectable Hormone Receptor-Positive Breast Carcinoma; Unresectable Triple-Negative Breast Carcinoma
National Cancer Institute (NCI)
 2025-08-04 PHASE1
NCT05082259 ACTIVE_NOT_RECRUITING
Advanced Cancer; Cervical Cancer; Triple Negative Breast Cancer
Institute of Cancer Research, United Kingdom
 2022-03-02 PHASE1
NCT05403450 TERMINATED
Relapsed/Refractory Peripheral T-cell Lymphoma
Taiho Oncology, Inc.
 2023-02-22 PHASE1
NCT04362007 TERMINATED
Relapsed or Refractory Peripheral T-cell Lymphoma(PTCL),Cutaneous T-cell Lymphoma(CTCL),Adult T-cell Leukemia/Lymphoma(ATLL)
Otsuka Pharmaceutical Co., Ltd.
 2020-07-14 PHASE1; PHASE2
NCT05245682 ACTIVE_NOT_RECRUITING
Head and Neck Carcinoma of Unknown Primary; Locally Advanced Head and Neck Squamous Cell Carcinoma; Locally Advanced Hypopharyngeal Squamous Cell Carcinoma; Locally Advanced Laryngeal Squamous Cell Carcinoma; Locally Advanced Nasopharyngeal Squamous Cell Carcinoma; Locally Advanced Oropharyngeal Squamous Cell Carcinoma
Emory University
 2022-02-17 EARLY_PHASE1
NCT02503423 ACTIVE_NOT_RECRUITING
Solid Tumors; Lymphoma
Taiho Oncology, Inc.
 2015-07-14 PHASE1; PHASE2
NCT05082259 Recruiting
Advanced Cancer|Cervical Cancer|Triple Negative Breast Cancer
Institute of Cancer Research United Kingdom|Astex Pharmaceuticals Inc.|Merck Sharp & Dohme LLC|Cancer Research UK
 2022-03-02 Phase 1
NCT04155580 TERMINATED
Acute Myeloid Leukemia
Astex Pharmaceuticals, Inc.
 2020-06-12 PHASE1
NCT04479800 COMPLETED
Healthy Volunteer
Astex Pharmaceuticals, Inc.
 2020-07-20 PHASE1
NCT04411030 COMPLETED
Healthy Volunteer
Astex Pharmaceuticals, Inc.
 2020-05-20 PHASE1
NCT04411030 Completed
Healthy Volunteer
Astex Pharmaceuticals Inc.
 2020-05-20 Phase 1
NCT02503423 Active not recruiting
Solid Tumors|Lymphoma
Astex Pharmaceuticals Inc.
 2015-07 Phase 1|Phase 2

(data from https://clinicaltrials.gov, updated on 2025-06-03)

Check the Tolinapant (ASTX660) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tolinapant functions as a potent, non-peptidomimetic antagonist of cIAP1/2 and XIAP that selectively blocks the interaction between SMAC-derived peptides and the BIR3 domains of XIAP and cIAP1, thereby suppressing anti-apoptotic signaling and inducing apoptosis in cancer cells. This mechanism of pro-apoptotic pathway reactivation directly underlies its clinical development for treating advanced solid tumors, lymphomas, and triple-negative breast carcinomas.

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.