Clinical Trials

Idasanutlin (RG7388) has been evaluated across multiple clinical trials spanning Phase 1 through Phase 3 for hematologic malignancies and solid tumors, including acute myeloid leukemia, polycythemia vera, glioblastoma, non-Hodgkin's lymphoma, atypical teratoid/rhabdoid tumors, and breast cancer. Conducted by academic institutions such as St. Jude Children's Research Hospital and University Hospital Heidelberg alongside industry sponsors like Hoffmann-La Roche, these monotherapy and combination studies encompassed completed, terminated, and withdrawn protocols. Overall, this research landscape has focused primarily on assessing safety, pharmacokinetics, and preliminary therapeutic activity.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05952687 WITHDRAWN
Rhabdoid Tumor; Atypical Teratoid/Rhabdoid Tumor; Atypical Teratoid/Rhabdoid Tumor of CNS; CNS Tumor
St. Jude Children's Research Hospital
 2024-03 PHASE1
NCT04029688 TERMINATED
Acute Myeloid Leukemia (AML); Acute Lymphoblastic Leukemia (ALL); Neuroblastoma; Solid Tumors
Hoffmann-La Roche
 2020-01-27 PHASE1; PHASE2
NCT05952687 Withdrawn
Rhabdoid Tumor|Atypical Teratoid/Rhabdoid Tumor|Atypical Teratoid/Rhabdoid Tumor of CNS|CNS Tumor
St. Jude Children''s Research Hospital
 2024-03 Phase 1
NCT03158389 COMPLETED
Glioblastoma, Adult
University Hospital Heidelberg
 2018-05-07 PHASE1; PHASE2
NCT03555149 TERMINATED
Colorectal Cancer
Hoffmann-La Roche
 2018-09-27 PHASE1; PHASE2
NCT02633059 COMPLETED
Loss of Chromosome 17p; Recurrent Plasma Cell Myeloma
Mayo Clinic
 2015-12-30 PHASE1; PHASE2
NCT03566485 TERMINATED
Stage III Breast Cancer; Stage IIIA Breast Cancer; Stage IIIB Breast Cancer; Stage IIIC Breast Cancer; Stage IV Breast Cancer; Estrogen Receptor-positive; HER2/Neu Negative
Vanderbilt-Ingram Cancer Center
 2018-07-10 PHASE1; PHASE2
NCT02670044 COMPLETED
Leukemia, Myeloid, Acute
Hoffmann-La Roche
 2016-03-09 PHASE1
NCT03850535 TERMINATED
Acute Myeloid Leukemia
Hoffmann-La Roche
 2019-03-25 PHASE1; PHASE2
NCT03135262 TERMINATED
Follicular Lymphoma; Lymphoma, Large B-Cell, Diffuse
Hoffmann-La Roche
 2017-06-15 PHASE1; PHASE2
NCT02545283 TERMINATED
Leukemia, Myeloid, Acute
Hoffmann-La Roche
 2015-12-30 PHASE3
NCT03287245 TERMINATED
Polycythemia Vera
Hoffmann-La Roche
 2018-02-21 PHASE2
NCT03362723 COMPLETED
Solid Tumors
Hoffmann-La Roche
 2017-11-27 PHASE1
NCT02624986 TERMINATED
Non-Hodgkin's Lymphoma
Hoffmann-La Roche
 2015-12-23 PHASE1; PHASE2
NCT02407080 COMPLETED
Polycythemia Vera; Essential Thrombocythemia
John Mascarenhas
 2015-04 PHASE1
NCT03287245 Terminated
Polycythemia Vera
Hoffmann-La Roche
 2018-02-21 Phase 2
NCT03362723 Completed
Solid Tumors
Hoffmann-La Roche
 2017-11-27 Phase 1
NCT02828930 COMPLETED
Solid Tumors
Hoffmann-La Roche
 2016-09-08 PHASE1

(data from https://clinicaltrials.gov, updated on 2024-03-27)

Check the Idasanutlin (RG7388) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Idasanutlin selectively binds to MDM2, disrupting the p53-MDM2 interaction to inhibit p53 degradation and restore downstream p53-mediated transcriptional activation. This pathway activation induces cell cycle arrest and apoptosis in tumor cells, thereby driving anti-neoplastic activity in clinical conditions such as acute myeloid leukemia, glioblastoma, and 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.