Trial reportTargeted oncology2026
Idasanutlin in Combination with Chemotherapy or Venetoclax in Pediatric and Young Adult Patients with Relapsed/Refractory Solid Tumors (iMATRIX Idasa): Results of a Phase I/II, Multicenter, Multi-arm Study.
Trial report in Targeted oncology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04029688 (A Phase I/II, Multicenter, Open-Label, Multi-Arm Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of Idasanutlin in Combination With Either Chemotherapy or Venetoclax in the Treatment of Pediatric and Young Adult Patients With Relapsed/Refractory Acute Leukemias or Solid Tumors), which is not on this map. Cited by 2 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase I/II, Multicenter, Open-Label, Multi-Arm Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of Idasanutlin in Combination With Either Chemotherapy or Venetoclax in the Treatment of Pediatric and Young Adult Patients With Relapsed/Refractory Acute Leukemias or Solid Tumors
Who cites it
2 citing papers in PubMed.
- The MDM2-p53 Axis in Osteosarcoma: Current Understanding of Regulatory Mechanisms and Targeted Therapeutic Strategies.Pharmaceuticals (Basel, Switzerland) · 2026Review
- Tumor suppressor network dysregulation in neuroblastoma: molecular mechanisms and precision therapeutic opportunities.Frontiers in cell and developmental biology · 2026Review
Corrections and comments
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Authors and funding
19 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundMDM2 regulates the P53 pathway and is a promising therapeutic target, particularly in pediatric cancers with wild-type (WT) TP53. Idasanutlin is an investigational MDM2 inhibitor that is highly selective and orally bioavailable. The combination of MDM2 inhibition with cytotoxic chemotherapy or Bcl-2 inhibition has been shown to improve activity in preclinical models.
objectiveiMATRIX idasa was designed to assess the safety, pharmacokinetics, and antitumor activity of idasanutlin in children and young adults with relapsed/refractory solid tumors. PATIENTS AND
methodsThis multicenter phase I/II study enrolled patients aged < 30 years with extracranial solid tumors. Patients received idasanutlin on days 1-5 of a 28-day cycle as a single agent or in combination with venetoclax or chemotherapy. The single-agent dose escalation utilized a modified continual reassessment method. The primary endpoints included the safety and determination of the maximum tolerated dose, characterization of the pharmacokinetics, and preliminary efficacy.
resultsOf 38 patients (median age 9 years [range: 2-23]), 26 with solid tumors received idasanutlin alone, and 12 with neuroblastoma received it in combination: six with venetoclax and six with chemotherapy (cyclophosphamide/topotecan). In total, 5 of 26 patients (19.2%) treated with single-agent idasanutlin experienced dose-limiting toxicities (including thrombocytopenia, neutropenia, and febrile neutropenia), which established a pediatric recommended phase II dose for idasanutlin of 4.5 mg/kg/day on days 1-5 of each 28-day cycle. The most frequently reported treatment-related adverse events were thrombocytopenia and neutropenia. Tolerable exposures were similar to what has been observed in adults. The objective response rate for patients with WT TP53 neuroblastoma who were treated with idasanutlin in combination with venetoclax or chemotherapy (primary efficacy endpoint) was 11.1% (N = 9; 95% confidence intervals, CI 0.28, 48.25) with one patient having a complete response. No other patients in the overall study population had an objective response.
conclusionsThe safety profile and tolerable exposure of idasanutlin in pediatrics was similar to that reported in adults. Limited clinical activity was observed in patients with solid tumors. On the basis of the negative benefit-risk assessment, the study was terminated, and the overall pediatric development program for idasanutlin was discontinued. CLINICAL
trial registrationClinicalTrials.gov NCT04029688, registered 19 July 2019.
Indexed as
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Registered trials
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