IRB Number
NCT Number
Open to Enrollment

Study Contact

Local Recruitment Point of Contact
Virginia Diaz
(210) 562-9149

Local Recruitment Point of Contact
Jaclyn Hung
+1 (210) 450-5358

Principal Investigator
Anne-Marie Langevin

Regulatory Point of Contact
Jaclyn Hung
+1 (210) 450-5358

Regulatory Point of Contact
Anne-Marie Langevin
(210) 567-7460


Eligible Ages
Between 2 and 17
Eligible Genders
Accepts Healthy Volunteers

Inclusion Criteria

    • Signed informed consent /assent
    • Age <18 at time of signing informed consent for Part 1
    • Patients aged 18-30 years are eligible only for Parts 2 and 3 of the study
    • Able to swallow tablets or liquids
    • Study Part 1 (single-agent therapy dose escalation): neuroblastoma or other solid tumor that has progressed or recurred despite standard therapy, and forwhich there is no therapy proven to prolong survival with an acceptable qualityof life
    • Adequate performance status: Lansky/Karnofsky = 50%?Standard minimum for creatinine/GFR, bilirubin, AST, ALT, LVEF/fractionalshortening
    • Compliance with contraception requirements
    • Evaluable disease (does not have to be measurable)

    • Tumor tissue from relapsed disease, obtained subsequent to last anti-cancer therapy regimen administered and obtained within 6 months prior to study enrollment

    • Life expectancy >=12 weeks (investigator judgment)

    • Adequate organ function

Exclusion Criteria

    • Primary CNS tumors or symptomatic CNS mets; CNS3 leukemia
    • Acute Promyelocytic leukemia, Burkitt ALL, or T-Cell lymphoblastic leukemia
    • Down syndrome, Li-Fraumeni syndrome, history of severe aplastic anemia/or any other bone marrow failure predisposition syndrome
    • WBC >50 x 109/L (pretreatment leucoreductionwith pheresisor hydroxyurea permitted)
    • Prior treatment with an MDM2-inhibitor
    • Prior treatment with venetoclax(for patients who potentially would be enrolled in a venetoclaxarm)
    • Medication restrictions 7-14 days prior to therapy start (see prohibited therapies)
    • Clinically uncontrolled Infections (see protocol for details on infections), Active GI disease, Active viral hepatitis or HIV
    • Presence of treatment related toxicity (CTCAE >=2 with exceptions), QTcF>450ms
    • Recent concomitant anti-cancer therapy (see protocol for details related to specific therapies)
    • Pregnant or breastfeeding
    • Received anti-coagulant or anti-platelet agent within 7 days or 5 half-lives prior to study treatment initiation
    • Underwent major surgical procedure within 21 days of study treatment initiation, or anticipate need for major surgical procedure

Study Design


Study Part 1: Single agent dose escalation and combo run-in

A) Dose escalation: in solid tumors to assess safety, tolerability, and pharmacokinetics of idasanutlin as a single-agent treatment in the pediatric population; and to identify the single-agent MTD/MAD and characterize DLTs

B) Early Combination: Patients from dose escalation after one cycle will either continue single-agent idasanutlin or start early combination of idasanutlin with chemotherapy

Arm Groups