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Lay Description

Category

  • Cancers and Other Neoplasms
  • Children's Health
  • Acute Lymphoblasic Leukemia
  • Acute Myeloid Leukemia
  • Neuroblastoma
  • Solid Tumor
IRB Number
20190796HU
NCT Number
ct.gov registration not required
Open to Enrollment
Yes

Eligibility

Eligible Ages
2 - 17
Eligible Genders
All
Accepts Healthy Volunteers
No

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

CURRENTLY ENROLLING ON STUDY PART 1

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

Study Contact


Regulatory Point of Contact
Jaclyn Hung
+1 (210) 450-5358
hungj@uthscsa.edu

Regulatory Point of Contact
Anne-Marie Langevin
(210) 567-7460
langevin@uthscsa.edu

Local Recruitment Point of Contact
Virginia Diaz
(210) 562-9149
diazvr@uthscsa.edu

Local Recruitment Point of Contact
Jaclyn Hung
+1 (210) 450-5358
hungj@uthscsa.edu

Principal Investigator
Anne-Marie Langevin