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A Study of the Drugs Prexasertib, Irinotecan, and Temozolomide in People With Desmoplastic Small Round Cell Tumor and Rhabdomyosarcoma

Phase 1
Completed
Conditions
Desmoplastic Small Round Cell Tumor
Rhabdomyosarcoma
Interventions
Registration Number
NCT04095221
Lead Sponsor
Memorial Sloan Kettering Cancer Center
Brief Summary

The purpose of this study is to test whether the study drug prexasertib is a safe and effective treatment for people with DSRCT or RMS when given in combination with the standard drugs irinotecan and temozolomide. The study will test different doses of prexasertib in combination with irinotecan and temozolomide to find the highest dose of prexasertib that causes few or mild side effects in participants.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
21
Inclusion Criteria
  • Consent/Assent: all patients and/or their parents or legally authorized representatives must sign written informed consent; assent, when appropriate, will be obtained according to institutional guidelines

  • Age: patients must be ≥12 months of age at the time of study enrollment

  • Diagnosis: patients must have histologically documented locally advanced or metastatic desmoplastic small round cell tumor or rhabdomyosarcoma (confirmed at MSK)

  • Therapeutic options: patient's current disease state must be one which has failed standard therapy and for which there is no known curative therapy

  • Disease Status: patients must have measurable disease based on RECIST 1.1

  • Performance level: Karnofsky ≥ 50% for patients > 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age

  • Prior Therapy: patients may have had any number of prior therapies, but must have recovered from the acute toxic effects of all prior anti-cancer therapy (other than alopecia) as described below and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment

    °patients who have previously received irinotecan and/or temozolomide will be allowed

    • 21 days must have elapsed after the last dose of cytotoxic or myelosuppressive chemotherapy
    • 7 days must have elapsed after the last dose of anti-cancer agents not known to be myelosuppressive
    • 14 days must have elapsed after radiation therapy, and toxicity related to prior radiation therapy must be recovered to grade ≤ 1
    • 21 days must have elapsed after the last dose of antibody therapy, and toxicity related to prior antibody therapy must be recovered to grade ≤ 1
  • Organ Function Requirements: Adequate bone marrow function defined as:

    • absolute neutrophil count (ANC) ≥ 1500/mm^3
    • platelet count ≥ 100,000/ mm^3
    • hemoglobin ≥ 8 g/dl
  • Adequate renal function defined as:

    • Creatinine clearance or radioisotope glomerular filtration rate ≥ 70 mL/min/1.73m2 OR
    • Serum creatinine based on age/gender derived from the Schwartz formula for estimating GFR53
  • Adequate liver function defined as:

    • Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal for age
    • AST or ALT ≤ 5 x upper limit of normal for patients with liver metastases
    • Serum albumin ≥ 2.5 g/dl
  • Adequate cardiac function defined as:

    • echocardiogram with left ventricular ejection fraction (LVEF) >45%
    • QTc < 470 ms on screening 12 lead electrocardiogram
  • Pregnancy/Contraception

    • post-menarchal females must have a negative urine or serum pregnancy test at screening and ≤ 24 hours prior to study treatment
    • males or females of reproductive potential must be willing to use a barrier method of contraception throughout the course of the study and for 6 months after participation
Exclusion Criteria
  • Patients for whom the investigator deems that irinotecan and temozolomide are not appropriate are not eligible.
  • Patients who have an uncontrolled infection are not eligible.
  • Patients who are pregnant or breast feeding are not eligible.
  • Patients who have a history of Torsades de Pointes, carry a diagnosis of congestive heart failure, or have a family history of prolonged QT syndrome are not eligible.
  • Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study are not eligible.
  • Patients with known hypersensitivity to irinotecan or its excipients are not eligible.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Prexasertib and IrinotecanPrexasertibPatients will have extent of disease scans following every 2 cycles (every 6 weeks on dose levels 0-3, and every 8 weeks on dose level -1 (if required). Patients will be allowed to continue therapy as long as they do not experience dose-limiting toxicities or progression of disease.
Prexasertib and IrinotecanIrinotecanPatients will have extent of disease scans following every 2 cycles (every 6 weeks on dose levels 0-3, and every 8 weeks on dose level -1 (if required). Patients will be allowed to continue therapy as long as they do not experience dose-limiting toxicities or progression of disease.
Primary Outcome Measures
NameTimeMethod
recommended phase II does of Prexasertib1 year

The RP2D is defined as the highest dose level associated with not more than 1 DLT out of 6 patients. A total of 5 dose levels are planned, including 2 back up levels. The DLT's will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. The dose escalation will follow a 3+3 design.

response2 years

will be evaluated in this study using the criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) guideline, version 1.1 (Primary response criteria)54. Changes in the largest diameter (unidimensional measurement) of the tumor lesions and the shortest diameter in the case of malignant lymph nodes are used in the RECIST criteria.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

Memorial Sloan Kettering Cancer Center

🇺🇸

New York, New York, United States

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