KPT-330 for Relapsed Childhood Leukemia

Palo Alto (17 mi)

Andrew E. Place, MD, PhD - Dana-Farber ...

Overseen byAndrew Place, MD, PhD

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Dana-Farber Cancer Institute

No Placebo Group

Trial Summary

What is the purpose of this trial?This research study involves participants who have acute lymphoblastic or acute myelogenous leukemia that has relapsed or has become resistant (or refractory) to standard therapies. This research study is evaluating a drug called KPT-330. Laboratory and other studies suggest that the study drug, KPT-330, may prevent leukemia cells from growing and may lead to the destruction of leukemia cells. It is thought that KPT-330 activates cellular processes that increase the death of leukemia cells. The main goal of this study is to evaluate the side effects of KPT-330 when it is administered to children and adolescents with relapsed or refractory leukemia.

Eligibility Criteria

This trial is for children and adolescents aged between 1 and 21 years with relapsed or refractory acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML), including other specific types of leukemia. Participants must have recovered from previous treatments, not be pregnant, agree to use contraception, and have no severe concurrent diseases.

Inclusion Criteria

My brain or spinal cord disease is at a manageable stage.

I agree not to breastfeed while participating in this study.

I (or my guardian) can understand and sign the consent form.

My condition did not improve after two initial treatments.

My leukemia has returned or is not responding to treatment.

My cancer has returned after treatment more than once.

I am between 1 and 21 years old.

I am mostly active and can do most of my daily activities.

Exclusion Criteria

I do not plan to receive any non-study cancer treatments during the trial.

I am eligible for a stem cell transplant from a donor.

I cannot take or tolerate medications by mouth.

My leukemia has spread to my brain.

My body does not respond to blood or platelet transfusions.

Treatment Details

The study tests KPT-330's effectiveness on participants with certain types of leukemia that haven't responded to standard therapies. The focus is on understanding the side effects when given to young patients. It explores how KPT-330 might help in destroying cancer cells by activating cell death processes.

1Treatment groups

Experimental Treatment

Group I: KPT-330Experimental Treatment1 Intervention

KPT-330 will be administered twice a week on Days 1 and 3 for four weeks. Starting dose 30 mg/m2.In the dose-escalation cohort, three patients will initially be enrolled at each dose level and will be monitored for a DLT during the 28-day treatment cycle before dose escalation may occur.