Overview

This trial has been completed.

Condition acute lymphoblastic leukemia
Treatments corticosteroid, rapamycin
Phase phase 1
Targets mTOR, FKBP-12
Sponsor Dana-Farber Cancer Institute
Collaborator Brigham and Women's Hospital
Start date July 2007
End date November 2009
Trial size 6 participants
Trial identifier NCT00874562, 06-429

Summary

This is a research study designed to look at the biological effects of two drugs on leukemia cells. In this study, we are comparing the effects of drugs called corticosteroids when used alone or with another drug called rapamycin. Rapamycin is a drug that prevents the body's immune system from working normally. It has been used for many years after kidney transplants to prevent rejection of the organ. Recent work suggests that rapamycin may also help treat leukemia and other cancers.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification pharmacokinetics/dynamics study
Intervention model parallel assignment
Masking open label
Arm
(Active Comparator)
Corticosteroid Alone
corticosteroid
Initial dose of corticosteroid given intravenously. After the first dose, corticosteroid will be taken orally every 8 hours for the duration of the five-day period
(Active Comparator)
Corticosteroid plus Rapamycin
corticosteroid
Initial dose of corticosteroid given intravenously. After the first dose, corticosteroid will be taken orally every 8 hours for the duration of the five-day period
rapamycin sirolimus
Taken orally mixed with water or orange juice

Primary Outcomes

Measure
Compare changes in gene expression signatures in steroid alone versus steroid plus rapamycin treated patients.
time frame: 3 years
Compare biologic measures of "pro-apoptotic state" in steroid alone versus steroid plus rapamycin treated patients
time frame: 3 years

Secondary Outcomes

Measure
Assess clinical response by percent reduction of peripheral leukemia blasts at day 2 of therapy and at completion of the 5-day investigational window
time frame: 3 years

Eligibility Criteria

Male or female participants at least 365 days old.

Inclusion Criteria: - Documented acute lymphoblastic leukemia (L1 or L2 subtypes) - First or subsequent relapse - 365 days of age or older - Greater than 7 days from any chemotherapy or immunotherapy, with the exception of intrathecal chemotherapy - Absolute peripheral leukemia blast count of 1000 cells/ul or greater - Patient (or parent/guardian if patient is less than 18 years of age) must sign informed consent Exclusion Criteria: - Burkitts leukemia (acute lymphoblastic leukemia L3 subtype) - Uncontrolled active infection - Pregnancy or mothers who are nursing - Patient currently taking rapamycin - Patients with significant liver dysfunction as outlined in protocol - Severe concurrent disease, which in the judgment of the investigator, would make the patient inappropriate for entry into the study - Active psychiatric disease, substance abuse, or mental illness that would interfere with cooperation with the requirements of the trial

Additional Information

Official title Rapamycin in Relapsed Acute Lymphoblastic Leukemia
Principal investigator Lewis Silverman, MD
Description - Participants will be randomized into two groups; one group will receive corticosteroid alone, and the other group will receive rapamycin and corticosteroid. - The length of treatment will be 5 days, during which time we will collect blood samples to measure the biologic effects of these drugs. Because these drugs will be given for a short period of time only, this study is not designed to treat or cure the participants leukemia. After the 5-day period, participants may resume other cancer-directed therapies.
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Dana-Farber Cancer Institute.