Rapamycin in Relapsed Acute Lymphoblastic Leukemia

NCT00874562 | Completed Phase 1 DMC

• 1 other identifier: 06-429
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Acute Lymphoblastic Leukemia

Keywords

rapamycin; corticosteroid; ALL

Outcome Measures

Primary Outcomes (2)

• Compare changes in gene expression signatures in steroid alone versus steroid plus rapamycin treated patients.

  3 years

• Compare biologic measures of "pro-apoptotic state" in steroid alone versus steroid plus rapamycin treated patients

  3 years

Secondary Outcomes (1)

• Assess clinical response by percent reduction of peripheral leukemia blasts at day 2 of therapy and at completion of the 5-day investigational window

  3 years

Study Arms (2)

Steroid Only

ACTIVE COMPARATOR

Corticosteroid Alone

Drug: Corticosteroid

Steroid plus Rapamycin

ACTIVE COMPARATOR

Corticosteroid plus Rapamycin

Drug: Corticosteroid; Drug: Rapamycin

Interventions

Corticosteroid DRUG

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

Steroid Only; Steroid plus Rapamycin

Rapamycin DRUG

Taken orally mixed with water or orange juice

Also known as: sirolimus

Steroid plus Rapamycin

Eligibility Criteria

• Age: 365 Days+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented acute lymphoblastic leukemia (L1 or L2 subtypes)
• First or subsequent relapse
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead
• Brigham and Women's Hospital: collaborator
• Boston Children's Hospital: collaborator
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Interventions

Adrenal Cortex Hormones; Sirolimus

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Macrolides; Lactones; Organic Chemicals

Study Officials

• Lewis Silverman, MD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: April 1, 2009
• First Posted: April 2, 2009
• Study Start: July 1, 2007
• Primary Completion: November 1, 2009
• Study Completion: April 1, 2016
• Last Updated: November 23, 2016

Record last verified: 2016-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)