Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Rapamycin in Relapsed Acute Lymphoblastic Leukemia

This study is ongoing, but not recruiting participants.
Brigham and Women's Hospital
Children's Hospital Boston
Information provided by (Responsible Party):
Lewis B. Silverman, M.D., Dana-Farber Cancer Institute Identifier:
First received: April 1, 2009
Last updated: February 10, 2015
Last verified: February 2015

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.

Condition Intervention
Acute Lymphoblastic Leukemia
Drug: Corticosteroid
Drug: Rapamycin

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Parallel Assignment
Masking: Open Label
Official Title: Rapamycin in Relapsed Acute Lymphoblastic Leukemia

Resource links provided by NLM:

Further study details as provided by Dana-Farber Cancer Institute:

Primary Outcome Measures:
  • Compare changes in gene expression signatures in steroid alone versus steroid plus rapamycin treated patients. [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Compare biologic measures of "pro-apoptotic state" in steroid alone versus steroid plus rapamycin treated patients [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Estimated Enrollment: 14
Study Start Date: July 2007
Estimated Study Completion Date: October 2015
Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Steroid Only
Corticosteroid Alone
Drug: 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: Steroid plus Rapamycin
Corticosteroid plus Rapamycin
Drug: 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
Drug: Rapamycin
Taken orally mixed with water or orange juice
Other Name: sirolimus

Detailed 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.

Ages Eligible for Study:   365 Days and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00874562

United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Sponsors and Collaborators
Dana-Farber Cancer Institute
Brigham and Women's Hospital
Children's Hospital Boston
Principal Investigator: Lewis Silverman, MD Dana-Farber Cancer Institute
  More Information

No publications provided

Responsible Party: Lewis B. Silverman, M.D., Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT00874562     History of Changes
Other Study ID Numbers: 06-429
Study First Received: April 1, 2009
Last Updated: February 10, 2015
Health Authority: United States: Institutional Review Board

Keywords provided by Dana-Farber Cancer Institute:

Additional relevant MeSH terms:
Leukemia, Lymphoid
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Immune System Diseases
Immunoproliferative Disorders
Lymphatic Diseases
Lymphoproliferative Disorders
Neoplasms by Histologic Type
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses processed this record on March 03, 2015