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Rapamycin for Immunosuppression and B Cell Modulation Post Stem Cell Transplant for Acute Lymphoblastic Leukemia (ALL)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00795886
Recruitment Status : Completed
First Posted : November 21, 2008
Results First Posted : May 7, 2012
Last Update Posted : July 31, 2013
Children's Hospital of Philadelphia
Information provided by (Responsible Party):
University of Utah

Brief Summary:


Primary objective: Evaluate toxicity of rapamycin when used for post-bone marrow transplant graft vs. host disease prophylaxis in children with acute lymphoblastic leukemia (ALL).

Investigator initiated; four participating institutions; Phase II pilot study

Condition or disease Intervention/treatment Phase
Acute Lymphoblastic Leukemia Drug: RAPAMYCIN Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 63 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: CHP-753, Rapamycin for Immunosuppression and B Cell Modulation Post Stem Cell Transplant for Acute Lymphoblastic Leukemia (ALL)
Study Start Date : August 2005
Actual Primary Completion Date : March 2010
Actual Study Completion Date : March 2010

Arm Intervention/treatment
Experimental: All participants Drug: RAPAMYCIN
Rapamycin (RAPA, RapamuneR) (sirolimus) is an immunosuppressive agent that was approved by the FDA in 1999. It is a macrocyclic lactone that is structurally similar to Tacrolimus (FK506) and binds to the same intracellular protein as FK506, FKBP1,2,3, but it has an entirely different mechanism of action and a different principal target protein. The target of the RAPA: FKBP complex is the mammalian target of rapamycin (mTOR). Unlike the calcineurin inhibitors cyclosporine (CSA) and - FK506, RAPA exerts its effects by inhibiting growth factor-driven transduction signals in the T-cell response to alloantigen, thus preventing proliferation among T and B lymphocytes3,4.
Other Name: (RAPA, RapamuneR) (sirolimus)

Primary Outcome Measures :
  1. Number of Participants With Transplant-related Mortality [ Time Frame: 24 months after transplant ]
    Death not associated with relapse. We determined that if transplant related mortality(TRM) 25% at day 100 or if Grade III-IV (severe, life threatening, or disabling) acute GVHD was >30% a stopping rule would be triggered.

  2. Two Year Overall Survival [ Time Frame: 24 months after transplant ]
    The probability that a given patient will be alive two years after transplantation. The Kaplan-Meier product limit method was used to compute the probability of overall survival to 2 years. Greenwood's formula was used to compute the standard error.

Secondary Outcome Measures :
  1. Percentage of Patients Developing Acute Graft vs. Host Disease (GVHD) [ Time Frame: 180 days ]
    Cumulative incidence of Grade 2-4 acute GVHD at 180 days.

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Pediatric patients' ages (0 - 21 years) with lymphoid malignancies considered for allogeneic bone marrow transplant from HLA-identical sibling donor, single antigen mismatched related or unrelated donor marrow /peripheral blood stem cell (PBSC) or cord blood available for marrow donation.

    First remission:

    • if remission not achieved by day28
    • high risk cytogenetic features, including t(9;22) or t(4;11) Second or third remission
  2. Signed informed consent.

Exclusion Criteria:

1. Organ criteria:

  1. Cardiac: ECHO shortening fraction <27%
  2. Renal: Creatinine clearance <60 ml/min/1.73 m2
  3. Hepatic: Bilirubin >1.5 mg/dl, transaminases <3x normal
  4. Infection: active viral, fungal or bacterial infection including HIV.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00795886

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United States, Utah
Primary Children's Medical Center
Salt Lake City, Utah, United States, 84112
Sponsors and Collaborators
University of Utah
Children's Hospital of Philadelphia
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Principal Investigator: Michael Pulsipher, MD Primary Children's Hospital

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Responsible Party: University of Utah Identifier: NCT00795886     History of Changes
Other Study ID Numbers: HCI14044
First Posted: November 21, 2008    Key Record Dates
Results First Posted: May 7, 2012
Last Update Posted: July 31, 2013
Last Verified: July 2013
Keywords provided by University of Utah:
Stem Cell Transplant
Additional relevant MeSH terms:
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Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs