Rapamycin for Immunosuppression and B Cell Modulation Post Stem Cell Transplant for Acute Lymphoblastic Leukemia (ALL)
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|ClinicalTrials.gov Identifier: NCT00795886|
Recruitment Status : Completed
First Posted : November 21, 2008
Results First Posted : May 7, 2012
Last Update Posted : July 31, 2013
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
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||63 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
U.S. FDA Resources
|Experimental: All participants||
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)
- 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.
- 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.
- 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.
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 ClinicalTrials.gov identifier (NCT number): NCT00795886
|United States, Utah|
|Primary Children's Medical Center|
|Salt Lake City, Utah, United States, 84112|
|Principal Investigator:||Michael Pulsipher, MD||Primary Children's Hospital|