Trial of Sirolimus and Methotrexate in Relapsed/Refractory Lymphoblastic Leukemia and Lymphoma

Sponsored by Children's Hospital of Philadelphia

Phase Quota
Phase 2

This is a phase 2 study looking at efficacy and toxicity of oral sirolimus in combination with oral methotrexate in children with refractory/relapsed ALL or NHL. Secondary objectives include characterizing the trough levels produced by administration of oral sirolimus in children with refractory/relapsed ALL/NHL and to evaluate the effect of sirolimus on intracellular targets related to mTOR inhibition.

Study Start Date: May 2010

Estimated Completion Date: December 2017

Specialties: Internal Medicine: Hematology/Oncology Oncology: Leukemia/Lymphoma,Pharmacology/Therapy Pharmacy: Chemotherapy/Oncology


  • Drug: Sirolimus and Methotrexate

Inclusion criteria

  • Patients </= 25 years of age, at time of enrollment, with second or greater relapse of ALL or NHL. For ALL must have histologic diagnosis with >10% blasts in the marrow and for lymphoblastic lymphoma or peripheral T-cell lymphoma must have radiologic or physical evidence of recurrence
  • Lansky > 50% or Karnofsky > 50%
  • Negative Pregnancy Test
  • Creatinine clearance or radioisotope GFR > 70ml/min/m2 OR serum creatinine based on age /gender
  • Pulse ox >94%
  • Total Bilirubin <1.5 x normal for age
  • ALT < 5 x normal for age
  • Albumin > 2g/dL
  • Shortening fraction by echo > 28% OR ejection fraction > 50% by gated radionuclide study

Exclusion criteria

  • Patient has known allergies to sirolimus,FK-506 or mTOR inhibitors
  • Patient is taking other investigational anti-neoplastic drugs
  • Patient received no myelosuppressive chemo within 14 days
  • < 14 days have elapsed since local palliative XRT (small port) < 28 days since prior craniospinal XRT or 50% radiation of pelvis <28 days if other substantial BM radiation
  • Hematopoietic growth factors within 7 days of entry (except erythropoietin.)
  • Patient has taken any biologic agents within 14 days
  • Post BMT/SCT - evidence of active GVHD, at least > 3 months must have elapsed
  • Patient has uncontrolled infection (if patients with fungal disease, stable for < 14 days and patients with bacteremia without negative blood culture
  • Existing non-hematologic toxicities > grade 2
    • Use of steroids or hydroxyurea is permitted upto 14 days prior to entry

Study Locations And Contact Information

  • The Childrens Hospital of Philadelphia, Philadelphia Pennsylvania
    Contact: Susan R Rheingold MD 215-590-3079
  • The Childrens Hospital of Philadelphia, Philadelphia Pennsylvania

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