for people ages 3 months to 30 years (full criteria)
at San Francisco, California
study started
estimated completion
Christopher C Dvorak, MD



The purpose of this study is to find out what effects, good and/or bad, the addition of clofarabine, a new chemotherapy agent, to a standard busulfan and fludarabine conditioning treatment has. The study will also look at what causes some people to have high drug levels of these medications in their body compared to other people that may have low drug levels even if they all receive the same dose of medication.

Official Title

A Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation


Myeloid Malignancy Bone Marrow Failure Syndrome Transfusion-dependent Red Blood Cell (RBC) Defect Congenital Immunodeficiency Metabolic Disease Severe Immune Dysregulation conditioning allogeneic hematopoietic cell transplantation HCT Immunologic Deficiency Syndromes Metabolic Diseases Pancytopenia Anemia, Aplastic Hemoglobinuria, Paroxysmal Fludarabine Fludarabine phosphate Busulfan Alemtuzumab Clofarabine Vidarabine


You can join if…

Open to people ages 3 months to 30 years

  • Patients must be ≥ 3 months and ≤30 years of age.
  • Stratum A: Non-Malignant Diseases, including:
  • Bone Marrow Failure Syndromes
  • Hemoglobinopathies or transfusion-dependent RBC defects
  • Congenital Immunodeficiencies
  • Metabolic Diseases known to be treatable with HCT (e.g. Hurler's)
  • Other Bone Marrow Stem Cell Defects (e.g. Osteopetrosis)
  • Severe Immune Dysregulation / Autoimmune Syndromes with at least transient prior response to immunosuppressive therapy
  • Stratum B: Myeloid Malignancies, including:
  • AML, in greater than first clinical remission, or in CR1 but with detectable disease (≥0.1% Blasts by MRD or Flow, or Positive Cytogenetics), or in CR1 but with a matched sibling UCB donor.
  • MDS
  • JMML
  • CML, with detectable disease by PCR
  • Patients must have a suitable donor based on the UCSF Pediatric BMT SOP. 10/10 (HLA-A, -B, -C, -DR, -DQ) matching will be done for related and adult unrelated donors; 8/8 (HLA-A, -B, -C, -DR) for umbilical cord blood donors. Patients with non-malignant diseases will generally be eligible only if they have a mismatched donor, or an accepted clinical reason to be considered high-risk for rejection.
  • Liver transaminases (AST/ALT) and Direct Bilirubin less than twice the upper limit of normal within 2 weeks of admission.
  • Cardiac Shortening Fraction ≥27% within 4 weeks of admission.
  • Creatinine clearance by Schwartz formula, GFR or 24 hr urine collection ≥50 cc/min/1.73 m2, within 4 weeks of admission.
  • Pulmonary diffusion capacity ≥50% of predicted corrected for anemia/lung volume within 4 weeks of admission. If unable to do PFT's, then no active lung disease by CXR and/or O2 Sat ≥90% on room air.

You CAN'T join if...

  • Fanconi Anemia
  • Dyskeratosis Congenita
  • A known syndrome with increased sensitivity to radiation or alkylating agents
  • Severe Combined Immunodeficiency Disease eligible for a non-myeloablative HCT trial
  • A mismatched donor for whom ex vivo T-cell depletion of the donor stem cells is planned


  • UCSF Comprehensive Cancer Center
    San Francisco California 94143 United States

Lead Scientist

  • Christopher C Dvorak, MD
    My Research Interests are divided into 3 areas of focus: 1. Supportive Care (especially Invasive Fungal Infections) following Hematopoietic Stem Cell Transplantation 2. Transplantation for Severe Combined Immunodeficiency 3.


currently not accepting new patients, but might later
Start Date
Completion Date
Christopher Dvorak
Study Type
Last Updated