Know Cancer

forgot password

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

3 Months
30 Years
Open (Enrolling)
Myeloid Malignancy, Bone Marrow Failure Syndrome, Transfusion-dependent Red Blood Cell (RBC) Defect, Congenital Immunodeficiency, Metabolic Disease, Severe Immune Dysregulation

Thank you

Trial Information

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

Inclusion Criteria:

- 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.



- 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.

Exclusion Criteria:

- 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

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Outcome Time Frame:

Participants will be followed for the duration of treatment, an expected average of 5 years.

Safety Issue:


Principal Investigator

Christopher C Dvorak, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of California, San Francisco


United States: Institutional Review Board

Study ID:

UCSF Protocol No. 110819



Start Date:

July 2012

Completion Date:

June 2019

Related Keywords:

  • 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
  • Neoplasms
  • Immunologic Deficiency Syndromes
  • Metabolic Diseases
  • Pancytopenia
  • Hemoglobinuria, Paroxysmal



UCSF Comprehensive Cancer Center San Francisco, California  94115