Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation
| Status: | Suspended |
|---|---|
| Conditions: | HIV / AIDS, Endocrine, Hematology |
| Therapuetic Areas: | Endocrinology, Hematology, Immunology / Infectious Diseases |
| Healthy: | No |
| Age Range: | Any - 30 |
| Updated: | 10/13/2018 |
| Start Date: | July 2012 |
| End Date: | June 2019 |
A Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation
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.
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.
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.
- 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.
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
planned
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