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| ID | Type | Description | Link |
|---|---|---|---|
| 19081 | Other Identifier | University of California, San Francisco |
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Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and individual factors cause changes in clofarabine and fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.
Fludarabine and clofarabine are nucleoside analogs with potent antitumor and immunosuppressive properties used in conditioning regimens of pediatric allogeneic hematopoietic cell transplantation (alloHCT) to promote stem cell engraftment.
This is a single-center, prospective, non-interventional pharmacokinetic (PK) study investigating the clinical pharmacology of combination nucleoside analogue therapy in 24 children undergoing alloHCT at University of California, San Francisco Benioff Children's Hospital.
Patients would receive clofarabine and fludarabine regardless of whether or not they decide to consent to PK sampling.
Clofarabine and fludarabine doses will not be adjusted based on PK data.
The investigators will apply the combination of a limited sampling strategy and population PK methodologies to determine specific factors influencing clofarabine and fludarabine exposure in pediatric alloHCT recipients and identify exposure-response relationships.
Subjects will undergo PK sampling of clofarabine and fludarabine drug concentrations over the duration of combination therapy (3 to 5 days).
To evaluate sources of variability impacting clofarabine and fludarabine exposure clinical data will be obtained from the patient's medical chart on each day of PK sampling.
To assess exposure-response relationships neutrophil engraftment, treatment-related toxicity, and survival data will be collected through day 100 post-transplant.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pediatric Bone Marrow Transplantation Recipients | Children undergoing alloHCT at UCSF Benioff Children's Hospital. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Clofarabine | Drug | Given IV |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine and clofarabine dual therapy for HCT in pediatric patients. | 2hours post start on infusion | |
| Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine and clofarabine dual therapy for HCT in pediatric patients. | 3hours post start of infusion | |
| Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine and clofarabine dual therapy for HCT in pediatric patients. | 6hours post start of infusion | |
| Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine and clofarabine dual therapy for HCT in pediatric patients. | 24hours post start of infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate the event free survival according to the AUC of fludarabine and clofarabine dual therapy | 1month post transplant | |
| Evaluate the event free survival according to the AUC of fludarabine and clofarabine dual therapy | 3 months post transplant |
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Inclusion Criteria:
Exclusion Criteria:
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The target population for the proposed study includes children 0-17 years of age undergoing alloHCT for the treatment of malignant and nonmalignant disorders. Patients receiving clofarabine and fludarabine over 3 to 5 days are eligible to participate. All patients enrolled in this study will undergo PK sampling on the inpatient pediatric BMT unit at UCSF Benioff Children's Hospital. The proposed research will not study any patients receiving clofarabine and fludarabine in a clinic or any other out-patient setting.
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| Name | Affiliation | Role |
|---|---|---|
| Janel Long-Boyle, PharmD, PhD | University of California, San Francisco | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, San Francisco | San Francisco | California | 94143 | United States |
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Plasma
| Fludarabine Injection |
| Drug |
Given IV |
|
| Evaluate the event free survival according to the AUC of fludarabine and clofarabine dual therapy | 1 year post transplant |
| ID | Term |
|---|---|
| D019337 | Hematologic Neoplasms |
| D007153 | Immunologic Deficiency Syndromes |
| D006453 | Hemoglobinopathies |
| D005199 | Fanconi Anemia |
| D013789 | Thalassemia |
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007154 | Immune System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D029502 | Anemia, Hypoplastic, Congenital |
| D000741 | Anemia, Aplastic |
| D000740 | Anemia |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
| D049914 | DNA Repair-Deficiency Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
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| ID | Term |
|---|---|
| D000077866 | Clofarabine |
| C024352 | fludarabine |
| ID | Term |
|---|---|
| D000227 | Adenine Nucleotides |
| D011685 | Purine Nucleotides |
| D011687 | Purines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
| D001087 | Arabinonucleosides |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D009711 | Nucleotides |
| D012265 | Ribonucleotides |
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