Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

NCT01316549 | Completed FDA Drug

• 2 other identifiers: P003719210081
• Study Type: observational
• Target: 67
• Locations: 1 country
• Sites: 1

Brief Summary

Fludarabine is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of 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 genetic factors cause changes in fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

Conditions

Hematologic Malignancies; Nonmalignant Diseases; Immunodeficiencies; Hemoglobinopathies; Genetic Inborn Errors of Metabolism; Fanconi Anemia; Thalassemia; Sickle Cell Disease

Keywords

fludarabine; pharmacokinetics; pediatric; allogeneic; hematopoietic cell transplantation

Outcome Measures

Primary Outcomes (4)

• Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine for HCT in pediatric patients.

  2hours post start of infusion

• Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine for HCT in pediatric patients.

  3hours post start of infusion

• Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine for HCT in pediatric patients.

  6hours post start of infusion

• Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine for HCT in pediatric patients.

  24hours post start of infusion

Secondary Outcomes (3)

• Event free survival according to the AUC of fludarabine

  1month post transplant

• Event free survival according to the AUC of fludarabine

  3months post transplant

• Event free survival according to the AUC of fludarabine

  1 year post transplant

Study Arms (1)

Inpatient Pediatric Bone Marrow Transplant Recipients

All patients enrolled in this study will be located on the inpatient pediatric bone marrow transplant unit at University of California, San Francisco Benioff Children's Hospital.

Drug: Fludarabine

Interventions

Fludarabine DRUG

Given as injection

Also known as: Fludara

Inpatient Pediatric Bone Marrow Transplant Recipients

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

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 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 fludarabine in a clinic or any other out-patient setting.

You may qualify if:

• Children 0-17 years of age
• Undergoing alloHCT for the treatment of malignant or nonmalignant disorder
• Receiving fludarabine-based preparative regimen

You may not qualify if:

• Any child 7-17 years of age unwilling to provide assent
• Parent or guardian unwilling to provide written consent

Sponsors & Collaborators

• University of California, San Francisco: lead
• Thrasher Research Fund: collaborator

Study Sites (1)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Related Publications (1)

• Ivaturi V, Dvorak CC, Chan D, Liu T, Cowan MJ, Wahlstrom J, Stricherz M, Jennissen C, Orchard PJ, Tolar J, Pai SY, Huang L, Aweeka F, Long-Boyle J. Pharmacokinetics and Model-Based Dosing to Optimize Fludarabine Therapy in Pediatric Hematopoietic Cell Transplant Recipients. Biol Blood Marrow Transplant. 2017 Oct;23(10):1701-1713. doi: 10.1016/j.bbmt.2017.06.021. Epub 2017 Jul 3.

  PMID: 28684371 DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

whole blood, DNA

MeSH Terms

Conditions

Hematologic Neoplasms; Immunologic Deficiency Syndromes; Hemoglobinopathies; Fanconi Anemia; Thalassemia; Anemia, Sickle Cell

Interventions

fludarabine; fludarabine phosphate

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Immune System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Anemia; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic

Study Officials

• Janel R Long-Boyle, PharmD, PhD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2011
• First Posted: March 16, 2011
• Study Start: January 1, 2011
• Primary Completion: April 30, 2016
• Study Completion: April 30, 2016
• Last Updated: October 4, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)