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Sickle Cell Disease Conditioning for Bone Marrow Transplant

NCT00968162 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2017-07-25

No results posted yet for this study

Summary

Most bone marrow transplants for children with sickle cell disease are performed using high doses of two chemotherapy agents: busulfan and cyclophosphamide for the pre-transplant conditioning. This approach produces cure in most cases (approximately 95%). It, however, has serious side effects, including seizures and infertility. The primary goal of this study is to determine how much we can lower the dosages of busulfan and cyclophosphamide by incorporating fludarabine, a safer chemotherapy agent, into conditioning. The secondary goal is to develop a better understanding of how bone marrow transplants cause neurologic problems like seizures.

Conditions

Interventions

DRUG

fludarabine

Conduct a pilot trial using a transplant protocol, in which the standard busulfan, cyclophosphamide and anti-thymocyte globulin conditioning regimen is modified by adding fludarabine, a highly immunosuppressive agent, in order to determine the feasibility of reducing the total dose of cyclophosphamide from its present standard of 200 mg/kg to 90 mg/kg and of busulfan from its present standard of 12.8 mg/kg (IV) to 6.4 mg/kg, using a four step dose de-escalation schema.

Sponsors & Collaborators

  • Emory University

    lead OTHER

Principal Investigators

  • John Horan, MD · Emory University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-02-28
Primary Completion
2014-03-31
Completion
2016-01-11

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00968162 on ClinicalTrials.gov