MedTrace Logo

Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia

NCT00258427 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2021-12-02

Study results available
· View outcomes & findings →

Summary

RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Giving combination chemotherapy before a donor stem cell transplant may make the transplant more likely to work. This may be an effective treatment for patients with high risk Fanconi's anemia.

PURPOSE: This clinical trial is studying how well combination chemotherapy works in treating high risk patients who are undergoing a donor stem cell transplant for Fanconi's anemia.

Conditions

  • Fanconi Anemia

Interventions

BIOLOGICAL

anti-thymocyte globulin

Given 15 mg/kg/day intravenously every 12 hours on Days -5 through -1.

BIOLOGICAL

filgrastim

given 5 mcg/kg/day intravenously on Day 1 (continue until absolute neutrophil count (ANC) ≥2.5 x 10\^9/L)

DRUG

busulfan

Busulfan 0.8 mg/kg intravenously (IV) every 12 hours on Days -7 and -6 (1.0 mg/kg IV if \<4 years old)

DRUG

cyclophosphamide

10 mg/kg intravenously (IV) on Days -5 through -2.

DRUG

fludarabine phosphate

35 mg/m\^2 intravenously (IV) on Days -5 through -2.

DRUG

methylprednisolone

1 mg/kg intravenously (IV) every 12 hours on Days -5 through -1.

BIOLOGICAL

Hematopoietic stem cell transplantation

Infused on Day 0 - Donor bone marrow or umbilical cord blood will be collected in the usual sterile manner using established parameters determined by the National Marrow Donor Program.

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Margaret MacMillan, MD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
44 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2002-03-26
Primary Completion
2020-10-10
Completion
2020-10-10

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

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 NCT00258427 on ClinicalTrials.gov