MedTrace Logo

Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)

NCT00885833 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2014-07-14

No results posted yet for this study

Summary

Wiskott-Aldrich syndrome (WAS) is a rare X-linked congenital immune-deficiency syndrome and hematopoietic stem cell transplantation (HSCT) has become a curative modality. But the transplant with the conventional conditioning resulted in high incidence of treatment related toxicities and non-myeloablative conditioning resulted in high incidence of engraftment failure. Recently, fludarabine based reduced toxicity myeloablative conditioning regimen was developed for adult myeloid malignancies with promising result of good engraftment and low treatment related toxicities. To increase the engraftment potential without serious complication, reduced toxicity myeloablative conditioning regimen composed of fludarabine, busulfan, and thymoglobulin is designed for Wiskott-Aldrich syndrome.

Conditions

  • Wiskott-Aldrich Syndrome

Interventions

DRUG

Fludarabine, Busulfan, Thymoglobulin

fludarabine (40 mg/m2 once daily i.v. on days -8, -7, -6, -5, -4 \& -3) busulfan (0.8 mg/kg every 6 hours i.v. on days -6, -5, -4, \& -3) thymoglobulin (2.5 mg/kg once daily i.v. on days -8, -7, -6 for cord blood and on days -4, -3, -2 for bone marrow or mobilized peripheral blood)

Sponsors & Collaborators

  • The Korean Society of Pediatric Hematology Oncology

    lead NETWORK

Principal Investigators

  • Hyo Seop Ahn, Ph. D · The Korean Society of Pediatric Hematology Oncology

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-02-28
Primary Completion
2012-03-31
Completion
2012-03-31

Countries

  • South Korea

Study Locations

More Related Trials

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 NCT00885833 on ClinicalTrials.gov