NCT00885833

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Feb 2007

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2007

Completed
2.2 years until next milestone

First Submitted

Initial submission to the registry

April 21, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 22, 2009

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2012

Completed
Last Updated

July 14, 2014

Status Verified

July 1, 2014

Enrollment Period

5.1 years

First QC Date

April 21, 2009

Last Update Submit

July 11, 2014

Conditions

Wiskott-Aldrich Syndrome

Keywords

Wiskott-Aldrich syndromeHSCT

Outcome Measures

Primary Outcomes (2)

  • To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for HSCT in WAS.

    Feb. 2007 to Jan. 2012

  • To evaluate the incidence and severity of toxicity and treatment related mortality.

    Feb. 2007 to Jan. 2012

Secondary Outcomes (3)

  • To evaluate overall and event free survival rate.

    Feb. 2007 to Jan. 2012

  • To evaluate acute and chronic graft versus host disease (GVHD).

    Feb. 2007 to Jan. 2012

  • To evaluate immunologic recovery after HSCT.

    Feb. 2007 to Jan. 2012

Study Arms (1)

Fludarabine

EXPERIMENTAL
Drug: Fludarabine, Busulfan, Thymoglobulin

Interventions

Fludarabine, Busulfan, ThymoglobulinDRUG

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)

Fludarabine

Eligibility Criteria

Age1 Year - 25 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of Wiskott-Aldrich syndrome with gene analysis.
  • Indicated for hematopoietic stem cell transplantation.
  • Age: no limitation.
  • Performance status: ECOG 0-2.
  • Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases:
  • Heart: a shortening fraction \> 30%, ejection fraction \> 45%.
  • Liver: total bilirubin \< 2 × upper limit of normal; ALT \< 3 × upper limit of normal.
  • Kidney: creatinine \<2 × normal or a creatinine clearance (GFR) \> 60 ml/min/1.73m2.
  • Patients must lack any active viral infections or active fungal infection.
  • Appropriate hematopoietic stem cell donor is available.
  • Patients (or one of parents if patients age \< 19) should sign informed consent.

You may not qualify if:

  • Pregnant or nursing women.
  • Malignant (except acute myeloid leukemia) or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
  • Psychiatric disorder that would preclude compliance.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Seoul National University Hospital

Seoul, Chongno-gu, 110-744, South Korea

Location

MeSH Terms

Conditions

Wiskott-Aldrich Syndrome

Interventions

fludarabineBusulfanthymoglobulin

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesLymphopeniaLeukopeniaCytopeniaHemorrhagic DisordersLeukocyte DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-LinkedPrimary Immunodeficiency DiseasesImmunologic Deficiency SyndromesImmune System Diseases

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur Compounds

Study Officials

  • Hyo Seop Ahn, Ph. D

    The Korean Society of Pediatric Hematology Oncology

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NETWORK

Study Record Dates

First Submitted

April 21, 2009

First Posted

April 22, 2009

Study Start

February 1, 2007

Primary Completion

March 1, 2012

Study Completion

March 1, 2012

Last Updated

July 14, 2014

Record last verified: 2014-07

Locations

KR(1)