Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders

Phase 1

Completed

• Conditions: Autoimmune Diseases; Systemic Sclerosis; Dermatomyositis; Systemic Lupus Erythematosus; Juvenile Rheumatoid Arthritis
• Interventions: Procedure: Stem Cell Transplantation; Device: CD34 selection

• Registration Number: NCT00010335
• Lead Sponsor: Fred Hutchinson Cancer Center

Brief Summary

OBJECTIVES: I. Determine the safety and long term complications of total body irradiation in combination with cyclophosphamide, anti-thymocyte globulin, and autologous CD34-selected peripheral blood stem cell (PBSC) transplantation in children with refractory autoimmune disorders.

II. Determine the efficacy of this treatment regimen in these patients. III. Determine the reconstitution of immunity after autologous CD34-selected PBSC transplantation in these patients.

IV. Determine engraftment of autologous CD34-selected PBSC in these patients.

Detailed Description

PROTOCOL OUTLINE: This is a multicenter study. Patients receive filgrastim (G-CSF) subcutaneously daily until peripheral blood stem cell (PBSC) collection is completed. CD34+ cells are separated from the rest of the PBSCs.

Patients undergo total body irradiation twice daily on days -5 and -4. Patients receive anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5 and cyclophosphamide IV on days -3 and -2. CD34-selected PBSCs are reinfused on day 0. Patients receive G-CSF IV daily beginning on day 0 and continuing until blood counts recover.

Patients are followed annually for 5 years and then every 5 years thereafter.

Study Timeline

• Study Start: 2000-11
• Study First Submitted QC: 2001-02-01
• Study First Submitted: 2001-02-02
• Study First Posted: 2001-02-02
• Primary Completion: 2010-07
• Study Completion: 2011-05
• Status Verified: 2015-03
• Last Update Submitted: 2015-03-04
• Last Update Posted: 2015-03-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Stem Cell Transplantation	Participants will receive a stem cell transplant.
1	CD34 selection	Participants will receive a stem cell transplant.

Primary Outcome Measures

Name	Time	Method
Mortality	Annually for 5 years and then every 5 years thereafter

Secondary Outcome Measures

Name	Time	Method
Immune reconstitution, engraftment, efficacy, late-effects	Annually for 5 years and then every 5 years thereafter

Trial Locations

Locations (1)

Fred Hutchinson Cancer Research Center; 🇺🇸 Seattle, Washington, United States