Trials Sponsors Conditions Drugs Pricing

Conditions

Autoimmune Diseases Dermatomyositis Juvenile Rheumatoid Arthritis Systemic Lupus Erythematosus Systemic Sclerosis

Keywords

arthritis & connective tissue diseasesdermatomyositisimmunologic disorders and infectious disordersjuvenile rheumatoid arthritisrare diseasesystemic lupus erythematosussystemic sclerosis

Outcome Measures

Primary Outcomes (1)

Mortality
Annually for 5 years and then every 5 years thereafter

Secondary Outcomes (1)

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

Study Arms (1)

1

EXPERIMENTAL

Participants will receive a stem cell transplant.

Procedure: Stem Cell TransplantationDevice: CD34 selection

Interventions

Stem Cell TransplantationProcedure

Participants will receive a stem cell transplantation along with irradiation and the drugs anti-thymocyte globulin, cyclophosphamide, and filgrastim as noted in the text of this record.

CD34 selectionDevice

CD34+ cells are separated from the rest of the peripheral blood stem cells.

Eligibility Criteria

Age2 Years - 18 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Diagnosis of 1 of the following based on American College of Rheumatology (ACR) Criteria: Severe juvenile rheumatoid arthritis (systemic onset or polyarticular course) Juvenile systemic lupus erythematosus Systemic sclerosis Dermatomyositis * Refractory to standard or aggressive therapy OR unacceptable toxicity from standard therapy * Reasonable expectation of possible improvement as evidenced by a good potential for rehabilitation therapy and adequate social factors * No serious CNS damage that would preclude significant functional recovery --Prior/Concurrent Therapy-- * Chemotherapy: At least 4 weeks since prior methotrexate or cyclophosphamide * Endocrine therapy: At least 4 weeks since prior intra-arterial steroids Juvenile rheumatoid arthritis patients should continue steroids without taper throughout mobilization and harvest of stem cells If receiving corticosteroids, must be continued without taper Other: * At least 4 weeks since prior anti-inflammatory agents such as non-steroidal anti-inflammatory drugs (NSAIDs) or sulfasalazine * At least 4 weeks since prior cyclosporine, tacrolimus, mycophenolate mofetil, azathioprine, penicillamine, or etanercept --Patient Characteristics-- * Life expectancy: At least 30 days * Hematopoietic: Absolute neutrophil count at least 1,000/mm3 OR Platelet count at least 100,000/mm3 No bone marrow aspirate or biopsy consistent with production defect (depletion of neutrophil precursors or megakaryocytes) No myelodysplasia * Hepatic: Bilirubin no greater than 2.5 mg/dL AST no greater than 300 U/L on two sequential tests No severe liver dysfunction within past month No active hepatitis A, B, or C * Renal: No end-stage glomerulonephritis or renal disease Creatinine clearance at least 40 mL/min * Cardiovascular: No uncontrolled malignant arrhythmia No New York Heart Association class III or IV congestive heart failure Ejection fraction at least 50% * Pulmonary: DLCO at least 45% (DLCO at least 70% for patients with pulmonary disease caused by documented processes other than primary autoimmune disorder, such as infectious pneumonia or aspiration pneumonia) No severe pulmonary hypertension (PAP greater than 50) without potential for significant improvement Other: * No medical or psychosocial reasons that would make hematopoietic stem cell collection intolerable * No increased anesthetic risks * No fever higher than 39 degrees C * No positive serology for toxoplasmosis * No active life threatening infection not responsive to therapy * No other disease or organ dysfunction that would limit survival * No known hypersensitivity to murine or equine proteins * No known primary immunodeficiency disease HIV negative

Sponsors & Collaborators

Fred Hutchinson Cancer Center
Otherlead

Study Sites (1)

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

MeSH Terms

Conditions

Scleroderma, SystemicLupus Erythematosus, SystemicDermatomyositisArthritis, JuvenileAutoimmune DiseasesArthritisConnective Tissue DiseasesImmune System DiseasesCommunicable DiseasesRare Diseases

Interventions

Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Skin and Connective Tissue DiseasesSkin DiseasesPolymyositis

Study Officials

Ann Woolfrey
Fred Hutchinson Cancer Center
STUDY CHAIR
Carol A. Wallace, MD
Fred Hutchinson Cancer Center
PRINCIPAL INVESTIGATOR

Enrollment

6 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

February 2, 2001

First Posted

February 2, 2001

Study Start

November 2000

Date type not reported

This date has not been verified as actual or estimated.

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (1)

Study Arms (1)

1

Interventions

Eligibility Criteria

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Intervention Hierarchy (Ancestors)

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (1)

Study Arms (1)

1

Interventions

Eligibility Criteria

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Intervention Hierarchy (Ancestors)

Source record

Locations