Autologous Hematopoietic Stem Cell Transplantation for Refractory Autoimmune Diseases

Phase 1

• Conditions: Autoimmune Diseases
• Interventions: Procedure: Autologous hematopoietic stem cell transplantation

• Registration Number: NCT00742300
• Lead Sponsor: Charite University, Berlin, Germany

Brief Summary

While glucocorticoids and immunosuppressants ameliorate manifestations of autoimmune diseases in many patients, current therapies are insufficient to control the disease in a subset of patients, and their clinical prognosis remains poor due to the development of vital organ failure, cumulative drug toxicity and to the increased risk of cardiovascular disease and malignancy. Immunoablative chemotherapy followed by autologous hematopoietic stem cell transplantation (ASCT) has recently emerged as a promising experimental therapy for severely affected patients, providing them the potential to achieve treatment-free, long-term remission. The rationale for applying ASCT to autoimmune diseases has been the hope that immunoablation could eliminate inflammation-driving pathogenic cells from the immune system, and that regeneration of the patients' immune system from hematopoietic precursors could re-establish immunological tolerance.

Detailed Description

Not available

Study Timeline

• Study Start: 1998-01
• Study First Submitted: 2008-08-26
• Study First Submitted QC: 2008-08-26
• Study First Posted: 2008-08-27
• Status Verified: 2008-11
• Last Update Submitted: 2008-11-21
• Last Update Posted: 2008-11-24

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Autoimmune disease
• Active disease with inadequate response to standard protocols (glucocorticoids and at least two different regimens of immunosuppressive drugs, such as intravenous cyclophosphamide 800-1000mg/application)
• Provision of informed consent by subject

Exclusion Criteria

• Active or chronic infections
• Uncontrolled arrhythmia or congestive heart failure (ejection fraction below 50% determined by echocardiogram)
• Lung fibrosis (transfer factor for carbon monoxide [TLCO] <45%)
• renal insufficiency (glomerular filtration rate below 40 ml/min)
• Pulmonary arterial hypertension (>40mmHg)
• History of malignancy
• Women who are pregnant or breastfeeding
• Use non-reliable methods of contraception

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
A	Autologous hematopoietic stem cell transplantation	Treatment Group

Primary Outcome Measures

Name	Time	Method
Disease-free survival	24 months
Overall Survival	24 months

Secondary Outcome Measures

Name	Time	Method
Immune Reconstitution	over 24 months
Organ-specific response parameters	24 months
Serological Response (Autoantibodies)	24 months

Trial Locations

Locations (1)

Charité Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany