This trial is active, not recruiting.

Condition autoimmune diseases
Treatment autologous hematopoietic stem cell transplantation
Phase phase 1/phase 2
Sponsor Charite University, Berlin, Germany
Start date January 1998
Trial identifier NCT00742300, CT-0198


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Treatment Group
autologous hematopoietic stem cell transplantation Mobilization: 2.0 g/m2 Cyclophosphamide followed by daily G-CSF (10 µg/kg, Amgen, Thousand Oaks, CA)
Transplantation of CD34-selected autologous hematopoietic stem cells after high-dose chemotherapy with cyclophosphamide (200mg/kg) and rabbit-antithymocyteglobulin (90mg/kg)

Primary Outcomes

Disease-free survival
time frame: 24 months
Overall Survival
time frame: 24 months

Secondary Outcomes

Immune Reconstitution
time frame: over 24 months
Organ-specific response parameters
time frame: 24 months
Serological Response (Autoantibodies)
time frame: 24 months

Eligibility Criteria

Male or female participants from 18 years up to 60 years old.

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

Additional Information

Official title Phase I/II Open-Label Monocentric Clinical Trial for Induction of Tolerance With CD34-Enriched Autologous Hematopoietic Stem Cell Transplantation After High-Dose Chemotherapy With Cyclophosphamide and Rabbit-Antithymocyte Globulin for Refractory Autoimmune Diseases
Principal investigator Renate Arnold, Prof. Dr. med.
Trial information was received from ClinicalTrials.gov and was last updated in November 2008.
Information provided to ClinicalTrials.gov by Charite University, Berlin, Germany.