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Autologous Stem Cell Transplantation for Refractory Systemic Lupus Erythematosus (ASSIST) (ASSIST)

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified August 2009 by Charite University, Berlin, Germany.
Recruitment status was:  Recruiting
Information provided by:
Charite University, Berlin, Germany Identifier:
First received: September 10, 2008
Last updated: July 20, 2011
Last verified: August 2009
While glucocorticoids and immunosuppressants ameliorate manifestations of SLE 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 investigators postulate that immunoablative therapy eliminates or effectively reduces the level of autoreactive T and B lymphocytes and then regeneration of de novo immunity resets the autoreactive immune system into a self-tolerant, protective immune system resulting in prolonged and treatment-free remission.

Condition Intervention Phase
Systemic Lupus Erythematosus
Procedure: Immunoablation and Autologous Hematopoietic Stem Cell Transplantation
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Phase II Multicenter Cohort Study of Immunoablation With Cyclophosphamide and Antithymocyte-Globulin and Transplantation of Autologous Cd34-Enriched Hemapoietic Stem Cells Versus Currently Available Immunosuppressive/Immunomodulatory Therapy for Treatment of Refractory Systemic Lupus Erythematosus

Resource links provided by NLM:

Further study details as provided by Charite University, Berlin, Germany:

Primary Outcome Measures:
  • SLEDAI [ Time Frame: 48 months ]

Secondary Outcome Measures:
  • Serologic response (autoantibodies) [ Time Frame: 48 months ]
  • Immune Reconstitution [ Time Frame: 48 months ]
  • Organ-specific response parameters [ Time Frame: 48 months ]

Estimated Enrollment: 30
Study Start Date: August 2008
Estimated Study Completion Date: August 2014
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Immunoablation and Autologous Hematopoietic Stem Cell Transplantation
Procedure: Immunoablation and Autologous Hematopoietic Stem Cell Transplantation
Transplantation of purified CD34+ autologous hematopoietic stem cells mobilized with cyclophosphamide (200mg/m2)and G-CSF (10µg/kg/d) after immunoablation with cyclophosphamide (200mg/kg)and rabbit-antithymocyteglobulin (90mg/kg)
Active Comparator: 2
Best currently available immunosuppressive/immunomodulatory therapy
Procedure: Immunoablation and Autologous Hematopoietic Stem Cell Transplantation
Transplantation of purified CD34+ autologous hematopoietic stem cells mobilized with cyclophosphamide (200mg/m2)and G-CSF (10µg/kg/d) after immunoablation with cyclophosphamide (200mg/kg)and rabbit-antithymocyteglobulin (90mg/kg)


Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Diagnosis of SLE according to American College of Rheumatology (ACR) classification criteria
  2. Age between 18 and 60 years, inclusive
  3. Provision of informed consent
  4. Active disease, refractory to standard immunosuppressive therapy defined as:

    • BILAG level A and a SLEDAI-score of at least 10, despite treatment with high-dose corticosteroids and pulse intravenous CYC at doses of 500-1000mg/m2 for at least 6 months or mycophenolate mofetil (MMF) at doses of at least 2g -
    • Lupus nephritis with renal biopsy performed within one year prior to screening showing glomerulonephritis WHO class III or IV
    • Parenchymal disease of heart or lung
    • Neuropsychiatric lupus
    • Autoimmune cytopenia OR
    • recurrence of disease activity (defined as BILAG level A and a SLEDAI of at least 10) within one year after successful induction therapy with cyclophosphamide or MMF in the presence of an adequate maintenance therapy with either cyclophosphamide (at least 500mg/m2 monthly), mycophenolate mofetil (at least 2g daily), azathioprine (at least 1.5mg/kg/d), methotrexate (at least 15mg weekly), cyclosporine (at least 3mg/kg/d) in patients with persistent anti-dsDNA antibodies

Exclusion Criteria:

  1. Severe concomitant disease or organ damage

    • renal: renal insufficiency with glomerular filtration rate below 40ml/min
    • cardiac: congestive heart failure, LVEF < 40% determined by echocardiogram, uncontrolled arrhythmia
    • pulmonary: mean pulmonary arterial pressure >50mmHg, DLCO < 40 % predicted
    • gastrointestinal: liver cirrhosis; SGOT, SGPT greater than 2 x the upper limit of normal, unless due to active lupus
  2. Ongoing cancer or history of malignancy within 5 years of screening
  3. Women who are pregnant or breastfeeding or use non-reliable methods of contraception
  4. Subjects with active systemic infection
  5. Subjects with history of active viral infection within 6 months prior to screening, known HIV-infection or chronic Hepatitis B or Hepatitis C
  6. History of allergic reaction to cyclophosphamide, G-CSF or ATG
  7. Use of immunosuppressive agents for indications other than SLE
  8. Any comorbidity that in the opinion of the investigator would jeopardize the ability of the subject to tolerate therapy
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00750971

Universitätsmedizin Charité
Berlin, Germany, 10117
Universitätsklinik Düsseldorf
Düsseldorf, Germany, 40225
Universitätsklinikum Essen
Essen, Germany, 45239 Essen
Universitätsklinik Heidelberg
Heidelberg, Germany, 69120
Universitätsklinik Köln
Köln, Germany, 50937
Universitäsklinik Mainz
Mainz, Germany, 55101
Universitätsklinik Tübingen
Tübingen, Germany, 72026
Universitätsklinik Würzburg
Würzburg, Germany, 97070
Sponsors and Collaborators
Charite University, Berlin, Germany
Principal Investigator: Falk Hiepe, Prof Universitätsmedizin Charité
  More Information


Responsible Party: Charité Universitätsmedizin, Dept. of Rheumatology and Clinical Immunology Identifier: NCT00750971     History of Changes
Other Study ID Numbers: CT-1306 
Study First Received: September 10, 2008
Last Updated: July 20, 2011

Keywords provided by Charite University, Berlin, Germany:
Stem Cell Transplantation

Additional relevant MeSH terms:
Lupus Erythematosus, Systemic
Connective Tissue Diseases
Autoimmune Diseases
Immune System Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists processed this record on February 20, 2017