Autologous Hematopoietic Stem Cell Transplantation for Refractory Autoimmune Diseases (ASTRAD)

November 21, 2008 updated by: Charite University, Berlin, Germany

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Berlin, Germany, 10117
        • Charite Universitatsmedizin Berlin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 60 years (ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: A
Treatment Group
Procedure: Autologous hematopoietic stem cell transplantation
Transplantation of CD34-selected autologous hematopoietic stem cells after high-dose chemotherapy with cyclophosphamide (200mg/kg) and rabbit-antithymocyteglobulin (90mg/kg)
Other Names:
  • Mobilization: 2.0 g/m2 Cyclophosphamide followed by daily G-CSF (10 µg/kg, Amgen, Thousand Oaks, CA)
  • Conditioning: 200mg/kg Cyclophosphamide (Endoxan), 90mg/kg rabbit-antithymocyteglobulin (ATG, Fresenius, Bad Homburg, Germany)
  • Stem cell selection: CliniMACS Device (Miltenyi Biotec, Bergisch Gladbach, Germany)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Overall Survival
Time Frame: 24 months
24 months
Disease-free survival
Time Frame: 24 months
24 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Immune Reconstitution
Time Frame: over 24 months
over 24 months
Organ-specific response parameters
Time Frame: 24 months
24 months
Serological Response (Autoantibodies)
Time Frame: 24 months
24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Charite University, Berlin, Germany

Investigators

  • Principal Investigator: Renate Arnold, Prof. Dr. med., Charite University, Berlin, Germany
  • Study Chair: Falk Hiepe, Prof. Dr. med., Charite University, Berlin, Germany

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 1998

Study Registration Dates

First Submitted

August 26, 2008

First Submitted That Met QC Criteria

August 26, 2008

First Posted (ESTIMATE)

August 27, 2008

Study Record Updates

Last Update Posted (ESTIMATE)

November 24, 2008

Last Update Submitted That Met QC Criteria

November 21, 2008

Last Verified

November 1, 2008

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CT-0198

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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