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Autologous Hematopoietic Stem Cell Transplantation for Refractory Autoimmune Diseases

NCT ID: NCT00742300

Last Updated: 2008-11-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

1998-01-31

Brief Summary

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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

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Conditions

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Autoimmune Diseases

Keywords

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ASCT Tolerance induction SLE Transplantation Autoimmune diseases

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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A

Treatment Group

Group Type EXPERIMENTAL

Autologous hematopoietic stem cell transplantation

Intervention Type PROCEDURE

Transplantation of CD34-selected autologous hematopoietic stem cells after high-dose chemotherapy with cyclophosphamide (200mg/kg) and rabbit-antithymocyteglobulin (90mg/kg)

Interventions

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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)

Intervention Type PROCEDURE

Other Intervention Names

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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)

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Charite University, Berlin, Germany

OTHER

Sponsor Role lead

Responsible Party

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Charité Universitätsmedizin Berlin

Principal Investigators

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Renate Arnold, Prof. Dr. med.

Role: PRINCIPAL_INVESTIGATOR

Charite University, Berlin, Germany

Falk Hiepe, Prof. Dr. med.

Role: STUDY_CHAIR

Charite University, Berlin, Germany

Locations

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Charité Universitätsmedizin Berlin

Berlin, , Germany

Site Status

Countries

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Germany

References

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Rosen O, Thiel A, Massenkeil G, Hiepe F, Haupl T, Radtke H, Burmester GR, Gromnica-Ihle E, Radbruch A, Arnold R. Autologous stem-cell transplantation in refractory autoimmune diseases after in vivo immunoablation and ex vivo depletion of mononuclear cells. Arthritis Res. 2000;2(4):327-36. doi: 10.1186/ar107. Epub 2000 Jun 8.

Reference Type RESULT
PMID: 11056673 (View on PubMed)

Jayne D, Passweg J, Marmont A, Farge D, Zhao X, Arnold R, Hiepe F, Lisukov I, Musso M, Ou-Yang J, Marsh J, Wulffraat N, Besalduch J, Bingham SJ, Emery P, Brune M, Fassas A, Faulkner L, Ferster A, Fiehn C, Fouillard L, Geromin A, Greinix H, Rabusin M, Saccardi R, Schneider P, Zintl F, Gratwohl A, Tyndall A; European Group for Blood and Marrow Transplantation; European League Against Rheumatism Registry. Autologous stem cell transplantation for systemic lupus erythematosus. Lupus. 2004;13(3):168-76. doi: 10.1191/0961203304lu525oa.

Reference Type RESULT
PMID: 15119545 (View on PubMed)

Rosen O, Hiepe F, Massenkeil G, Thiel A, Arnold R. Relapse of systemic lupus erythematosus. Lancet. 2001 Mar 10;357(9258):807-8. doi: 10.1016/S0140-6736(05)71239-3. No abstract available.

Reference Type RESULT
PMID: 11254005 (View on PubMed)

Thiel A, Alexander T, Schmidt CA, Przybylski GK, Kimmig S, Kohler S, Radtke H, Gromnica-Ihle E, Massenkeil G, Radbruch A, Arnold R, Hiepe F. Direct assessment of thymic reactivation after autologous stem cell transplantation. Acta Haematol. 2008;119(1):22-7. doi: 10.1159/000117824. Epub 2008 Feb 22.

Reference Type RESULT
PMID: 18292651 (View on PubMed)

Rosen O, Massenkeil G, Hiepe F, Pest S, Hauptmann S, Radtke H, Burmester G, Thiel A, Radbruch A, Heymer B, Arnold R. Cardiac death after autologous stem cell transplantation (ASCT) for treatment of systemic sclerosis (SSc): no evidence for cyclophosphamide-induced cardiomyopathy. Bone Marrow Transplant. 2001 Mar;27(6):657-8. doi: 10.1038/sj.bmt.1702829.

Reference Type RESULT
PMID: 11319598 (View on PubMed)

Alexander T, Thiel A, Rosen O, Massenkeil G, Sattler A, Kohler S, Mei H, Radtke H, Gromnica-Ihle E, Burmester GR, Arnold R, Radbruch A, Hiepe F. Depletion of autoreactive immunologic memory followed by autologous hematopoietic stem cell transplantation in patients with refractory SLE induces long-term remission through de novo generation of a juvenile and tolerant immune system. Blood. 2009 Jan 1;113(1):214-23. doi: 10.1182/blood-2008-07-168286. Epub 2008 Sep 29.

Reference Type DERIVED
PMID: 18824594 (View on PubMed)

Other Identifiers

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CT-0198

Identifier Type: -

Identifier Source: org_study_id