Crohn's Disease Stem Cell Transplantation - Full Text View

Sponsor:	Northwestern University
Information provided by (Responsible Party):	Northwestern University
ClinicalTrials.gov Identifier:	NCT00271947

Purpose

Crohn's disease (CD) is a chronic illness, immunologically mediated, probably induced by the exposure of the intestine to an antigen or antigens similar to the intestine, to which immunologic tolerance is lost or a dysregulated immunity ensues. The disease has a variable course, from a mild, intermittently active illness requiring only symptomatic therapy to a fulminant illness requiring potentially dangerous immunosuppressive therapy, surgery or both. The molecular defect causing CD has not been characterized, but probably involves aberrant T cell function. Although CD often responds to immunosuppressive medication including corticosteroids, azathioprine and 6-mercaptopurine, to anti inflammatory drugs such as 5 aminosalicylate (5 ASA), or to some antimicrobial agents, including metronidazole, no therapy has been curative. In patients with severe CD, who have been unresponsive to corticosteroids, azathioprine, 5 ASA, metronidazole, and infliximab, we propose to compare the efficacy of Crohn's disease non-myeloablative autologous hematopoietic stem cell transplantation (CDNST) to standard therapy. Subsequent disease activity will be followed by (1) Crohn's disease activity index (CDAI), (2) a more global severity index, the Crohn's Severity Index, (3) type and amount of therapy for CD, and (4) clinical, hematologic and biochemical studies.

Condition	Intervention	Phase
Crohn's Disease	Procedure: stem cell transplantation	Phase II

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Crohn's Disease Non-myeloablative Autologous Hematopoietic Stem Cell Transplantation (CDNST) Versus Standard Therapy

Resource links provided by NLM:

Genetics Home Reference related topics: Crohn disease

MedlinePlus related topics: Crohn's Disease

U.S. FDA Resources

Further study details as provided by Northwestern University:

Primary Outcome Measures:

CDAI [ Time Frame: Clinical remission defined as a CDAI less than 150 and clinical improvement defined as decline in CDI> or = 70 one year following entry. ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	110
Study Start Date:	April 2005
Estimated Study Completion Date:	April 2015
Estimated Primary Completion Date:	April 2013 (Final data collection date for primary outcome measure)

Intervention Details:

Autologous Hematopoietic Stem Cell Transplantation will be performed on all participants randomized to transplant arm.

Eligibility

Ages Eligible for Study:	18 Years to 55 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Age 18 years or older and less than age 55 years at time of pretransplant evaluation.
An established clinical diagnosis of severe CD that has failed therapy with prednisone, azathioprine, 5 ASA products and metronidazole, and has failed an anti-TNF alpha inhibitor. Failure is defined as a CDAI (appendix A) 225-400.
Pre-study peripheral blood counts must include a platelet count greater than 100,000/ul and an absolute neutrophil count greater than 1500/ul.
In those randomized to receive the transplant, a stem cell harvest greater than 2.0 x 106 CD34 cells/kg is required.
Ability to give informed consent.

Exclusion Criteria:

HIV positive.
History of coronary artery disease, or congestive heart failure.
Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as head and neck cancer, or stage I breast cancer will be considered on an individual basis.
Positive pregnancy test, lactation, inability or unwillingness to pursue effective means of birth control, failure to accept or comprehend irreversible sterility as a side effect of therapy.
Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
FEV 1/FVC < 50% of predicted, DLCO < 50% of predicted.
Resting LVEF < 40%.
Bilirubin > 2.0 mg/dl, transferase (AST) > 2x upper limit of normal.
Serum creatinine > 2.0 mg/dl.
Platelet count less than 100,000/ul, ANC less than 1500/ul.
Patients presenting with intestinal perforation or toxic megacolon, or a suppurative problem that will require urgent surgery. In addition, the patient may not have any active infection. The presence of intestinal stomas does not exclude the patient from study.
Splenomegaly (palpable spleen on physical exam).
Inability to collect > 2.0 x 106 CD34+ cells/kg.
Positive pregnancy test.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00271947

Locations

United States, Illinois
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States, 60611

Sponsors and Collaborators

Northwestern University

Investigators

Principal Investigator:

Robert Craig, MD

Northwestern University

More Information

No publications provided

Responsible Party:	Northwestern University
ClinicalTrials.gov Identifier:	NCT00271947 History of Changes
Other Study ID Numbers:	CD Randomized
Study First Received:	January 2, 2006
Last Updated:	August 19, 2011
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Crohn Disease
Inflammatory Bowel Diseases
Gastroenteritis

Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases

ClinicalTrials.gov processed this record on February 09, 2012