Immune Ablation and Stem Cell Support for Crohn's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00179842
Recruitment Status : Withdrawn
First Posted : September 16, 2005
Last Update Posted : September 23, 2015
Information provided by:
Ann & Robert H Lurie Children's Hospital of Chicago

Brief Summary:
The investigators anticipate that this study will also form the basis to clarify further the role of the immune system in Crohn's disease.

Condition or disease Intervention/treatment Phase
Crohn Disease Procedure: Stem Cell Transplant Phase 2

Detailed Description:
This study is designed to ablate an aberrant immune system and then, similar to the use of marrow transplants for immunodeficient patients, reconstitute a new immune system with lymphocyte depleted stem cells. Subsequent disease activity will be followed by (1) Crohn's disease activity index (CDAI), (2) quality of life instrument (IBDQ), (3) type and amount of therapy for CD, and (4) flow cytometry of peripheral blood lymphocyte subsets. In addition, the subjects will undergo periodic absorption function testing, to assess small intestinal function. We anticipate that this study will also form the basis to clarify further the role of the immune system in Crohn's disease.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Immune Ablation and Hematopoietic Stem Cell Support in Patients With Severe Crohn's Disease
Study Completion Date : December 2008

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Crohn's Disease

Primary Outcome Measures :
  1. To investigate the safety and efficacy of treating severe CD with high dose cyclophosphamide and antithymocyte globulin rescued with T cell depleted autologous stem cells. Data will be used to evaluate the efficiency of a future phase III protocol. [ Time Frame: Minimum of 2 years ]

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Less than physiologic age 60 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 to have a sustained decrement of 70 points in the CDAI after one course of anti-TNF alpha, Infliximad. The patient is being considered for therapy with cyclosporin A (CSA) or surgical resection. The CDAI (appendix A) is 250-400.
  • Pre-study peripheral blood counts must include a platelet count greater than 100,000/ul and an absolute neutrophil count greater than 1500/ul.
  • Stem cell harvest greater than 1.4 x 106 CD34 cells/kg after CD34+ selection (to continue to transplant.)

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 I/FVC < 50% of predicted, DLCO < 50% of predicted.
  • Resting LVEF < 40%
  • Bilirubin > 2.0 mg/dl, transferase (AST) > 2x upper limit of normal, unless the abnormalities are secondary to Crohn's disease.
  • 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.

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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00179842

United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Principal Investigator: Morris Kletzel, M.D. Ann & Robert H Lurie Children's Hospital of Chicago

Responsible Party: Dr. Morris Kletzel, Children's Memorial Hospital Identifier: NCT00179842     History of Changes
Other Study ID Numbers: BMT 1202
First Posted: September 16, 2005    Key Record Dates
Last Update Posted: September 23, 2015
Last Verified: January 2009

Additional relevant MeSH terms:
Crohn Disease
Inflammatory Bowel Diseases
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases