Hematopoietic Stem Cell Support in Patients With Severe 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: NCT00278577
Recruitment Status : Terminated
First Posted : January 18, 2006
Last Update Posted : February 6, 2014
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Brief Summary:

This disease is believed to be caused by immune cells (called lymphocytes) attacking tissue. Risk of death is highest in people with active acute disease. In addition, progressive Crohn's Disease leads to further loss of bowel function, which may eventually result in the need for artificial nutritional support (parenteral nutrition).

This study involves high dose chemotherapy followed by return (infusion) of blood stem cells. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The high dose chemotherapy consists of cyclophosphamide and anti lymphocyte antibody (a protein that depletes cells that cause damage to the body). The purpose of the intense chemotherapy is to destroy the immune system completely. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and anti lymphocyte antibody.

Condition or disease Intervention/treatment Phase
CROHN'S DISEASE Biological: Immune Ablation and Hematopoietic Stem Cell Support Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 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 Start Date : April 2001
Actual Primary Completion Date : February 2012
Actual Study Completion Date : February 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Crohn's Disease

Arm Intervention/treatment
Experimental: Autologous Hematopoietic Stem Cell Transplant Biological: Immune Ablation and Hematopoietic Stem Cell Support
Autologous Hematopoietic Stem Cell Transplant

Primary Outcome Measures :
  1. 11.1 CDAI - If the index worsens by 50 points for more than 4 weeks, the disease will be considered progressive; if it improves by 70 points for more than four weeks, it will be considered improved; otherwise it will be considered stable. [ Time Frame: 5 years after transplant ]

Information from the National Library of Medicine

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

Inclusion Criteria:

  1. Less than physiologic age 60 at time of pretransplant evaluation
  2. 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) 250-400 or a Craig Severity Score that is > 17 (appendix D)
  3. Pre-study peripheral blood counts must include a platelet count greater than 100,000/ul and an absolute neutrophil count greater than 1500/ul.
  4. Stem cell harvest greater than 1.4 x 106 CD34 cells/kg after CD34+ selection (to continue to transplant)
  5. Ability to give informed consent

Exclusion Criteria

  1. HIV positive
  2. History of coronary artery disease, or congestive heart failure
  3. 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
  4. 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
  5. 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
  6. Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible
  7. FEV I/FVC < 50% of predicted, DLCO < 50% of predicted
  8. Resting LVEF < 40%
  9. Bilirubin > 2.0 mg/dl, transferase (AST) > 2x upper limit of normal, unless the abnormalities are secondary to Crohn's disease
  10. Serum creatinine > 2.0 mg/dl
  11. Platelet count less than 100,000/ul, ANC less than 1500/ul
  12. 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.

Information from the National Library of Medicine

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): NCT00278577

United States, Illinois
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Richard Burt, MD
Principal Investigator: Robert Craig, MD Northwestern University

Responsible Party: Richard Burt, MD, MD, Northwestern University Identifier: NCT00278577     History of Changes
Obsolete Identifiers: NCT00014703
Other Study ID Numbers: NU 97CD1
First Posted: January 18, 2006    Key Record Dates
Last Update Posted: February 6, 2014
Last Verified: April 2013

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