Immune Ablation and Stem Cell Support for Crohn's Disease

This study has been withdrawn prior to enrollment.
Sponsor:
Information provided by:
Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier:
NCT00179842
First received: September 10, 2005
Last updated: January 27, 2009
Last verified: January 2009
  Purpose

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 Intervention Phase
Crohn Disease
Procedure: Stem Cell Transplant
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Immune Ablation and Hematopoietic Stem Cell Support in Patients With Severe Crohn's Disease

Resource links provided by NLM:


Further study details as provided by Ann & Robert H Lurie Children's Hospital of Chicago:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 25
Estimated Study Completion Date: December 2008
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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00179842

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

No publications provided

Responsible Party: Dr. Morris Kletzel, Children's Memorial Hospital
ClinicalTrials.gov Identifier: NCT00179842     History of Changes
Other Study ID Numbers: BMT 1202
Study First Received: September 10, 2005
Last Updated: January 27, 2009
Health Authority: United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on August 21, 2014