Immune Ablation and Stem Cell Support for Crohn's Disease

September 21, 2015 updated by: Ann & Robert H Lurie Children's Hospital of Chicago

Immune Ablation and Hematopoietic Stem Cell Support in Patients With Severe Crohn's Disease

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

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Children's Memorial Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Minimum of 2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Completion

December 1, 2008

Study Registration Dates

First Submitted

September 10, 2005

First Submitted That Met QC Criteria

September 10, 2005

First Posted (Estimate)

September 16, 2005

Study Record Updates

Last Update Posted (Estimate)

September 23, 2015

Last Update Submitted That Met QC Criteria

September 21, 2015

Last Verified

January 1, 2009

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BMT 1202

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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