Crohn's Disease Stem Cell Transplantation

• Sponsor: Northwestern University (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00271947
• Collaborator: (none)
• Enrollment: 1
• Location: 1
• Arm: 1
• Duration (Months): 96
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

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 or Disease	Intervention/Treatment	Phase
Crohn's Disease	Biological: Autologous Stem Cell Transplantation	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 1 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Crohn's Disease Non-myeloablative Autologous Hematopoietic Stem Cell Transplantation (CDNST) Versus Standard Therapy
• Study Start Date: Apr 1, 2005
• Actual Primary Completion Date: Apr 1, 2012
• Actual Study Completion Date: Apr 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Autologous Stem Cell Transplantation Autologous Stem Cell Transplantation will be performed after the conditioning regimen	Biological: Autologous Stem Cell Transplantation; Autologous Stem Cell Transplantation will be performed after conditioning regimen

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Remission or Clinical Improvement as Assessed by Crohn's Disease Activity Index (CDAI) Scores [baseline]

   Clinical remission defined as a CDAI less than 150 and clinical improvement defined as decline in CDI> or = 70 one year following entry. The participant was not assessed according to the criteria of the outcome measure, because the participant was lost for follow-up.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 55 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Locations

	Site	City	State	Country	Postal Code
1	Northwestern University, Feinberg School of Medicine	Chicago	Illinois	United States	60611

Sponsors and Collaborators

• Northwestern University

Investigators

• Principal Investigator: Robert Craig, MD, Northwestern University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Richard Burt, MD, MD, Northwestern University
• ClinicalTrials.gov Identifier: NCT00271947
• Other Study ID Numbers: CD Randomized
• First Posted: Jan 4, 2006
• Last Update Posted: Mar 27, 2014
• Last Verified: Feb 1, 2014

• Additional relevant MeSH terms: Crohn Disease

Study Results

Participant Flow

Recruitment Details	enrolled (n=1)

Arm/Group Title	Autologous Stem Cell Transplantation
Arm/Group Description	stem cell transplantation : Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning regimen
Period Title: Overall Study
STARTED	1
COMPLETED	0
NOT COMPLETED	1

Baseline Characteristics

Arm/Group Title	Stem Cell Transplantation
Arm/Group Description	stem cell transplantation : Autologous Hematopoietic Stem Cell Transplantation will be performed on all participants randomized to transplant arm.
Overall Participants	1
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	1 100%
>=65 years	0 0%
Sex: Female, Male (Count of Participants)
Female	1 100%
Male	0 0%
Region of Enrollment (participants) [Number]
United States	1 100%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Remission or Clinical Improvement as Assessed by Crohn's Disease Activity Index (CDAI) Scores
Description	Clinical remission defined as a CDAI less than 150 and clinical improvement defined as decline in CDI> or = 70 one year following entry. The participant was not assessed according to the criteria of the outcome measure, because the participant was lost for follow-up.
Time Frame	baseline

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Stem Cell Transplantation
Arm/Group Description	stem cell transplantation : Autologous Hematopoietic Stem Cell Transplantation will be performed on all participants randomized to transplant arm.
Measure Participants	0

Adverse Events

Time Frame	6 months
Adverse Event Reporting Description	CDAI
Arm/Group Title	Stem Cell Transplantation
Arm/Group Description	stem cell transplantation : Autologous Hematopoietic Stem Cell Transplantation will be performed on all participants randomized to transplant arm.
All Cause Mortality
	Stem Cell Transplantation
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Stem Cell Transplantation
	Affected / at Risk (%)	# Events
Total	0/1 (0%)
Other (Not Including Serious) Adverse Events
	Stem Cell Transplantation
	Affected / at Risk (%)	# Events
Total	0/1 (0%)

Limitations/Caveats

Potential participants refused to be randomized. Just one participant was enrolled on study, the study is terminated early.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.