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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 16 February 2021
Main ID:  NCT00692939
Date of registration: 03/06/2008
Prospective Registration: Yes
Primary sponsor: Paul Szabolcs
Public title: Autologous Stem Cell Transplantation for Crohn's Disease
Scientific title: Autologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Adult Patients With Severe Crohn's Disease
Date of first enrolment: June 26, 2012
Target sample size: 20
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT00692939
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 1/Phase 2
Countries of recruitment
United States
Contacts
Name:     Shawna H McIntyre, RN
Address: 
Telephone: 412-692-5552
Email: mcintyresm@upmc.edu
Affiliation: 
Name:     Paul Szabolcs, MD
Address: 
Telephone:
Email:
Affiliation:  University of Pittsburgh
Key inclusion & exclusion criteria

5.1 Inclusion Criteria

1. Subject and/or guardian must be able to understand and provide informed consent.

2. Male or female, 10 through 60 years old, inclusive at time of informed consent.

3. Examples of subjects for whom stem cell transplant therapy would be appropriate
include, but are not limited to:

- Patients who have had prior surgery and subsequent severe recurrent disease in
spite of aggressive maintenance therapy, necessitating consideration of further
extensive surgical resections.

- Patients who have diffuse small bowel and colonic disease and who are refractory
to aggressive medical treatment, and not eligible for treatment using a surgical
approach without the risk of precipitating short bowel syndrome and dependence of
parenteral nutrition or who have other conditions that preclude surgery

- Patients with a persistently high Harvey Bradshaw Index (HBI) (>6), CDAI (>250)
or Pediatric CD Activity Index (PCDAI>45) (44) score or those in the lower,
moderate range (HBI = 6), (CDAI < 250), (PCDAI 30-45), but who are dependent on
daily doses of corticosteroids, that are unable to be withdrawn, and aggressive
medical treatment to maintain moderate disease status.

- Patients who have resistant complications of CD unresponsive to medical
management including multiple enteric fistulas, enterovesicular or enterovaginal
fistulas, severe perianal disease, debilitating arthritis, severe skin lesions
(pyoderma), and severe bony complications of the disease and therapy (aseptic
necrosis, pathologic fractures).

- Patients who developed severe complications to while receiving medical management
such as pancreatitis following 6-Mercaptopurine, colitis following 5-ASA or those
with severe hypersensitivity to TNFalpha inhibitors (infliximab, adalimumab,
certolizumab pegol), anti-integrin agents (natalizumab, vedolizumab) or
anti-IL12/23 agents (ustekinumab).

- Patients with stomas are eligible.

4. No surgical therapeutic option secondary to risk of short bowel syndrome or patient
refusal.

5. Harvey Bradshaw Index (HBI) or CD activity score >5, CDAI >250 or PCDAI >30.

6. Platelet count greater than 100,000/mm3.

7. Absolute neutrophil count greater than 1500/mm3 (unless secondary to 6MP therapy).

8. Creatinine = 2.0 mg/dL.

9. No history of coronary artery disease; resting LVEF = 40% or shortening fraction =
26%.

10. FEV1/FVC = 60% predicted for age; DLCO = 60% predicted value for age.

11. Negative pregnancy test for females = 10 years old or who have reached menarche,
unless surgically sterilized.

12. All females or childbearing potential and sexually active males must agree to use a
FDA approved method of birth control for up to 24 months after PBSC transplant or for
as long as they are taking any medication that may harm a pregnancy, an unborn child
or may cause a birth defect.

5.2 Exclusion Criteria

1. Patients who have not been treated with adequate dosing of 6-MP, 5-ASA products and
metronidazole.

2. Patients who achieved a sustained, corticosteroid free response to anti-TNF alpha
therapy, anti-integrin therapy or anti-IL12/23 therapy after a 4 month course of
treatment.

3. Toxic megacolon, intestinal perforation

4. Conjugated bilirubin > 2.0 mg/dL.

5. Pregnancy or nursing mother

6. HIV/HTLV seropositive, HBsAg, or HCV RNA positive by PCR

7. Active infection, as determined by the appropriate confirmatory testing e.g. blood
cultures, PCR testing, etc., within two weeks of mobilization and high dose
chemotherapy.

8. Past or current medical problems or findings from physical examination or laboratory
testing that are not listed above, which, in the opinion of the investigator, may pose
additional risks from participation in the study, may interfere with the participant's
ability to comply with study requirements or that may impact the quality or
interpretation of the data obtained from the study.



Age minimum: 10 Years
Age maximum: 60 Years
Gender: All
Health Condition(s) or Problem(s) studied
Crohn's Disease
Intervention(s)
Biological: autologous CD34-selected peripheral blood stem cells transplant
Drug: Alemtuzumab
Drug: ATG
Drug: Cyclophosphamide
Drug: G-CSF
Drug: Melphalan
Drug: Mesna
Drug: Rituximab
Drug: Thiotepa
Primary Outcome(s)
Change and duration in the Crohn's Disease Activity Index (CDAI). [Time Frame: Change from Baseline to 24 months post Bone Marrow Transplant]
Change and duration in the Harvey Bradshaw Index (HBI). [Time Frame: Change from Baseline to 24 months post Bone Marrow Transplant]
Change and duration in the Pediatric Crohn's Disease Activity Index (PCDAI). [Time Frame: Change from Baseline to 24 months post Bone Marrow Transplant]
Number of participants with life-threatening infections. [Time Frame: From baseline to 24 months post bone marrow transplant]
Number of participants with regimen-related toxicities. [Time Frame: From baseline to 24 months post bone marrow transplant]
Secondary Outcome(s)
Biomarker identification for relapse [Time Frame: From baseline to 24 months post bone marrow transplant]
Number of days it takes for Absolute Neutrophil Count (ANC) to reach greater than 500. [Time Frame: 3 consecutive days once ANC is greater than 500.]
Number of days it takes for Platelet count to reach greater than 20,000/mm3 [Time Frame: From baseline to 24 months post Bone Marrow Transplant.]
Number of days it takes for T cell Recovery [Time Frame: 24 months post Bone Marrow Transplant]
Number of participants who have long term cardiac complications [Time Frame: 24 months post Bone Marrow Transplant]
Number of participants who have long term endocrine complications [Time Frame: 24 months post Bone Marrow Transplant]
Number of participants with active advanced Crohn's disease who have immune dysregulation evolution and correction. [Time Frame: From Baseline to 24 months post bone marrow transplant]
Secondary ID(s)
PRO11090340
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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