Status:
WITHDRAWN
Pilot Study of Stem Cell Transplantation for Children and Young Adults With Refractory Crohn's Disease.
Lead Sponsor:
TriStar Health
Conditions:
Crohn's Disease
Eligibility:
All Genders
10-30 years
Phase:
NA
Brief Summary
This is a study for people with Crohn's Disease (CD) that are not responsive to standard treatment. CD is a chronic disease with an auto-immune component that goes away and relapses over the years and...
Detailed Description
Crohn's Disease (CD) is an immunologically mediated chronic illness that has a relapsing and remitting course, most commonly presenting in the 2nd or 3rd decade and causing lifelong impairment of heal...
Eligibility Criteria
Inclusion
- All the subjects considered eligible for the study will be screened and reviewed by the Gastroenterology (GI) physicians prior to enrollment (Dedrick Moulton, MD or his designee at Vanderbilt Children's Hospital) 2. Age ≥ 10 and \< 30 years 3. Disease status:
- Confirmed diagnosis of Crohn's Disease: Diagnosis of Crohn's disease that has been established based on typical endoscopic/histologic and/or radiological appearances.
- Active disease, defined as: Pediatric Crohn's Disease Activity Index (PCDAI) \>30 (see Appendix I) or Crohn's Disease Activity Index (CDAI) of \>250 (see Appendix II) at any time within 3 months prior to enrollment and any one of the following- i. Endoscopic evidence of active disease confirmed on histology within 3 months prior to enrollment, or ii. Clear evidence of active small bowel Crohn's disease on small bowel imaging within 3 months prior to enrollment.
- Refractory disease- Moderate to severe disease that has been unresponsive to current or prior therapy with mercaptopurine and/or azathioprine (thiopurines), methotrexate and anti-TNF therapy. Patients should have relapsing disease (i.e. ≥ 1 exacerbation/year) or corticosteroid dependence despite current or prior thiopurines, methotrexate and anti-TNF maintenance therapy or clear demonstration of intolerance or toxicity to these drugs. Patients who fail induction therapy with corticosteroids and anti-TNF therapy, and are therefore not eligible to receive maintenance therapy with thiopurines or methotrexate will also be candidates for enrollment.
- 4\. Negative stool culture, C. difficile, and negative CMV pcr (in stool or colonic biopsy). Patients with CMV colitis will receive a trial of anti-viral treatment and only responders will be considered eligible for inclusion.
- 5\. Patients with a prior ileostomy or colostomy may enter the study. For this group of patients', physician's global assessment will be used to assess clinical activity of CD, as Pediatric CDAI and CDAI scoring method may not be representative of disease activity.
- 6\. Patients with abscesses are eligible to enroll once the abscesses or any other significant infection has resolved.
Exclusion
- Pregnancy or unwillingness to use adequate contraception during the study- if a woman is of childbearing age.
- HIV infection.
- Organ function criteria-
- Renal: creatinine clearance \< 50 ml/min/1.73m2 (measured or estimated).
- Cardiac: left ventricular ejection fraction \<30% by multigated radionuclide angiography (MUGA) or a shortening fraction of \< 25% by cardiac echocardiogram.
- Pulmonary Function tests: DLCO \< 30% or patient on oxygen.
- Hepatic: serum bilirubin \> 3 mg%; AST and ALT \> 3x ULN for the institutional lab.
- Uncontrolled Hypertension (using age based criteria) despite at least 2 anti- hypertensive agents.
- Active Infection or risk thereof-
- Current abscess or significant active infection
- Perianal infection is not an exclusion criterion, provided there is drainage with or without placement of drain.
- Abnormal chest x- ray (CXR) consistent with active infection or neoplasm.
- Severe diarrhea due to short small bowel; patients believed to have \< 700 mm of small bowel and diarrhea attributable to this will be excluded.
- Patients with toxic megacolon, active bowel obstruction or intestinal perforation.
- Unable to collect minimum of 3 x106/kg CD34+ cell dose. These patients will be excluded from receiving the preparative regimen.
Key Trial Info
Start Date :
August 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2019
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT02225795
Start Date
August 1 2014
End Date
December 1 2019
Last Update
February 7 2018
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