Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease
- Conditions
- Crohn's Disease
- Interventions
- Drug: Prochymal®Drug: Placebo
- Registration Number
- NCT00482092
- Lead Sponsor
- Mesoblast, Inc.
- Brief Summary
Protocol 603 is enrolling subjects with moderate-to-severe Crohn's disease who are intolerant to, or have previously failed therapy with, at least one steroid and at least one immunosuppressant and a biologic monoclonal anti-body to tumor necrosis factor alpha. The protocol investigates the safety and efficacy of using PROCHYMAL® adult human stem cells to induce remission. PROCHYMAL is delivered through a vein in the arm four times over two weeks, for approximately an hour each time.
- Detailed Description
A significant number of individuals with Crohn's disease do not find relief with existing steroidal, immunosuppressive, or biologic therapies, and are forced to seek surgery or other drastic measures for treatment.
PROCHYMAL® adult human stem cells are manufactured from healthy, volunteer donors, extensively tested, and are stored to be available as needed. Human and animal studies have shown that the cells do not require any donor-recipient matching. The cells may have both immunosuppressive and healing benefits in Crohn's disease. The cells naturally migrate specifically to sites of inflammation, so their effects are believed to be local and self-limiting rather than systemic.
Protocol 603 is enrolling subjects to evaluate the ability of PROCHYMAL to induce remission in subjects with moderate-to-severe disease (Crohn's disease activity index -- CDAI -- of between 250 and 450, inclusive) who have failed or been intolerant of at least one drug in each of the steroid, immunosuppressant, and biologic classes.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 330
- failed (within last 2 yr) or intolerant of at least one steroid AND at least one immunosuppressant AND at EXACTLY one biologic
- CDAI between 250 and 450, inclusive
- endoscopically or radiographically confirmed Crohn's disease of ileus or colon or both
- C-Reactive Protein Test (CRP) of at least 5 mg/l (0.5 mg/dl)*OR* CDAI of at least 300
- weight between 40 and 150 kg, inclusive
- adequate renal function
- negative tuberculosis skin (PPD) test (or evaluated low risk of TB activation)
- HIV or hepatitis infection active
- allergy to CT contrast agents, or to bovine or porcine products
- symptomatic fibrostenotic Crohn's disease
- permanent ostomy
- biologic therapy within past 90 d
- prednisone greater than 20 mg/d within past month
- short-bowel syndrome
- total parenteral nutrition
- abnormal liver function
- malignancy active within past 5 years (except completely resected basal or squamous cell carcinoma of skin)
- enteric pathogens, including C. difficile
- history of colonic mucosal dysplasia
- current or prior evidence of tuberculosis (TB) (unless risk of activation or re-activation deemed low)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Prochymal® - Low dose Prochymal® Participants will receive a total dose of Prochymal® 600 x 10\^6 cells, IV infusion, on four days, once daily. Placebo Placebo Participants will receive matching placebo administered as intravenous (IV) infusions. Prochymal® - High dose Prochymal® Participants will receive a total dose of Prochymal® 1200 x 10\^6 cells, IV infusion, on four days, once daily.
- Primary Outcome Measures
Name Time Method Disease remission (CDAI at or below 150) 28 days
- Secondary Outcome Measures
Name Time Method Improvement in quality of life (IBDQ) 28 days Reduction in number of draining fistulas 28 days Disease improvement (Reduction by at least 100 points in CDAI) 28 days
Trial Locations
- Locations (56)
University of Maryland, Baltimore
🇺🇸Baltimore, Maryland, United States
Chevy Chase Clinical Research
🇺🇸Chevy Chase, Maryland, United States
University of Chicago Medical Center
🇺🇸Chicago, Illinois, United States
Allegheney Center for Digestive Health
🇺🇸Pittsburgh, Pennsylvania, United States
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
University of Texas Health Science Center
🇺🇸Houston, Texas, United States
Indiana University Medical Center
🇺🇸Indianapolis, Indiana, United States
Cotton-O'Neil Clinical Research Center
🇺🇸Topeka, Kansas, United States
University of California, San Francisco
🇺🇸San Francisco, California, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Rochester General Hospital
🇺🇸Rochester, New York, United States
Royal Melbourne Hospital
🇦🇺Melbourne, Victoria, Australia
National Institutes of Health
🇺🇸Bethesda, Maryland, United States
University of Southern California University Hospital
🇺🇸Los Angeles, California, United States
Western States Clinical Research
🇺🇸Wheat Ridge, Colorado, United States
Clinical Research of West Florida
🇺🇸Clearwater, Florida, United States
Shafran Gastroenterology Center
🇺🇸Winter Park, Florida, United States
Atlanta Gastroenterology Associates
🇺🇸Atlanta, Georgia, United States
Carle Clinic Association
🇺🇸Urbana, Illinois, United States
University of Kentucky Hospital
🇺🇸Lexington, Kentucky, United States
Gulf Coast Research
🇺🇸Baton Rouge, Louisiana, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Clinical Pharmacology Study Group
🇺🇸Worcester, Massachusetts, United States
University of Minnesota Hospital
🇺🇸Minneapolis, Minnesota, United States
Center for Clinical Studies
🇺🇸Dearborn, Michigan, United States
Brigham and Womens Hospital
🇺🇸Boston, Massachusetts, United States
St Louis Center for Clinical Studies
🇺🇸Saint Louis, Missouri, United States
Center for Digestive Health
🇺🇸Troy, Michigan, United States
Weill Cornell Medical College
🇺🇸New York, New York, United States
Dartmouth Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
Mount Sinai School of Medicine
🇺🇸New York, New York, United States
Rochester Institute for Digestive Diseases
🇺🇸Rochester, New York, United States
Charlotte Gastroenterology and Hepatology
🇺🇸Charlotte, North Carolina, United States
University Hospital and Medical Center
🇺🇸Stony Brook, New York, United States
Gastroenterology United of Tulsa
🇺🇸Tulsa, Oklahoma, United States
Options Health Research
🇺🇸Tulsa, Oklahoma, United States
Gastroenterology Center of the Midsouth
🇺🇸Germantown, Tennessee, United States
Nashville GI Specialists
🇺🇸Nashville, Tennessee, United States
University of Texas Medical Branch
🇺🇸Galveston, Texas, United States
University of Vermont
🇺🇸Burlington, Vermont, United States
Digestive and Liver Disease Specialists
🇺🇸Norfolk, Virginia, United States
Royal Adelaide Hospital
🇦🇺Adelaide, South Australia, Australia
University of Alberta Hospital
🇨🇦Edmonton, Alberta, Canada
University of Calgary
🇨🇦Calgary, Alberta, Canada
Health Science Centre
🇨🇦Winnipeg, Manitoba, Canada
London Health Sciences Centre
🇨🇦London, Ontario, Canada
Mount Sinai Hospital
🇨🇦Toronto, Ontario, Canada
Waikato Hospital
🇳🇿Hamilton, New Zealand
University of Otago
🇳🇿Christchurch, New Zealand
Borland-Groover Clinic
🇺🇸Jacksonville, Florida, United States
Seattle Gastroenterology Associates
🇺🇸Seattle, Washington, United States
Pinehurst Medical Clinic
🇺🇸Pinehurst, North Carolina, United States
Baylor University Medical Center
🇺🇸Dallas, Texas, United States
McGuire Research Institute
🇺🇸Richmond, Virginia, United States
University of Louisville Hospital
🇺🇸Louisville, Kentucky, United States
Wake Forest University
🇺🇸Winston-Salem, North Carolina, United States