Clinical trial NCT00543374: Extended Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-Resistant Moderate-to-Severe Crohn's Disease
Official title: A Phase III, Multicenter, Placebo-controlled, Randomized, Double-blind Durability and Retreatment Study to Evaluate the Safety and Efficacy of PROCHYMAL® (ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Intravenous Infusion for the Maintenance and Re-induction of Clinical Benefit and Remission in Subjects Experiencing Treatment-refractory Moderate-to-severe Crohn's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Crohn's Disease
Lead sponsor: Mesoblast, Ltd.
Sponsor class: INDUSTRY
First posted: 2007-10-15
Enrollment: 98
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Protocol 610 is enrolling subjects who successfully achieved clinical benefit (reduction in Crohn's Disease Activity Index (CDAI) of at least 100 points) in Protocol 603. Protocol 610 is evaluating the length of initial effect of PROCHYMAL® adult human mesenchymal stem cells and the ability of these cells to successfully re-induce clinical benefit.

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 610 is an extension study offered only to those subjects who successfully achieved clinical benefit (reduction of CDAI of at least 100 points) in Protocol 603.

Eligibility criteria: Inclusion Criteria:

* Participation in Osiris Protocol 603, with drop of at least 100 points in CDAI at Day 28

Exclusion Criteria:

* Substance abuse
* Failure to receive full dose of all interventions in Protocol 603

Primary outcomes:
measure: Duration of clinical benefit (Crohn's disease activity index)
timeFrame: 6 months
measure: Re-induction of clinical benefit (Crohn's disease activity index)
timeFrame: 6 months
Secondary outcomes:
measure: Improvement in quality of life (Inflammatory Bowel Disease Questionnaire [IBDQ] instrument)
timeFrame: 6 months
Other outcomes:
Locations:
city: Anaheim
country: United States
facility: Advanced Clinical Research Institute
state: California
city: Long Beach
country: United States
facility: Veteran's Administration Medical Center (does not require vet status)
state: California
city: Los Angeles
country: United States
facility: University of Southern California University Hospital
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Wheat Ridge
country: United States
facility: Western States Clinical Research
state: Colorado
city: Hamden
country: United States
facility: Gastroenterology Center of Connecticut
state: Connecticut
city: Clearwater
country: United States
facility: Clinical Research of West Florida
state: Florida
city: Jacksonville
country: United States
facility: Borland-Groover Clinic
state: Florida
city: Miami
country: United States
facility: Venture Research Institute
state: Florida
city: Winter Park
country: United States
facility: Shafran Gastroenterology Center
state: Florida
city: Atlanta
country: United States
facility: Atlanta Gastroenterology Associates
state: Georgia
city: Chicago
country: United States
facility: University of Chicago Medical Center
state: Illinois
city: Urbana
country: United States
facility: Carle Clinic Association
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Medical Center
state: Indiana
city: Topeka
country: United States
facility: Cotton-O'Neil Clinical Research Center
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky Hospital
state: Kentucky
city: Louisville
country: United States
facility: University of Louisville Hospital
state: Kentucky
city: Baton Rouge
country: United States
facility: Gulf Coast Research
state: Louisiana
city: Bethesda
country: United States
facility: National Institutes of Health
state: Maryland
city: Chevy Chase
country: United States
facility: Metropolitan Gastroenterology Group
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Womens Hospital
state: Massachusetts
city: Worcester
country: United States
facility: Clinical Pharmacology Study Group
state: Massachusetts
city: Dearborn
country: United States
facility: Center for Clinical Studies
state: Michigan
city: Troy
country: United States
facility: Center for Digestive Health
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota Hospital
state: Minnesota
city: Saint Louis
country: United States
facility: St Louis Center for Clinical Studies
state: Missouri
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Cedar Knolls
country: United States
facility: Gastroenterology Research Associates
state: New Jersey
city: Teaneck
country: United States
facility: Holy Name Hospital
state: New Jersey
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Rochester
country: United States
facility: Rochester Institute for Digestive Diseases
state: New York
city: Rochester
country: United States
facility: Rochester General Hospital
state: New York
city: Stony Brook
country: United States
facility: University Hospital and Medical Center
state: New York
city: Charlotte
country: United States
facility: Charlotte Gastroenterology and Hepatology
state: North Carolina
city: Pinehurst
country: United States
facility: Pinehurst Medical Clinic
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest University
state: North Carolina
city: Dayton
country: United States
facility: Dayton Science Institute
state: Ohio
city: Tulsa
country: United States
facility: Gastroenterology United of Tulsa
state: Oklahoma
city: Tulsa
country: United States
facility: Options Health Research
state: Oklahoma
city: Pittsburgh
country: United States
facility: Allegheney Center for Digestive Health
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Germantown
country: United States
facility: Gastroenterology Center of the Midsouth
state: Tennessee
city: Nashville
country: United States
facility: Nashville GI Specialists
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: Baylor University Medical Center
state: Texas
city: Galveston
country: United States
facility: University of Texas Medical Branch
state: Texas
city: Houston
country: United States
facility: Memorial Hermann Hospital
state: Texas
city: Burlington
country: United States
facility: University of Vermont
state: Vermont
city: Norfolk
country: United States
facility: Digestive and Liver Disease Specialists
state: Virginia
city: Richmond
country: United States
facility: McGuire Research Institute
state: Virginia
city: Seattle
country: United States
facility: Seattle Gastroenterology Associates
state: Washington
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta Hospital
state: Alberta
city: Winnipeg
country: Canada
facility: Health Sciences Centre
state: Manitoba
city: London
country: Canada
facility: London Health Sciences Centre
state: Ontario
city: Toronto
country: Canada
facility: Mount Sinai Hospital
state: Ontario
Study officials:
affiliation: Mesoblast, Inc.
name: Mahboob Rahman, MD
role: STUDY_DIRECTOR
References: