Clinical trial NCT01240915: A Study To Investigate The Safety And Possible Clinical Benefit Of Multistem(r) In Patients With Moderate To Severe Ulcerative Colitis
Official title: A Phase 2 Randomized, Double-blind, Placebo-controlled, Parallel Group, Multi-center Study To Investigate The Safety And Efficacy Of Multistem (Pf-05285401) In Subjects With Moderate To Severe Ulcerative Colitis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Colitis, Ulcerative
Keywords: safety efficacy MultiStem(r) moderate to severe Ulcerative Colitis
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
Collaborators: name: Healios K.K.
First posted: 2010-11-15
Enrollment: 105
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: MultiStem(r) is a new biological product, manufactured from human stem cells obtained from adult bone marrow or other nonembryonic tissue sources. Factors expressed by MultiStem cells are believed to reduce inflammation and regulate immune system function, protect damaged or injured cells and tissue, promote formation of new blood vessels, and augment tissue repair and healing. MultiStem cell treatment resulted in significant efficacy in a mouse model of Graft versus Host Disease with almost complete reversal of gastrointestinal pathology (similar to pathology that would be expected in Ulcerative Colitis). These data, together with safety data generated in 2 other clinical trials, suggest that MultiStem has the potential to be a new treatment option for patients with ulcerative colitis. This is the first study of MultiStem in this patient population and will cautiously explore the safety/toleration and potential benefit of this new treatment in patients with moderate to severe disease.


Eligibility criteria: Inclusion Criteria:

* Subjects must have a documented diagnosis of ulcerative colitis at least 6 months prior to screening.
* Subjects must have active moderate-to-severe ulcerative colitis based on Mayo score.
* Subjects must have Modified Baron endoscopic score of at least 2 determined within 7 days of first dosing.
* Subjects must have failed or be intolerant (as determined by the investigator) of at least one of the following treatments for UC: Oral corticosteroids, azathioprine or 6-mercaptopurine (6-MP), or anti-tumor necrosis factor (TNF) therapy, eg, infliximab or adalimumab.
* Subjects must be on stable steroid doses.

Exclusion Criteria:

* Subjects who have abnormal organ and marrow function.
* Subjects with a diagnosis of indeterminate colitis, or clinical findings suggestive of Crohn's disease.
* Subjects who meet Truelove-Witts criteria for severe ulcerative colitis.
* Subjects receiving or who are expected to receive Infliximab or other biologic treatment within 8 weeks of the Day 1 study visit.
* Subjects receiving or who are expected to receive Cyclosporine, mycophenolate, or tacrolimus within 4 weeks of the Day 1 study visit.

Primary outcomes:
measure: Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8
timeFrame: Baseline and Week 8
measure: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4
timeFrame: Baseline and Week 4
measure: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8
timeFrame: Baseline and Week 8
measure: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to Week 52
measure: Number of Participants With Treatment-Emergent AEs by System Organ Class (SOC)
timeFrame: Baseline up to Week 52
measure: Number of Treatment-Emergent AEs by Severity
timeFrame: Baseline up to Week 52
Secondary outcomes:
measure: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16
timeFrame: Baseline, Week 12, Week 16
measure: Number of Participants With Laboratory Test Abnormalities
timeFrame: Baseline up to Week 24
measure: Number of Participants With Potentially Clinically Significant Vital Signs Findings
timeFrame: Baseline up to Week 52
measure: Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16
timeFrame: Baseline, Weeks 4, 8, 12 and 16
measure: Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16
timeFrame: Baseline, Weeks 4, 8, 12 and 16
measure: Percentage of Participants With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16
timeFrame: Week 4, 8, 12 and 16
measure: Percentage of Participants in Endoscopic Remission at Week 8
timeFrame: Week 8
measure: Percentage of Participants in Clinical Remission at Week 8
timeFrame: Week 8
measure: Percentage of Participants With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16
timeFrame: Baseline, Weeks 4, 8, 12 and 16
measure: Percentage of Participants With Endoscopic Response at Week 8
timeFrame: Week 8
measure: Percentage of Participants in Clinical Response at Week 8
timeFrame: Week 8
measure: Change From Baseline in Total Mayo Scores at Week 8
timeFrame: Baseline, Week 8
measure: Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16
timeFrame: Baseline, Weeks 4, 8, 12 and 16
measure: Change From Baseline in Biopsy Histology Scores at Week 8
timeFrame: Baseline and Week 8
measure: Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16
timeFrame: Baseline and Week 16
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: San Francisco
country: United States
facility: USCF Endoscopy Unit at Mount Zion
state: California
city: Lafayette
country: United States
facility: Clinical Research of the Rockies
state: Colorado
city: Lakewood
country: United States
facility: Rocky Mountain Gastroenterology Associates, LLC
state: Colorado
city: Littleton
country: United States
facility: Arapahoe Gastroenterology, PC
state: Colorado
city: Washington
country: United States
facility: Metropolitan Gastroenterology Group, PC
state: District of Columbia
city: Clearwater
country: United States
facility: Gastroenterology Consultants of Clearwater
state: Florida
city: Clearwater
country: United States
facility: West Coast Endoscopy Center
state: Florida
city: Clearwater
country: United States
facility: Clinical Research of West Florida, Inc.
state: Florida
city: Jacksonville
country: United States
facility: Borland-Groover Clinic
state: Florida
city: Jacksonville
country: United States
facility: Jacksonville Center for Endoscopy
state: Florida
city: South Miami
country: United States
facility: Miami Research Associates
state: Florida
city: Chicago
country: United States
facility: University of Chicago Medical Center
state: Illinois
city: Topeka
country: United States
facility: Cotton-O'Neil Clinical Research Center, Digestive Health
state: Kansas
city: Louisville
country: United States
facility: University of Louisville Healthcare Outpatient Center
state: Kentucky
city: Louisville
country: United States
facility: University of Louisville Hospital
state: Kentucky
city: Chevy Chase
country: United States
facility: Metropolitan Gastroenterology Group, PC - Chevy Chase Clinical Research
state: Maryland
city: Towson
country: United States
facility: Endoscopic Microsurgery Associates, PA
state: Maryland
city: Chesterfield
country: United States
facility: Clinical Research Institute of Michigan, LLC
state: Michigan
city: Troy
country: United States
facility: Center for Digestive Health
state: Michigan
city: Utica
country: United States
facility: Utica Surgery Center
state: Michigan
city: Columbia
country: United States
facility: Surgery Center of Columbia
state: Missouri
city: Mexico
country: United States
facility: Center for Digestive and Liver Diseases, Inc.
state: Missouri
city: New York
country: United States
facility: Present, Chapman, Steinlauf and Marion
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: New York
country: United States
facility: Asher Kornbluth, MD PC
state: New York
city: Charlotte
country: United States
facility: Charlotte Gastroenterology and Hepatology, PLLC
state: North Carolina
city: Raleigh
country: United States
facility: Wake Internal Medicine Consultants, Inc.
state: North Carolina
city: Raleigh
country: United States
facility: Wake Research Associates
state: North Carolina
city: Winston Salem
country: United States
facility: Wake Forest University University Baptist Medical Center - Internal Medicine
state: North Carolina
city: Germantown
country: United States
facility: Gastro One
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Norfolk
country: United States
facility: Digestive and Liver Disease Specialists
state: Virginia
city: Norfolk
country: United States
facility: Sentara Leigh Hospital
state: Virginia
city: Norfolk
country: United States
facility: Sentara Norfolk General Hospital
state: Virginia
city: Richmond
country: United States
facility: McGuire DVAMC
state: Virginia
city: Seattle
country: United States
facility: University of Washington Medical Center
state: Washington
city: Brussels
country: Belgium
facility: Hopital Erasme / Gastroenterology
city: Bruxelles
country: Belgium
facility: Pfizer Clinical Research Unit
city: Edmonton
country: Canada
facility: University of Alberta Hospital
state: Alberta
city: Edmonton
country: Canada
facility: Northern Alberta Clinical Trials and Research Center
state: Alberta
city: Edmonton
country: Canada
facility: Zeidler Ledcor Centre - University of Alberta
state: Alberta
city: Montreal
country: Canada
facility: Maisonneuve-Rosemont Hospital
state: Quebec
city: Frankfurt am Main
country: Germany
facility: Agaplesion Markus Krankenhaus
city: Halle
country: Germany
facility: Universitaetsklinikum Halle
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Budapest
country: Hungary
facility: Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak/I. Belgyogyaszat es Gastroenterologia
city: Gyula
country: Hungary
facility: Pandy Kalman Megyei Korhaz, III. sz. Belosztaly-Gasztroenterologia
city: Mosonmagyarovar
country: Hungary
facility: Karolina Korhaz Rendelointezet, Belgyogyaszat
city: Szekszard
country: Hungary
facility: Tolna Megyei Balassa Janos Korhaz / II. Belgyogyaszat
city: Milano
country: Italy
facility: Azienda Ospedaliera Luigi Sacco
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova
city: Roma
country: Italy
facility: Policlinico Universitario Agostino Gemelli
city: Bratislava
country: Slovakia
facility: Gastroenterologicke a hepatologicke oddelenie, V. interna klinika LFUK a UN Bratislava, Ruzinov
city: Trnava
country: Slovakia
facility: Gastroenterologicka ambulancia, GEA s.r.o.
city: Goteborg
country: Sweden
facility: Sahlgrenska Universitetssjukhuset Medicinkliniken
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset, GastroCentrum Medicin
city: Uppsala
country: Sweden
facility: Akademiska sjukhuset, Mag tarmmottagningen
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: