A Study To Investigate The Safety And Possible Clinical Benefit Of Multistem(r) In Patients With Moderate To Severe Ulcerative Colitis

NCT ID: NCT01240915

Last Updated: 2016-02-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 105 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-02-28
• Study Completion Date: 2014-11-30

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.

Conditions

Colitis, Ulcerative

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Cohort 1

The first 9 subjects will be recruited into Cohort 1 and will receive either placebo (n=3) or MultiStem low dose (n=6) as an intravenous infusion on Day 1. The first five patients enrolled constitute a subgroup of Cohort 1 and these patients will receive multiple doses, once every day for 7 days for 3 doses (Day 1 and Weeks 1 \& 2).

Group Type: EXPERIMENTAL

placebo

Intervention Type: DRUG

once every 7 days for 1- 3 doses

MultiStem low dose

Intervention Type: DRUG

1-3 doses

placebo

Intervention Type: DRUG

Single dose at week 8

MultiStem low dose

Intervention Type: DRUG

Single dose at week 8

Cohort 2

This group will receive either placebo (n=3) or MultiStem high dose (n=6) as an intravenous infusion on Day 1. The subjects then receive the opposite dose of study medication at Week 8.

Group Type: EXPERIMENTAL

placebo

Intervention Type: DRUG

Single dose Day 1

MultiStem high dose

Intervention Type: DRUG

Single dose Day 1

placebo

Intervention Type: DRUG

Single dose at week 8

MultiStem high dose

Intervention Type: DRUG

Single dose at week 8

Cohort 3

These subjects (total n=88 evaluable patients) will receive either Placebo or MultiStem (1:1 randomization) as an intravenous infusion on Day 1. In addition all subjects in Cohort 3 will receive a single infusion of either MultiStem or Placebo at Week 8, depending on their randomization schedule. A total of \~22 patients will receive an additional infusion of MultiStem, \~44 patients will receive the alternative blinded therapy to that which they received for Day 1 infusion, and \~22 patients will receive an additional infusion of placebo.

Group Type: EXPERIMENTAL

placebo

Intervention Type: DRUG

Single dose Day 1

MultiStem high dose

Intervention Type: DRUG

Single dose Day 1

placebo

Intervention Type: DRUG

Single dose at week 8

MultiStem high dose

Intervention Type: DRUG

Single dose at week 8

Interventions

placebo

once every 7 days for 1- 3 doses

Intervention Type: DRUG

MultiStem low dose

1-3 doses

Intervention Type: DRUG

placebo

Single dose at week 8

Intervention Type: DRUG

MultiStem low dose

Single dose at week 8

Intervention Type: DRUG

placebo

Single dose Day 1

Intervention Type: DRUG

MultiStem high dose

Single dose Day 1

Intervention Type: DRUG

placebo

Single dose at week 8

Intervention Type: DRUG

MultiStem high dose

Single dose at week 8

Intervention Type: DRUG

placebo

Single dose Day 1

Intervention Type: DRUG

MultiStem high dose

Single dose Day 1

Intervention Type: DRUG

placebo

Single dose at week 8

Intervention Type: DRUG

MultiStem high dose

Single dose at week 8

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Healios K.K.

INDUSTRY

Sponsor Role: collaborator

Pfizer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

University of California San Francisco

San Francisco, California, United States

USCF Endoscopy Unit at Mount Zion

San Francisco, California, United States

Clinical Research of the Rockies

Lafayette, Colorado, United States

Rocky Mountain Gastroenterology Associates, LLC

Lakewood, Colorado, United States

Arapahoe Gastroenterology, PC

Littleton, Colorado, United States

Metropolitan Gastroenterology Group, PC

Washington D.C., District of Columbia, United States

Gastroenterology Consultants of Clearwater

Clearwater, Florida, United States

West Coast Endoscopy Center

Clearwater, Florida, United States

Clinical Research of West Florida, Inc.

Clearwater, Florida, United States

Borland-Groover Clinic

Jacksonville, Florida, United States

Jacksonville Center for Endoscopy

Jacksonville, Florida, United States

Miami Research Associates

South Miami, Florida, United States

University of Chicago Medical Center

Chicago, Illinois, United States

Cotton-O'Neil Clinical Research Center, Digestive Health

Topeka, Kansas, United States

University of Louisville Healthcare Outpatient Center

Louisville, Kentucky, United States

University of Louisville Hospital

Louisville, Kentucky, United States

Metropolitan Gastroenterology Group, PC - Chevy Chase Clinical Research

Chevy Chase, Maryland, United States

Endoscopic Microsurgery Associates, PA

Towson, Maryland, United States

Clinical Research Institute of Michigan, LLC

Chesterfield, Michigan, United States

Center for Digestive Health

Troy, Michigan, United States

Utica Surgery Center

Utica, Michigan, United States

Surgery Center of Columbia

Columbia, Missouri, United States

Center for Digestive and Liver Diseases, Inc.

Mexico, Missouri, United States

Present, Chapman, Steinlauf and Marion

New York, New York, United States

Mount Sinai School of Medicine

New York, New York, United States

Asher Kornbluth, MD PC

New York, New York, United States

Charlotte Gastroenterology and Hepatology, PLLC

Charlotte, North Carolina, United States

Wake Internal Medicine Consultants, Inc.

Raleigh, North Carolina, United States

Wake Research Associates

Raleigh, North Carolina, United States

Wake Forest University University Baptist Medical Center - Internal Medicine

Winston-Salem, North Carolina, United States

Gastro One

Germantown, Tennessee, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Digestive and Liver Disease Specialists

Norfolk, Virginia, United States

Sentara Leigh Hospital

Norfolk, Virginia, United States

Sentara Norfolk General Hospital

Norfolk, Virginia, United States

McGuire DVAMC

Richmond, Virginia, United States

University of Washington Medical Center

Seattle, Washington, United States

Hopital Erasme / Gastroenterology

Brussels, , Belgium

Pfizer Clinical Research Unit

Brussels, , Belgium

University of Alberta Hospital

Edmonton, Alberta, Canada

Northern Alberta Clinical Trials and Research Center

Edmonton, Alberta, Canada

Zeidler Ledcor Centre - University of Alberta

Edmonton, Alberta, Canada

Maisonneuve-Rosemont Hospital

Montreal, Quebec, Canada

Agaplesion Markus Krankenhaus

Frankfurt am Main, , Germany

Universitaetsklinikum Halle

Halle, , Germany

Medizinische Hochschule Hannover

Hanover, , Germany

Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak/I. Belgyogyaszat es Gastroenterologia

Budapest, , Hungary

Pandy Kalman Megyei Korhaz, III. sz. Belosztaly-Gasztroenterologia

Gyula, , Hungary

Karolina Korhaz Rendelointezet, Belgyogyaszat

Mosonmagyaróvár, , Hungary

Tolna Megyei Balassa Janos Korhaz / II. Belgyogyaszat

Szekszárd, , Hungary

Azienda Ospedaliera Luigi Sacco

Milan, , Italy

Azienda Ospedaliera di Padova

Padua, , Italy

Policlinico Universitario Agostino Gemelli

Roma, , Italy

Gastroenterologicke a hepatologicke oddelenie, V. interna klinika LFUK a UN Bratislava, Ruzinov

Bratislava, Slovakia, Slovakia

Gastroenterologicka ambulancia, GEA s.r.o.

Trnava, , Slovakia

Sahlgrenska Universitetssjukhuset Medicinkliniken

Gothenburg, , Sweden

Karolinska Universitetssjukhuset, GastroCentrum Medicin

Stockholm, , Sweden

Akademiska sjukhuset, Mag tarmmottagningen

Uppsala, , Sweden

Countries

United States; Belgium; Canada; Germany; Hungary; Italy; Slovakia; Sweden

Related Links

https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=B3041001&StudyName=A%20Study%20To%20Investigate%20The%20Safety%20And%20Possible%20Clinical%20Benefit%20Of%20Multistem%28r%29%20In%20Patients%20With%20Moderate%20To%20Severe%20Ulcerative%20Colit

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Other Identifiers

2010-022766-27

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

B3041001

Identifier Type: -

Identifier Source: org_study_id