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Colitis, UlcerativePhase 2NCT01240915

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

A Phase 2 study testing MultiStem low dose and MultiStem high dose in people with Colitis, Ulcerative, run by Pfizer at 58 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Colitis, Ulcerative
What is being tested?
MultiStem low dose and MultiStem high dose
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
105 participants
Where
58 sitesin 8 countries
Study dates
Feb 2011to Nov 2014
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

58 sites in 8 countries.

58 sites in 8 countries

United States37 sites
  • Charlotte Gastroenterology and Hepatology, PLLC

    Charlotte, North Carolina, 28207

  • Clinical Research Institute of Michigan, LLC

    Chesterfield, Michigan, 48047

  • Metropolitan Gastroenterology Group, PC - Chevy Chase Clinical Research

    Chevy Chase, Maryland, 20815

  • University of Chicago Medical Center

    Chicago, Illinois, 60637

  • Gastroenterology Consultants of Clearwater

    Clearwater, Florida, 33756

  • West Coast Endoscopy Center

    Clearwater, Florida, 33756

  • Clinical Research of West Florida, Inc.

    Clearwater, Florida, 33765

  • Surgery Center of Columbia

    Columbia, Missouri, 65201

  • Gastro One

    Germantown, Tennessee, 38138

  • Borland-Groover Clinic

    Jacksonville, Florida, 32256

  • Jacksonville Center for Endoscopy

    Jacksonville, Florida, 32256

  • Clinical Research of the Rockies

    Lafayette, Colorado, 80026

  • Rocky Mountain Gastroenterology Associates, LLC

    Lakewood, Colorado, 80215

  • Arapahoe Gastroenterology, PC

    Littleton, Colorado, 80120

  • University of Louisville Healthcare Outpatient Center

    Louisville, Kentucky, 40202

  • University of Louisville Hospital

    Louisville, Kentucky, 40202

  • Center for Digestive and Liver Diseases, Inc.

    Mexico, Missouri, 65265

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232-1375

  • Present, Chapman, Steinlauf and Marion

    New York, New York, 10028

  • Mount Sinai School of Medicine

    New York, New York, 10029

  • Asher Kornbluth, MD PC

    New York, New York, 10128

  • Digestive and Liver Disease Specialists

    Norfolk, Virginia, 23502

  • Sentara Leigh Hospital

    Norfolk, Virginia, 23502

  • Sentara Norfolk General Hospital

    Norfolk, Virginia, 23507

  • Wake Internal Medicine Consultants, Inc.

    Raleigh, North Carolina, 27612

  • Wake Research Associates

    Raleigh, North Carolina, 27612

  • McGuire DVAMC

    Richmond, Virginia, 23249

  • University of California San Francisco

    San Francisco, California, 94115

  • USCF Endoscopy Unit at Mount Zion

    San Francisco, California, 94115

  • University of Washington Medical Center

    Seattle, Washington, 98195

  • Miami Research Associates

    South Miami, Florida, 33143

  • Cotton-O'Neil Clinical Research Center, Digestive Health

    Topeka, Kansas, 66606

  • Endoscopic Microsurgery Associates, PA

    Towson, Maryland, 21204

  • Center for Digestive Health

    Troy, Michigan, 48098

  • Utica Surgery Center

    Utica, Michigan, 48317

  • Metropolitan Gastroenterology Group, PC

    Washington D.C., District of Columbia, 20006

  • Wake Forest University University Baptist Medical Center - Internal Medicine

    Winston-Salem, North Carolina, 27157

Other trials in United States

Canada4 sites
  • University of Alberta Hospital

    Edmonton, Alberta, T6G 2B7

  • Northern Alberta Clinical Trials and Research Center

    Edmonton, Alberta, T6G 2C8

  • Zeidler Ledcor Centre - University of Alberta

    Edmonton, Alberta, T6G 2X8

  • Maisonneuve-Rosemont Hospital

    Montreal, Quebec, H1T 2M4

Other trials in Canada

Hungary4 sites
  • Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak/I. Belgyogyaszat es Gastroenterologia

    Budapest, 1125

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

    Gyula, 5700

  • Karolina Korhaz Rendelointezet, Belgyogyaszat

    Mosonmagyaróvár, 9200

  • Tolna Megyei Balassa Janos Korhaz / II. Belgyogyaszat

    Szekszárd, 7100

Other trials in Hungary

Germany3 sites
  • Agaplesion Markus Krankenhaus

    Frankfurt am Main, 60431

  • Universitaetsklinikum Halle

    Halle, 06120

  • Medizinische Hochschule Hannover

    Hanover, 30625

Other trials in Germany

Italy3 sites
  • Azienda Ospedaliera Luigi Sacco

    Milan, 20157

  • Azienda Ospedaliera di Padova

    Padova, 35128

  • Policlinico Universitario Agostino Gemelli

    Roma, 00168

Other trials in Italy

Sweden3 sites
  • Sahlgrenska Universitetssjukhuset Medicinkliniken

    Gothenburg, 413 45

  • Karolinska Universitetssjukhuset, GastroCentrum Medicin

    Stockholm, 171 76

  • Akademiska sjukhuset, Mag tarmmottagningen

    Uppsala, 751 85

Other trials in Sweden

Belgium2 sites
  • Hopital Erasme / Gastroenterology

    Brussels, 1070

  • Pfizer Clinical Research Unit

    Brussels, 1070

Other trials in Belgium

Slovakia2 sites
  • Gastroenterologicke a hepatologicke oddelenie, V. interna klinika LFUK a UN Bratislava, Ruzinov

    Bratislava, Slovakia, 826 06

  • Gastroenterologicka ambulancia, GEA s.r.o.

    Trnava, 917 01

Other trials in Slovakia

About this study

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

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.

Being tested
placebo, MultiStem low dose, MultiStem high dose
Keywords
safety efficacy MultiStem(r) moderate to severe Ulcerative Colitis
Medical subject headings
Colitis, Ulcerative

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • placeboDrug

    once every 7 days for 1- 3 doses

  • MultiStem low doseDrug

    1-3 doses

  • placeboDrug

    Single dose at week 8

  • MultiStem low doseDrug

    Single dose at week 8

  • placeboDrug

    Single dose Day 1

  • MultiStem high doseDrug

    Single dose Day 1

  • placeboDrug

    Single dose at week 8

  • MultiStem high doseDrug

    Single dose at week 8

  • placeboDrug

    Single dose Day 1

  • MultiStem high doseDrug

    Single dose Day 1

  • placeboDrug

    Single dose at week 8

  • MultiStem high doseDrug

    Single dose at week 8

Study groups in NCT01240915
Study groupRoleWhat they receive
Cohort 1ExperimentalThe 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).
Receives: Drug: placebo; Drug: MultiStem low dose
Cohort 2ExperimentalThis 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.
Receives: Drug: placebo; Drug: MultiStem high dose
Cohort 3ExperimentalThese 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.
Receives: Drug: placebo; Drug: MultiStem high dose

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8

    Modified Baron Score is an instrument designed to measure endoscopic activity of ulcerative colitis. It classifies the mucosal inflammation in 4 grades (0=normal, 1=granular mucosa with an abnormal vascular pattern, 2=friable mucosa, 3=microulceration with spontaneous bleeding, 4=gross ulceration with spontaneous bleeding).

    Measured: Baseline and Week 8

  2. Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4

    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.

    Measured: Baseline and Week 4

  3. Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8

    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.

    Measured: Baseline and Week 8

  4. Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    An AE was any untoward medical occurrence in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pre-treatment state. AEs included both SAEs and non-SAEs.

    Measured: Baseline up to Week 52

  5. Number of Participants With Treatment-Emergent AEs by System Organ Class (SOC)

    Measured: Baseline up to Week 52

  6. Number of Treatment-Emergent AEs by Severity

    The intensity grades were defined as follows: mild=does not interfere with participant's usual function; moderate=interferes to some extent with participant's usual function; severe=interferes significantly with participant's usual function.

    Measured: Baseline up to Week 52

Secondary outcomes (15)
  1. Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16

    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.

    Measured: Baseline, Week 12, Week 16

  2. Number of Participants With Laboratory Test Abnormalities

    The total number of participants with laboratory test abnormalities with or without regard to baseline abnormality was assessed. Laboratory parameters included: hematology (hemoglobin, hematocrit, red blood cell [RBC] count, platelet count, white blood cell [WBC] count, total neutrophils, eosinophils, monocytes, basophils, lymphocytes, erythrocyte sedimentation rate); chemistry (blood urea nitrogen and creatinine, glucose, calcium, sodium, potassium, chloride, total bicarbonate, aspartate aminotransferase, alanine aminotransferase, total bilirubin, alkaline phosphatase, uric acid, albumin, total protein; urinalysis (pH, glucose, protein, blood, ketones, nitrites, leukocyte esterase, microscopy (only if urine dipstick was positive for blood, protein, nitrites or leukocyte esterase); other (follicle-stimulating hormone, human chorionic gonadotropin, stool microbiology, creatinine kinase, direct bilirubin, indirect bilirubin, gamma-glutamyl transferase, international normalized ratio.

    Measured: Baseline up to Week 24

  3. Number of Participants With Potentially Clinically Significant Vital Signs Findings

    Vital signs assessment included pulse rate, systolic blood pressure (SBP) and diastolic blood pressure (DBP). Criteria for vital sign values meeting potential clinical concern included: SBP <90 millimeters of mercury (mm Hg) and >=30 mm Hg increase/decrease from baseline, DBP <50 mm Hg and >=20 mm Hg increase/decrease from baseline, pulse rate <40 or >120 beats per minute (bpm),

    Measured: Baseline up to Week 52

  4. Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16

    Fecal calprotectin, a very stable marker, is a 36kDa calcium and zinc binding protein which is neutrophil-derived. It represents 60% of cytosolic proteins in granulocytes and is a measurement of neutrophil migration to the gastrointestinal tract.

    Measured: Baseline, Weeks 4, 8, 12 and 16

  5. Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16

    CRP is an acute-phase protein which provides an objective criterion of inflammatory activity. CRP has a short half-life (19 hours) and therefore rises early after the onset of inflammation and rapidly decreases after resolution of the inflammation. It is induced by interleukin-6, TNF-alpha and other pro-inflammatory cytokines that are produced within the intestinal lamina propria.

    Measured: Baseline, Weeks 4, 8, 12 and 16

  6. Percentage of Participants With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16

    Mayo subscores for rectal bleeding range from 0 to 3 (0=no blood seen; 1=streaks of blood with stool less than half the time; 2=obvious blood with stool most of the time; 3=blood alone passes).

    Measured: Week 4, 8, 12 and 16

  7. Percentage of Participants in Endoscopic Remission at Week 8

    Modified Baron Score is an instrument designed to measure endoscopic activity of ulcerative colitis. It classifies the mucosal inflammation in 4 grades (0=normal, 1=granular mucosa with an abnormal vascular pattern, 2=friable mucosa, 3=microulceration with spontaneous bleeding, 4=gross ulceration with spontaneous bleeding). Endoscopic remission is defined as modified Baron Endoscopic Score equal to 0.

    Measured: Week 8

  8. Percentage of Participants in Clinical Remission at Week 8

    Clinical remission is defined as a total Mayo score of 2 points or lower, with no individual subscores exceeding 1 point.

    Measured: Week 8

  9. Percentage of Participants With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16

    Mayo subscores for rectal bleeding range from 0 to 3 (0=no blood seen; 1=streaks of blood with stool less than half the time; 2=obvious blood with stool most of the time; 3=blood alone passes). A decrease from baseline score indicates improvement.

    Measured: Baseline, Weeks 4, 8, 12 and 16

  10. Percentage of Participants With Endoscopic Response at Week 8

    Endoscopic response is defined as a decrease in modified Baron endoscopic score from baseline of at least 2 points.

    Measured: Week 8

  11. Percentage of Participants in Clinical Response at Week 8

    Clinical response is defined as a decrease in total Mayo score from baseline of at least 3 points and at least 30 percent (%), with an accompanying decrease in the subscore for rectal bleeding of at least 1 point or an absolute subscore for rectal bleeding of 0 or 1.

    Measured: Week 8

  12. Change From Baseline in Total Mayo Scores at Week 8

    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis, with total score ranging from 0 to 12. It consists of 4 subscores (stool frequency, rectal bleeding, findings of flexible proctosigmoidoscopy, and physician global assessment [PGA]), each graded from 0 to 3, with higher scores indicating more severe disease.

    Measured: Baseline, Week 8

  13. Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16

    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo Score ranges from 0 to 12 points. It consists of 4 subscores, each graded from 0 to 3 with higher scores indicating more severe disease. A Partial Mayo Score (Mayo score without endoscopy) ranges from 0 (normal or inactive disease) to 9 (severe disease) and calculated as the sum of 3 subscores (stool frequency, rectal bleeding and PGA) with each ranging from 0 to 3 (0=normal, 1=mild, 2=moderate, 3=severe). Higher scores indicate more severe disease.

    Measured: Baseline, Weeks 4, 8, 12 and 16

  14. Change From Baseline in Biopsy Histology Scores at Week 8

    A 15 to 25 centimeter (cm) biopsy sample of inflamed mucosal tissue was taken from the worst affected area and scored using the Riley Index. The Riley Index is a histologic scoring system for the assessment of the activity and severity of ulcerative colitis, ranging from 0 to 24. It consists of 6 histologic features (acute inflammatory cell infiltrate, crypt abscesses, mucin depletion, surface epithelial integrity, chronic inflammatory cell infiltrate, and crypt architectural irregularities), all scored on a 4-point scale (higher scores indicate more severe disease).

    Measured: Baseline and Week 8

  15. Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16

    Patient-reported diary data assessed the number of bowel movements (BM) per day when not having a flare and the presence of blood in the stools (rectal bleeding [RB]), if any.

    Measured: Baseline and Week 16

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Nov 2010 (estimated)
  2. Study startFeb 2011
  3. Primary completionNov 2014
  4. Study completionNov 2014

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Investigator, Outcomes assessor
Enrolment
105 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01240915
Sponsor's protocol ID
B3041001
Other IDs
2010-022766-27
Status last verified
Jan 2016
First submitted
10 Nov 2010
Last updated
23 Feb 2016
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing MultiStem low dose and MultiStem high dose in people with Colitis, Ulcerative, run by Pfizer at 58 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 23 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 58 sites in United States, Canada, Hungary, Germany, Italy and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 Feb 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8 (time frame: Baseline and Week 8). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Feb 2016.

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