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Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease

Primary Purpose

Crohn's Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Prochymal®
Placebo
Sponsored by
Mesoblast, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Crohn's Disease focused on measuring Crohn's disease, adult stem cell therapy

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 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)

Exclusion Criteria:

  • 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)

Sites / Locations

  • University of Southern California University Hospital
  • University of California, San Francisco
  • Western States Clinical Research
  • Clinical Research of West Florida
  • Borland-Groover Clinic
  • Shafran Gastroenterology Center
  • Atlanta Gastroenterology Associates
  • University of Chicago Medical Center
  • Carle Clinic Association
  • Indiana University Medical Center
  • Cotton-O'Neil Clinical Research Center
  • University of Kentucky Hospital
  • University of Louisville Hospital
  • Gulf Coast Research
  • University of Maryland, Baltimore
  • National Institutes of Health
  • Chevy Chase Clinical Research
  • Massachusetts General Hospital
  • Brigham and Womens Hospital
  • Clinical Pharmacology Study Group
  • Center for Clinical Studies
  • Center for Digestive Health
  • University of Minnesota Hospital
  • St Louis Center for Clinical Studies
  • Dartmouth Hitchcock Medical Center
  • Weill Cornell Medical College
  • Mount Sinai School of Medicine
  • Rochester Institute for Digestive Diseases
  • Rochester General Hospital
  • University Hospital and Medical Center
  • Charlotte Gastroenterology and Hepatology
  • Pinehurst Medical Clinic
  • Wake Forest University
  • Gastroenterology United of Tulsa
  • Options Health Research
  • Allegheney Center for Digestive Health
  • University of Pittsburgh
  • Gastroenterology Center of the Midsouth
  • Nashville GI Specialists
  • Vanderbilt University Medical Center
  • Baylor University Medical Center
  • University of Texas Medical Branch
  • University of Texas Health Science Center
  • University of Vermont
  • Digestive and Liver Disease Specialists
  • McGuire Research Institute
  • Seattle Gastroenterology Associates
  • Royal Adelaide Hospital
  • Royal Melbourne Hospital
  • University of Calgary
  • University of Alberta Hospital
  • Health Science Centre
  • London Health Sciences Centre
  • Mount Sinai Hospital
  • University of Otago
  • Waikato Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Placebo Comparator

Active Comparator

Active Comparator

Arm Label

Placebo

Prochymal® - Low dose

Prochymal® - High dose

Arm Description

Participants will receive matching placebo administered as intravenous (IV) infusions.

Participants will receive a total dose of Prochymal® 600 x 10^6 cells, IV infusion, on four days, once daily.

Participants will receive a total dose of Prochymal® 1200 x 10^6 cells, IV infusion, on four days, once daily.

Outcomes

Primary Outcome Measures

Disease remission (CDAI at or below 150)

Secondary Outcome Measures

Disease improvement (Reduction by at least 100 points in CDAI)
Improvement in quality of life (IBDQ)
Reduction in number of draining fistulas

Full Information

First Posted
May 30, 2007
Last Updated
January 11, 2022
Sponsor
Mesoblast, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT00482092
Brief Title
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 Study to Evaluate the Safety and Efficacy of PROCHYMAL® (ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Intravenous Infusion for the Induction of Remission in Subjects Experiencing Treatment-refractory Moderate-to-severe Crohn's Disease
Study Type
Interventional

2. Study Status

Record Verification Date
January 2022
Overall Recruitment Status
Completed
Study Start Date
September 17, 2007 (Actual)
Primary Completion Date
September 15, 2014 (Actual)
Study Completion Date
September 15, 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mesoblast, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Crohn's Disease
Keywords
Crohn's disease, adult stem cell therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
330 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive matching placebo administered as intravenous (IV) infusions.
Arm Title
Prochymal® - Low dose
Arm Type
Active Comparator
Arm Description
Participants will receive a total dose of Prochymal® 600 x 10^6 cells, IV infusion, on four days, once daily.
Arm Title
Prochymal® - High dose
Arm Type
Active Comparator
Arm Description
Participants will receive a total dose of Prochymal® 1200 x 10^6 cells, IV infusion, on four days, once daily.
Intervention Type
Drug
Intervention Name(s)
Prochymal®
Other Intervention Name(s)
Adult Human Mesenchymal Stem Cells
Intervention Description
Prochymal® IV infusion
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Prochymal® Placebo-matching IV infusion
Primary Outcome Measure Information:
Title
Disease remission (CDAI at or below 150)
Time Frame
28 days
Secondary Outcome Measure Information:
Title
Disease improvement (Reduction by at least 100 points in CDAI)
Time Frame
28 days
Title
Improvement in quality of life (IBDQ)
Time Frame
28 days
Title
Reduction in number of draining fistulas
Time Frame
28 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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) Exclusion Criteria: 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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mahboob Rahman, MD
Organizational Affiliation
Mesoblast, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of Southern California University Hospital
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
University of California, San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
Western States Clinical Research
City
Wheat Ridge
State/Province
Colorado
ZIP/Postal Code
80033
Country
United States
Facility Name
Clinical Research of West Florida
City
Clearwater
State/Province
Florida
ZIP/Postal Code
33765
Country
United States
Facility Name
Borland-Groover Clinic
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32256
Country
United States
Facility Name
Shafran Gastroenterology Center
City
Winter Park
State/Province
Florida
ZIP/Postal Code
32789
Country
United States
Facility Name
Atlanta Gastroenterology Associates
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
University of Chicago Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
Carle Clinic Association
City
Urbana
State/Province
Illinois
ZIP/Postal Code
61801
Country
United States
Facility Name
Indiana University Medical Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Cotton-O'Neil Clinical Research Center
City
Topeka
State/Province
Kansas
ZIP/Postal Code
66606
Country
United States
Facility Name
University of Kentucky Hospital
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
Facility Name
University of Louisville Hospital
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Gulf Coast Research
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
Facility Name
University of Maryland, Baltimore
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
National Institutes of Health
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Chevy Chase Clinical Research
City
Chevy Chase
State/Province
Maryland
ZIP/Postal Code
20815
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Brigham and Womens Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Clinical Pharmacology Study Group
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01610
Country
United States
Facility Name
Center for Clinical Studies
City
Dearborn
State/Province
Michigan
ZIP/Postal Code
48124
Country
United States
Facility Name
Center for Digestive Health
City
Troy
State/Province
Michigan
ZIP/Postal Code
48098
Country
United States
Facility Name
University of Minnesota Hospital
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
St Louis Center for Clinical Studies
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63128
Country
United States
Facility Name
Dartmouth Hitchcock Medical Center
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
Facility Name
Weill Cornell Medical College
City
New York
State/Province
New York
ZIP/Postal Code
10028
Country
United States
Facility Name
Mount Sinai School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Rochester Institute for Digestive Diseases
City
Rochester
State/Province
New York
ZIP/Postal Code
14607
Country
United States
Facility Name
Rochester General Hospital
City
Rochester
State/Province
New York
ZIP/Postal Code
14621
Country
United States
Facility Name
University Hospital and Medical Center
City
Stony Brook
State/Province
New York
ZIP/Postal Code
11794
Country
United States
Facility Name
Charlotte Gastroenterology and Hepatology
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28207
Country
United States
Facility Name
Pinehurst Medical Clinic
City
Pinehurst
State/Province
North Carolina
ZIP/Postal Code
28734
Country
United States
Facility Name
Wake Forest University
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Gastroenterology United of Tulsa
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74135
Country
United States
Facility Name
Options Health Research
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74137
Country
United States
Facility Name
Allegheney Center for Digestive Health
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15212
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Gastroenterology Center of the Midsouth
City
Germantown
State/Province
Tennessee
ZIP/Postal Code
37138
Country
United States
Facility Name
Nashville GI Specialists
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37205
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Baylor University Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75246
Country
United States
Facility Name
University of Texas Medical Branch
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555
Country
United States
Facility Name
University of Texas Health Science Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Vermont
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05401
Country
United States
Facility Name
Digestive and Liver Disease Specialists
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23502
Country
United States
Facility Name
McGuire Research Institute
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23249
Country
United States
Facility Name
Seattle Gastroenterology Associates
City
Seattle
State/Province
Washington
ZIP/Postal Code
98133
Country
United States
Facility Name
Royal Adelaide Hospital
City
Adelaide
State/Province
South Australia
Country
Australia
Facility Name
Royal Melbourne Hospital
City
Melbourne
State/Province
Victoria
Country
Australia
Facility Name
University of Calgary
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 4N1
Country
Canada
Facility Name
University of Alberta Hospital
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2X8
Country
Canada
Facility Name
Health Science Centre
City
Winnipeg
State/Province
Manitoba
ZIP/Postal Code
R3A 1R9
Country
Canada
Facility Name
London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5A5
Country
Canada
Facility Name
Mount Sinai Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X5
Country
Canada
Facility Name
University of Otago
City
Christchurch
Country
New Zealand
Facility Name
Waikato Hospital
City
Hamilton
Country
New Zealand

12. IPD Sharing Statement

Learn more about this trial

Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease

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