We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00482092
Recruitment Status : Completed
First Posted : June 4, 2007
Last Update Posted : January 13, 2022
Sponsor:
Information provided by (Responsible Party):
Mesoblast, Ltd. ( Mesoblast, Inc. )

Tracking Information
First Submitted Date  ICMJE May 30, 2007
First Posted Date  ICMJE June 4, 2007
Last Update Posted Date January 13, 2022
Actual Study Start Date  ICMJE September 17, 2007
Actual Primary Completion Date September 15, 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 31, 2007)
Disease remission (CDAI at or below 150) [ Time Frame: 28 days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 31, 2007)
  • Disease improvement (Reduction by at least 100 points in CDAI) [ Time Frame: 28 days ]
  • Improvement in quality of life (IBDQ) [ Time Frame: 28 days ]
  • Reduction in number of draining fistulas [ Time Frame: 28 days ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease
Official Title  ICMJE 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
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Crohn's Disease
Intervention  ICMJE
  • Drug: Prochymal®
    Prochymal® IV infusion
    Other Name: Adult Human Mesenchymal Stem Cells
  • Drug: Placebo
    Prochymal® Placebo-matching IV infusion
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Participants will receive matching placebo administered as intravenous (IV) infusions.
    Intervention: Drug: Placebo
  • Active Comparator: Prochymal® - Low dose
    Participants will receive a total dose of Prochymal® 600 x 10^6 cells, IV infusion, on four days, once daily.
    Intervention: Drug: Prochymal®
  • Active Comparator: Prochymal® - High dose
    Participants will receive a total dose of Prochymal® 1200 x 10^6 cells, IV infusion, on four days, once daily.
    Intervention: Drug: Prochymal®
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 19, 2014)
330
Original Estimated Enrollment  ICMJE
 (submitted: May 31, 2007)
258
Actual Study Completion Date  ICMJE September 15, 2014
Actual Primary Completion Date September 15, 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   New Zealand,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00482092
Other Study ID Numbers  ICMJE CRD 603
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Mesoblast, Ltd. ( Mesoblast, Inc. )
Original Responsible Party Not Provided
Current Study Sponsor  ICMJE Mesoblast, Inc.
Original Study Sponsor  ICMJE Osiris Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Mahboob Rahman, MD Mesoblast, Inc.
PRS Account Mesoblast, Ltd.
Verification Date January 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP