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

Brief Summary

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”

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.

  • Study Type: Interventional
  • Study Design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
  • Study Primary Completion Date: December 2016

Detailed Clinical Trial 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.

Interventions Used in this Clinical Trial

  • Drug: adult human mesenchymal stem cells
    • Intravenous infusion of suspension of adult human mesenchymal stem cells, total of 1200 million (high dose) or 600 million (low dose) cells infused in four visits over two weeks.
  • Drug: Placebo
    • Placebo

Arms, Groups and Cohorts in this Clinical Trial

  • Placebo Comparator: 1
    • Placebo
  • Active Comparator: 2
    • Low dose (600 million cells total over four infusions in two weeks)
  • Active Comparator: 3
    • High dose (1200 million cells delivered in four infusions over two weeks)

Outcome Measures for this Clinical Trial

Primary Measures

  • Disease remission (CDAI at or below 150)
    • Time Frame: 28 days
      Safety Issue?: No

Secondary Measures

  • Disease improvement (Reduction by at least 100 points in CDAI)
    • Time Frame: 28 days
      Safety Issue?: No
  • Improvement in quality of life (IBDQ)
    • Time Frame: 28 days
      Safety Issue?: No
  • Reduction in number of draining fistulas
    • Time Frame: 28 days
      Safety Issue?: No

Criteria for Participation in this Clinical Trial

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
  • 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 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 TB (unless risk of activation or re-activation deemed low)

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 18 Years

Maximum Age for this Clinical Trial: 70 Years

Are Healthy Volunteers Accepted for this Clinical Trial: No

Clinical Trial Investigator Information

  • Lead Sponsor
    • Mesoblast International Sàrl
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Monica Luchi, MD, Study Director, Mesoblast International Sarl

Source

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The URL of this page is:
http://clinicaltrialsfeeds.org/clinical-trials/show/NCT00482092