Evaluation of PROCHYMAL® for Treatment-refractory Moderate-to-severe Crohn's Disease

A Multicenter, Open-label Study to Evaluate the Safety of PROCHYMAL® (Remestemcel-L) Intravenous Infusion in Subjects Who Have Received Previous Remestemcel-L Induction Treatment for Treatment-refractory Moderate-to-severe Crohn's Disease

To provide open-label re-treatment with PROCHYMAL to participants enrolled in companion Protocol 603 to evaluate the safety in participants with active Crohn's disease who are resistant to standard Crohn's disease therapies.

Study Overview

• Status: Completed
• Conditions: Crohn's Disease
• Intervention / Treatment: Drug: Prochymal®

Detailed Description

Participants will receive infusions of PROCHYMAL on Day 42, Day 84, and Day 126 after initial infusion of PROCHYMAL in Protocol 603. Each infusion will contain 200 million cells. As participants will be required to be in Protocol 603 during the entire duration of their participation in Protocol 611, all concomitant medication and safety information will be monitored by Protocol 603 and the combination of data from the two protocols.

• Study Type: Interventional
• Enrollment (Actual): 73
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • South Australia
    • Adelaide, South Australia, Australia
      • Royal Adelaide Hospital
  • Victoria
    • Melbourne, Victoria, Australia
      • Royal Melbourne Hospital
• New Zealand
  • Christchurch, New Zealand
    • University of Otago
  • Hamilton, New Zealand
    • Waikato Hospital
• United States
  • California
    • San Francisco, California, United States, 94115
      • University of California, San Francisco
  • Florida
    • Clearwater, Florida, United States, 33765
      • Clinical Research of West Florida
    • Winter Park, Florida, United States, 32789
      • Shafran Gastroenterology Center
  • Illinois
    • Chicago, Illinois, United States, 60611
      • University of Chicago
  • Kansas
    • Topeka, Kansas, United States, 66606
      • Cotton-O'Neil Clinical Research Center
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland, Baltimore
    • Chevy Chase, Maryland, United States, 20815
      • Chevy Chase Clinical Research
  • Missouri
    • St Louis, Missouri, United States, 63128
      • Saint Louis Center for Clinical Research
    • St Louis, Missouri, United States, 63128
      • St. Louis Center for Clinical Studies
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Dartmouth Hitchcock Medical Center
  • New York
    • New York, New York, United States, 10029
      • Mount Sinai School of Medicine
    • New York, New York, United States, 10028
      • Weill Cornell Medical College
  • Tennessee
    • Germantown, Tennessee, United States, 38138
      • Gastroenterology Center of the Midsouth, PC
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University Medical Center
  • Texas
    • Dallas, Texas, United States, 75246
      • Baylor University Medical Center
  • Virginia
    • Richmond, Virginia, United States, 23249
      • McGuire Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participant must have qualified for, enrolled in, and provided written informed consent form (ICF) and authorization for use and disclosure of protected health information (PHI) for Protocol 603 after the August 3, 2010.
• Participant successfully completed all screening assessments in Protocol 603 as required by Protocol 603.
• Participant successfully completed the full course of each of the four infusions of investigational agent on Days 0, 3, 7, and 14 of Protocol 603 after August 3, 2010.
• Participant must enroll in Protocol 611 on or before the 45th day after first infusion in Protocol 603.
• Participant must provide written ICF and authorization for use and disclosure of PHI for Protocol 611.

Exclusion Criteria:

• Participant is unwilling or unable to adhere to requirements of Protocol 611.
• Participant had confirmed respiratory distress during a PROCHYMAL infusion in any prior PROCHYMAL study.
• Participant had a serious adverse event in any previous PROCHYMAL study that was deemed by the principal investigator of that study to be possibly or probably related to PROCHYMAL and also that was within 48 h after a PROCHYMAL infusion.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Prochymal®; Infusions of Prochymal on days 42-45, 84-87, and 126-129 after first infusion in Protocol 603. Each infusion of PROCHYMAL (remestemcel-L) will contain 200 million cells.	Drug: Prochymal®; PROCHYMAL will be administered IV in a total volume of 300 ml (200 million cells) at a rate of 4-6 ml/minute. Treatments will be administered on Days 42-45, Days 84-87, and Days 126-129 following first infusion in Protocol 603.; Other Names: Remestemcel-L; Adult human mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease remission	Crohn's Disease Activity Index (CDAI) at or below 150 and increase in IBDQ	180 Days after first infusion in Protocol 603

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease Improvement	CDAI response to treatments is defined as a CDAI of 150 or below, or a reduction in CDAI of at least 100 points.	180 Days after first infusion in Protocol 603
Improvement in Quality of Life (IBDQ)	IBDQ response to treatment is defined as IBDQ of 170 or above, or an increase in IBDQ of at least 16 points.	180 Days after first infusion in Protocol 603
Number of Adverse events as a measure of safety		180 Days after first infusion in Protocol 603
Infusional toxicity as a measure of safety and tolerability	Infusional toxicity will be evaluated by continuously monitoring the subject's vital signs and O2 saturation by pulse oximetry from the time of PROCHYMAL administration until two hours after infusion is complete.	180 Days after first infusion in Protocol 603

Collaborators and Investigators

• Sponsor: Mesoblast, Inc.
• Investigators: Study Director: Christopher James, PA, Mesoblast, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 29, 2010
• Primary Completion (Actual): September 15, 2014
• Study Completion (Actual): September 15, 2014

Study Registration Dates

• First Submitted: November 2, 2010
• First Submitted That Met QC Criteria: November 2, 2010
• First Posted (Estimated): November 3, 2010

Study Record Updates

• Last Update Posted (Actual): March 11, 2026
• Last Update Submitted That Met QC Criteria: March 9, 2026
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: Crohn's Disease; Adult Stem Cell Therapy; inflammation of the gastrointestinal tract
• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Crohn Disease; Anti-Infective Agents; Antiviral Agents; Anti-Inflammatory Agents; remestemcel-l
• Other Study ID Numbers: CRD 611

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No