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

Phase 3

Completed

• Conditions: Crohn's Disease
• Interventions: Drug: adult human mesenchymal stem cells

• Registration Number: NCT01233960
• Lead Sponsor: Mesoblast, Inc.

Brief Summary

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

Detailed Description

Subjects 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 subjects 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 Timeline

• Study First Submitted: 2010-11-02
• Study First Submitted QC: 2010-11-02
• Study First Posted: 2010-11-03
• Study Start: 2010-11-29
• Primary Completion: 2014-09-15
• Study Completion: 2014-09-15
• Status Verified: 2020-03
• Disposition First Submitted: 2021-12-03
• Disposition First Submitted QC: 2021-12-03
• Last Update Submitted: 2021-12-03
• Disposition First Posted: 2021-12-08
• Last Update Posted: 2021-12-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 73

Inclusion Criteria

• Subject 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.
• Subject successfully completed all screening assessments in Protocol 603 as required by Protocol 603.
• Subject 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.
• Subject must enroll in Protocol 611 on or before the 45th day after first infusion in Protocol 603.
• Subject must provide written ICF and authorization for use and disclosure of PHI for Protocol 611.

Exclusion Criteria

• Subject is unwilling or unable to adhere to requirements of Protocol 611.
• Subject had confirmed respiratory distress during a PROCHYMAL infusion in any prior PROCHYMAL study.
• Subject 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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Prochymal	adult human mesenchymal stem cells	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Disease Improvement	180 Days after first infusion in Protocol 603	CDAI response to treatments is defined as a CDAI of 150 or below, or a reduction in CDAI of at least 100 points.
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	180 Days after first infusion in Protocol 603	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.
Improvement in Quality of Life (IBDQ)	180 Days after first infusion in Protocol 603	IBDQ response to treatment is defined as IBDQ of 170 or above, or an increase in IBDQ of at least 16 points.

Trial Locations

Locations (20)

Royal Melbourne Hospital; 🇦🇺 Melbourne, Victoria, Australia

Shafran Gastroenterology Center; 🇺🇸 Winter Park, Florida, United States

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

Baylor University Medical Center; 🇺🇸 Dallas, Texas, United States

Royal Adelaide Hospital; 🇦🇺 Adelaide, South Australia, Australia

University of California, San Francisco; 🇺🇸 San Francisco, California, United States

Clinical Research of West Florida; 🇺🇸 Clearwater, Florida, United States

Cotton-O'Neil Clinical Research Center; 🇺🇸 Topeka, Kansas, United States

University of Maryland, Baltimore; 🇺🇸 Baltimore, Maryland, United States

University of Chicago; 🇺🇸 Chicago, Illinois, United States

Chevy Chase Clinical Research; 🇺🇸 Chevy Chase, Maryland, United States

St. Louis Center for Clinical Studies; 🇺🇸 Saint Louis, Missouri, United States

Saint Louis Center for Clinical Research; 🇺🇸 Saint Louis, Missouri, United States

Mount Sinai School of Medicine; 🇺🇸 New York, New York, United States

Weill Cornell Medical College; 🇺🇸 New York, New York, United States

McGuire Research Institute; 🇺🇸 Richmond, Virginia, United States

Gastroenterology Center of the Midsouth, PC; 🇺🇸 Germantown, Tennessee, United States

Waikato Hospital; 🇳🇿 Hamilton, New Zealand

University of Otago; 🇳🇿 Christchurch, New Zealand

Dartmouth HItchcock Medical Center; 🇺🇸 Lebanon, New Hampshire, United States