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

Primary Purpose

Crohn's Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
adult human mesenchymal stem cells
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, inflammation of the gastrointestinal tract

Eligibility Criteria

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

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.

Sites / Locations

  • University of California, San Francisco
  • Clinical Research of West Florida
  • Shafran Gastroenterology Center
  • University of Chicago
  • Cotton-O'Neil Clinical Research Center
  • University of Maryland, Baltimore
  • Chevy Chase Clinical Research
  • Saint Louis Center for Clinical Research
  • St. Louis Center for Clinical Studies
  • Dartmouth HItchcock Medical Center
  • Weill Cornell Medical College
  • Mount Sinai School of Medicine
  • Gastroenterology Center of the Midsouth, PC
  • Vanderbilt University Medical Center
  • Baylor University Medical Center
  • McGuire Research Institute
  • Royal Adelaide Hospital
  • Royal Melbourne Hospital
  • University of Otago
  • Waikato Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Prochymal

Arm Description

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.

Outcomes

Primary Outcome Measures

Disease remission
Crohn's Disease Activity Index (CDAI) at or below 150 and increase in IBDQ

Secondary Outcome Measures

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.
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.
Number of Adverse events as a measure of safety
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.

Full Information

First Posted
November 2, 2010
Last Updated
December 3, 2021
Sponsor
Mesoblast, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01233960
Brief Title
Evaluation of PROCHYMAL® for Treatment-refractory Moderate-to-severe Crohn's Disease
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2020
Overall Recruitment Status
Completed
Study Start Date
November 29, 2010 (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
No

5. Study Description

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

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, inflammation of the gastrointestinal tract

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
73 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Prochymal
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
adult human mesenchymal stem cells
Other Intervention Name(s)
Prochymal, remestemcel-L
Intervention Description
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.
Primary Outcome Measure Information:
Title
Disease remission
Description
Crohn's Disease Activity Index (CDAI) at or below 150 and increase in IBDQ
Time Frame
180 Days after first infusion in Protocol 603
Secondary Outcome Measure Information:
Title
Disease Improvement
Description
CDAI response to treatments is defined as a CDAI of 150 or below, or a reduction in CDAI of at least 100 points.
Time Frame
180 Days after first infusion in Protocol 603
Title
Improvement in Quality of Life (IBDQ)
Description
IBDQ response to treatment is defined as IBDQ of 170 or above, or an increase in IBDQ of at least 16 points.
Time Frame
180 Days after first infusion in Protocol 603
Title
Number of Adverse events as a measure of safety
Time Frame
180 Days after first infusion in Protocol 603
Title
Infusional toxicity as a measure of safety and tolerability
Description
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.
Time Frame
180 Days after first infusion in Protocol 603

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: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mahboob Rahman
Organizational Affiliation
Mesoblast, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of California, San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
Clinical Research of West Florida
City
Clearwater
State/Province
Florida
ZIP/Postal Code
33765
Country
United States
Facility Name
Shafran Gastroenterology Center
City
Winter Park
State/Province
Florida
ZIP/Postal Code
32789
Country
United States
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
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 Maryland, Baltimore
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Chevy Chase Clinical Research
City
Chevy Chase
State/Province
Maryland
ZIP/Postal Code
20815
Country
United States
Facility Name
Saint Louis Center for Clinical Research
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63128
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
Gastroenterology Center of the Midsouth, PC
City
Germantown
State/Province
Tennessee
ZIP/Postal Code
38138
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
McGuire Research Institute
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23249
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 Otago
City
Christchurch
Country
New Zealand
Facility Name
Waikato Hospital
City
Hamilton
Country
New Zealand

12. IPD Sharing Statement

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

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