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

Study Overview
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Brief Summary
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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
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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

Official Title
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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

Conditions
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Crohn's Disease

Intervention / Treatment
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* Drug: adult human mesenchymal stem cells


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Prochymal<br>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: adult human mesenchymal stem cells<br>* 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.<br>* Other names: remestemcel-L;|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Crohn's Disease, Adult Stem Cell Therapy, inflammation of the gastrointestinal tract