Extended Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-Resistant Moderate-to-Severe Crohn's Disease

Study Overview
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Brief Summary
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Protocol 610 is enrolling subjects who successfully achieved clinical benefit (reduction in Crohn's Disease Activity Index (CDAI) of at least 100 points) in Protocol 603. Protocol 610 is evaluating the length of initial effect of PROCHYMAL® adult human mesenchymal stem cells and the ability of these cells to successfully re-induce clinical benefit.

Detailed Description
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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 610 is an extension study offered only to those subjects who successfully achieved clinical benefit (reduction of CDAI of at least 100 points) in Protocol 603.

Official Title
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A Phase III, Multicenter, Placebo-controlled, Randomized, Double-blind Durability and Retreatment Study to Evaluate the Safety and Efficacy of PROCHYMAL® (ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Intravenous Infusion for the Maintenance and Re-induction of Clinical Benefit and Remission in Subjects Experiencing Treatment-refractory Moderate-to-severe Crohn's Disease

Conditions
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Crohn's Disease

Intervention / Treatment
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* Drug: Placebo
* Drug: PROCHYMAL adult human mesenchymal stem cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participation in Osiris Protocol 603, with drop of at least 100 points in CDAI at Day 28 Exclusion Criteria: Substance abuse Failure to receive full dose of all interventions in Protocol 603

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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Placebo | Drug: Placebo<br>* Placebo<br>|
| Active Comparator: PROCHYMAL Low dose<br>Low dose (total of 600 million cells) | Drug: PROCHYMAL adult human mesenchymal stem cells<br>* intravenous infusion four times over two weeks; possibly repeated once<br>|
| Active Comparator: PROCHYMAL High dose<br>High dose (total of 1200 cells) | Drug: PROCHYMAL adult human mesenchymal stem cells<br>* intravenous infusion four times over two weeks; possibly repeated once<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Duration of clinical benefit (Crohn's disease activity index) |  | 6 months | 
| Re-induction of clinical benefit (Crohn's disease activity index) |  | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Improvement in quality of life (Inflammatory Bowel Disease Questionnaire [IBDQ] instrument) |  | 6 months |