Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease

• Sponsor: Mesoblast, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00482092
• Collaborator: (none)
• Enrollment: 330
• Locations: 56
• Arms: 3
• Actual Duration (Months): 83.9
• Patients Per Site: 5.9
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

Protocol 603 is enrolling subjects with moderate-to-severe Crohn's disease who are intolerant to, or have previously failed therapy with, at least one steroid and at least one immunosuppressant and a biologic monoclonal anti-body to tumor necrosis factor alpha. The protocol investigates the safety and efficacy of using PROCHYMAL® adult human stem cells to induce remission. PROCHYMAL is delivered through a vein in the arm four times over two weeks, for approximately an hour each time.

Condition or Disease	Intervention/Treatment	Phase
Crohn's Disease	Drug: Prochymal®; Drug: Placebo	Phase 3

Detailed Description

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 603 is enrolling subjects to evaluate the ability of PROCHYMAL to induce remission in subjects with moderate-to-severe disease (Crohn's disease activity index -- CDAI -- of between 250 and 450, inclusive) who have failed or been intolerant of at least one drug in each of the steroid, immunosuppressant, and biologic classes.

Study Design

• Study Type: Interventional
• Actual Enrollment: 330 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase III, Multicenter, Placebo-controlled, Randomized, Double-blind Study to Evaluate the Safety and Efficacy of PROCHYMAL® (ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Intravenous Infusion for the Induction of Remission in Subjects Experiencing Treatment-refractory Moderate-to-severe Crohn's Disease
• Actual Study Start Date: Sep 17, 2007
• Actual Primary Completion Date: Sep 15, 2014
• Actual Study Completion Date: Sep 15, 2014

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo Participants will receive matching placebo administered as intravenous (IV) infusions.	Drug: Placebo; Prochymal® Placebo-matching IV infusion
Active Comparator: Prochymal® - Low dose Participants will receive a total dose of Prochymal® 600 x 10^6 cells, IV infusion, on four days, once daily.	Drug: Prochymal®; Prochymal® IV infusion; Other Names: Adult Human Mesenchymal Stem Cells
Active Comparator: Prochymal® - High dose Participants will receive a total dose of Prochymal® 1200 x 10^6 cells, IV infusion, on four days, once daily.	Drug: Prochymal®; Prochymal® IV infusion; Other Names: Adult Human Mesenchymal Stem Cells

Outcome Measures

Primary Outcome Measures

1. Disease remission (CDAI at or below 150) [28 days]

Secondary Outcome Measures

1. Disease improvement (Reduction by at least 100 points in CDAI) [28 days]

2. Improvement in quality of life (IBDQ) [28 days]

3. Reduction in number of draining fistulas [28 days]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 70 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• failed (within last 2 yr) or intolerant of at least one steroid AND at least one immunosuppressant AND at EXACTLY one biologic

• CDAI between 250 and 450, inclusive

• endoscopically or radiographically confirmed Crohn's disease of ileus or colon or both

• C-Reactive Protein Test (CRP) of at least 5 mg/l (0.5 mg/dl)OR CDAI of at least 300

• weight between 40 and 150 kg, inclusive

• adequate renal function

• negative tuberculosis skin (PPD) test (or evaluated low risk of TB activation)

Exclusion Criteria:

• HIV or hepatitis infection active

• allergy to CT contrast agents, or to bovine or porcine products

• symptomatic fibrostenotic Crohn's disease

• permanent ostomy

• biologic therapy within past 90 d

• prednisone greater than 20 mg/d within past month

• short-bowel syndrome

• total parenteral nutrition

• abnormal liver function

• malignancy active within past 5 years (except completely resected basal or squamous cell carcinoma of skin)

• enteric pathogens, including C. difficile

• history of colonic mucosal dysplasia

• current or prior evidence of tuberculosis (TB) (unless risk of activation or re-activation deemed low)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Southern California University Hospital	Los Angeles	California	United States	90033
2	University of California, San Francisco	San Francisco	California	United States	94115
3	Western States Clinical Research	Wheat Ridge	Colorado	United States	80033
4	Clinical Research of West Florida	Clearwater	Florida	United States	33765
5	Borland-Groover Clinic	Jacksonville	Florida	United States	32256
6	Shafran Gastroenterology Center	Winter Park	Florida	United States	32789
7	Atlanta Gastroenterology Associates	Atlanta	Georgia	United States	30342
8	University of Chicago Medical Center	Chicago	Illinois	United States	60637
9	Carle Clinic Association	Urbana	Illinois	United States	61801
10	Indiana University Medical Center	Indianapolis	Indiana	United States	46202
11	Cotton-O'Neil Clinical Research Center	Topeka	Kansas	United States	66606
12	University of Kentucky Hospital	Lexington	Kentucky	United States	40536
13	University of Louisville Hospital	Louisville	Kentucky	United States	40202
14	Gulf Coast Research	Baton Rouge	Louisiana	United States	70808
15	University of Maryland, Baltimore	Baltimore	Maryland	United States	21201
16	National Institutes of Health	Bethesda	Maryland	United States	20892
17	Chevy Chase Clinical Research	Chevy Chase	Maryland	United States	20815
18	Massachusetts General Hospital	Boston	Massachusetts	United States	02114
19	Brigham and Womens Hospital	Boston	Massachusetts	United States	02115
20	Clinical Pharmacology Study Group	Worcester	Massachusetts	United States	01610
21	Center for Clinical Studies	Dearborn	Michigan	United States	48124
22	Center for Digestive Health	Troy	Michigan	United States	48098
23	University of Minnesota Hospital	Minneapolis	Minnesota	United States	55455
24	St Louis Center for Clinical Studies	Saint Louis	Missouri	United States	63128
25	Dartmouth Hitchcock Medical Center	Lebanon	New Hampshire	United States	03756
26	Weill Cornell Medical College	New York	New York	United States	10028
27	Mount Sinai School of Medicine	New York	New York	United States	10029
28	Rochester Institute for Digestive Diseases	Rochester	New York	United States	14607
29	Rochester General Hospital	Rochester	New York	United States	14621
30	University Hospital and Medical Center	Stony Brook	New York	United States	11794
31	Charlotte Gastroenterology and Hepatology	Charlotte	North Carolina	United States	28207
32	Pinehurst Medical Clinic	Pinehurst	North Carolina	United States	28734
33	Wake Forest University	Winston-Salem	North Carolina	United States	27157
34	Gastroenterology United of Tulsa	Tulsa	Oklahoma	United States	74135
35	Options Health Research	Tulsa	Oklahoma	United States	74137
36	Allegheney Center for Digestive Health	Pittsburgh	Pennsylvania	United States	15212
37	University of Pittsburgh	Pittsburgh	Pennsylvania	United States	15213
38	Gastroenterology Center of the Midsouth	Germantown	Tennessee	United States	37138
39	Nashville GI Specialists	Nashville	Tennessee	United States	37205
40	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232
41	Baylor University Medical Center	Dallas	Texas	United States	75246
42	University of Texas Medical Branch	Galveston	Texas	United States	77555
43	University of Texas Health Science Center	Houston	Texas	United States	77030
44	University of Vermont	Burlington	Vermont	United States	05401
45	Digestive and Liver Disease Specialists	Norfolk	Virginia	United States	23502
46	McGuire Research Institute	Richmond	Virginia	United States	23249
47	Seattle Gastroenterology Associates	Seattle	Washington	United States	98133
48	Royal Adelaide Hospital	Adelaide	South Australia	Australia
49	Royal Melbourne Hospital	Melbourne	Victoria	Australia
50	University of Calgary	Calgary	Alberta	Canada	T2N 4N1
51	University of Alberta Hospital	Edmonton	Alberta	Canada	T6G 2X8
52	Health Science Centre	Winnipeg	Manitoba	Canada	R3A 1R9
53	London Health Sciences Centre	London	Ontario	Canada	N6A 5A5
54	Mount Sinai Hospital	Toronto	Ontario	Canada	M5G 1X5
55	University of Otago	Christchurch		New Zealand
56	Waikato Hospital	Hamilton		New Zealand

Sponsors and Collaborators

• Mesoblast, Inc.

Investigators

• Study Director: Mahboob Rahman, MD, Mesoblast, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Mesoblast, Inc.
• ClinicalTrials.gov Identifier: NCT00482092
• Other Study ID Numbers: CRD 603; NCT00609232
• First Posted: Jun 4, 2007
• Last Update Posted: Jan 13, 2022
• Last Verified: Jan 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Mesoblast, Inc.: Crohn's disease; adult stem cell therapy
• Additional relevant MeSH terms: Crohn Disease; Remestemcel-l

Study Results

No Results Posted as of Jan 13, 2022