Prochymal™ Adult Human Mesenchymal Stem Cells for Treatment of Moderate-to-severe Crohn's Disease

NCT00294112 | Completed Phase 2 FDA Drug

• 1 other identifier: OSIRIS-601-602
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 4

Brief Summary

Human mesenchymal stem cells (MSCs), derived from healthy adult volunteer human donors, can be obtained from bone marrow donation and cultured in the laboratory. MSCs have shown the ability to find injured tissue, reduce and control inflammation, and assist in tissue repair. Prochymal™ MSCs will be infused into patients with moderate-to-severe Crohn's disease. Infusions will occur on two separate days, 7-10 days apart. Patients will be monitored for reduced Crohn's disease symptoms.

Conditions

Crohn's Disease

Keywords

mesenchymal stem cells

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Reduction in Crohn's Disease Activity Index (CDAI) of at Least 100 Points

  The CDAI is a composite index of 8 disease variables (stool frequency, severity of abdominal pain, degree of general well-being, presence or absence of extra-intestinal manifestations or fistula, use or non-use of antidiarrheal agents, presence or absence of an abdominal mass, hematocrit, and body weight). Scores range from approximately 0 to 600, with a higher score indicating more disease activity.

  28 days

Secondary Outcomes (9)

• Number of Participants with Reduction in CDAI of at Least 70 points

  28 days

• Improvement as Assessed by the Inflammatory Bowel Disease Questionnaire (IBDQ)

  28 days

• Time to Improvement in IBDQ

  28 days

• Number of Participants with Reduction of at Least 50% in Fistulas in Participants with Fistulas Draining Under Moderate Compression

  28 days

• Number of Participants with Induction of Remission as Defined by Reduction of CDAI to Below 150

  28 days

Study Arms (2)

High dose

EXPERIMENTAL

High dose (8 million cells per kg of body weight)

Drug: Prochymal™ adult human mesenchymal stem cells; Drug: adult human mesenchymal stem cells

Low dose

EXPERIMENTAL

Low dose: 2 million cells per kg body weight

Drug: Prochymal™ adult human mesenchymal stem cells; Drug: adult human mesenchymal stem cells

Interventions

Prochymal™ adult human mesenchymal stem cells DRUG

Cells in plasmalyte and containing dimethylsulfoxide

Also known as: PROCHYMAL

High dose; Low dose

adult human mesenchymal stem cells DRUG

two infusions, one week apart, each comprising adult human mesenchymal stem cells

Also known as: PROCHYMAL

High dose; Low dose

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject must be 18 to 70 years of age, inclusive.
• If female and of child-bearing age, subject must be non-pregnant, non-breast-feeding, and use adequate contraception. If male, subject must use adequate contraception.
• Subject must have endoscopically or radiographically active Crohn's disease
• Subject must have a Crohn's disease activity index (CDAI) of at least 220.
• Subject must have a C-reactive protein (CRP) of at least 5 mg/l.
• Subject must have ileocolitis, colitis, or ileitis.
• At some time during the course of the subject's Crohn's disease (CD), subject must have received both steroids and immunosuppressive agents (for example, azothioprine, 6-mercaptopurine, or methotrexate) which did not control the CD.
• Subject may be receiving antibiotics, 5-aminosalicylic acid, azathioprine, 6-mercaptopurine, methotrexate, prednisone, or any similar drugs at the time of enrollment.
• The dose of 5-aminosalicylic acid (5-ASA) must have been stable for at least 4 weeks prior to enrollment.
• The dose of steroids must have been stable for at least 4 weeks prior to enrollment.
• The dose of antibiotics must have been stable for at least 4 weeks prior to enrollment.
• The dose of immunosuppressants (for example, azathioprine, 6-mercaptopurine \[6-MP\], or methotrexate) must have been stable for at least 8 weeks prior to enrollment and the subject on therapy for at least three months prior to enrollment.
• Subject must have adequate renal function as defined by a calculated creatinine clearance of greater than 30 ml/min using the Cockcroft-Gault equation, and a serum creatinine concentration of less than 2.0 mg/dl.
• Subject must be available for all specified assessments at the study site through day 30.
• Subject must provide a written informed consent form (ICF) and authorization for use of and disclosure of personal health information (PHI).

You may not qualify if:

• Subject has any alcohol or substance abuse within 6 months of randomization.
• Subject has evidence of fibrostenotic obstructive Crohn's disease.
• Subject has an active infection with HIV or hepatitis B or C.
• Subject has had surgery or trauma within 28 d prior to enrollment.
• Subject has a known allergy to computed tomography (CT) contrast agents.
• Subject has a known allergy to bovine or porcine products.
• Subject has body mass greater than 150 kg.
• Subject has had a stricture of the bowel requiring hospitalization within 6 months prior to enrollment.
• Subject has had bowel surgery other than perianal (for example, fistulotomy, seton placement, or abscess drainage) within 6 months prior to enrollment.
• Subject has received infliximab; adalimumab; or other antibody, protein, or biological therapy not specifically approved by the United States Food and Drug Administration (FDA) for Crohn's disease for 90 days (d) prior to enrollment in study.
• Subject has received prednisone greater than 20 mg/d at any time 28 d prior to enrollment in study.
• Subject has a permanent colostomy or ileostomy.
• Subject has aspartate aminotransferase (AST), alkaline phosphatase (ALP), or alanine transaminase (ALT) more than 2.5 times the upper limit of normal at screening.
• Subject has evidence of active malignancy other than resected basal or squamous cell carcinoma of the skin, or prior history of active malignancy that has not been in remission for at least 5 years.
• Subject has history of bacteremia or other serious bacterial or fungal infection in past 3 months other than a treated urinary tract infection or drained perianal abscess.

Sponsors & Collaborators

• Mesoblast, Inc.: lead

Study Sites (4)

Osiris Clinical Site

Baton Rouge, Louisiana, 70801, United States

Location

Osiris Clinical Site

Charlotte, North Carolina, 28105, United States

Location

Osiris Clinical Site

Pittsburgh, Pennsylvania, 15106, United States

Location

Osiris Clinical Site

Richmond, Virginia, 23173, United States

Location

MeSH Terms

Conditions

Crohn Disease

Interventions

remestemcel-l

Condition Hierarchy (Ancestors)

Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Study Officials

• Mahboob Rahman, MD

  Mesoblast, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 17, 2006
• First Posted: February 20, 2006
• Study Start: March 13, 2006
• Primary Completion: July 21, 2006
• Study Completion: July 21, 2006
• Last Updated: April 2, 2020

Record last verified: 2020-03

Locations

US (4)