Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00294112
Recruitment Status : Completed
First Posted : February 20, 2006
Last Update Posted : April 2, 2020
Sponsor:
Information provided by (Responsible Party):
Mesoblast, Ltd. ( Mesoblast, Inc. )

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.


Condition or disease Intervention/treatment Phase
Crohn's Disease Drug: Prochymal™ adult human mesenchymal stem cells Drug: adult human mesenchymal stem cells Phase 2

Detailed Description:

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. Patients will receive high or low dose. Study is open label.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Open-label, Randomized Study to Evaluate the Safety and Efficacy of PROCHYMAL™ IBD (ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Intravenous Infusion for the Treatment of Subjects Experiencing Moderate-to-severe Crohn's Disease That is Refractory to Steroids and Immune Suppressants
Actual Study Start Date : March 13, 2006
Actual Primary Completion Date : July 21, 2006
Actual Study Completion Date : July 21, 2006

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Crohn's Disease

Arm Intervention/treatment
Experimental: High dose
High dose (8 million cells per kg of body weight)
Drug: Prochymal™ adult human mesenchymal stem cells
Cells in plasmalyte and containing dimethylsulfoxide
Other Name: PROCHYMAL

Drug: adult human mesenchymal stem cells
two infusions, one week apart, each comprising adult human mesenchymal stem cells
Other Name: PROCHYMAL

Experimental: Low dose
Low dose: 2 million cells per kg body weight
Drug: Prochymal™ adult human mesenchymal stem cells
Cells in plasmalyte and containing dimethylsulfoxide
Other Name: PROCHYMAL

Drug: adult human mesenchymal stem cells
two infusions, one week apart, each comprising adult human mesenchymal stem cells
Other Name: PROCHYMAL




Primary Outcome Measures :
  1. Number of Participants with Reduction in Crohn's Disease Activity Index (CDAI) of at Least 100 Points [ Time Frame: 28 days ]
    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.


Secondary Outcome Measures :
  1. Number of Participants with Reduction in CDAI of at Least 70 points [ Time Frame: 28 days ]
    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.

  2. Improvement as Assessed by the Inflammatory Bowel Disease Questionnaire (IBDQ) [ Time Frame: 28 days ]
    The IBDQ measures disease-specific quality of life by assessing bowel symptoms, systemic symptoms, emotional function, and social function. Subscores can range from 1 (worst) to 7 (best). The total IBDQ is calculated as the sum of the responses to the individual IBDQ questions. The total score ranges from 32 to 224. An increase in score from baseline indicates improvement.

  3. Time to Improvement in IBDQ [ Time Frame: 28 days ]
  4. Number of Participants with Reduction of at Least 50% in Fistulas in Participants with Fistulas Draining Under Moderate Compression [ Time Frame: 28 days ]
  5. Number of Participants with Induction of Remission as Defined by Reduction of CDAI to Below 150 [ Time Frame: 28 days ]
    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.

  6. Time to Reduction in CDAI of at Least 100 Points [ Time Frame: 28 days ]
  7. Time to Reduction in CDAI of at Least 70 Points [ Time Frame: 28 days ]
  8. Time to Induction of Remission as Defined by Reduction of CDAI to Below 150. [ Time Frame: 28 days ]
  9. Number of Participants with Adverse Events [ Time Frame: Up to approximately 2 years ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Subject must be 18 to 70 years of age, inclusive.
  2. 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.
  3. Subject must have endoscopically or radiographically active Crohn's disease
  4. Subject must have a Crohn's disease activity index (CDAI) of at least 220.
  5. Subject must have a C-reactive protein (CRP) of at least 5 mg/l.
  6. Subject must have ileocolitis, colitis, or ileitis.
  7. 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.
  8. 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.
  9. 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.
  10. Subject must be available for all specified assessments at the study site through day 30.
  11. Subject must provide a written informed consent form (ICF) and authorization for use of and disclosure of personal health information (PHI).

Exclusion Criteria:

  1. Subject has any alcohol or substance abuse within 6 months of randomization.
  2. Subject has evidence of fibrostenotic obstructive Crohn's disease.
  3. Subject has an active infection with HIV or hepatitis B or C.
  4. Subject has had surgery or trauma within 28 d prior to enrollment.
  5. Subject has a known allergy to computed tomography (CT) contrast agents.
  6. Subject has a known allergy to bovine or porcine products.
  7. Subject has body mass greater than 150 kg.
  8. Subject has had a stricture of the bowel requiring hospitalization within 6 months prior to enrollment.
  9. Subject has had bowel surgery other than perianal (for example, fistulotomy, seton placement, or abscess drainage) within 6 months prior to enrollment.
  10. 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.
  11. Subject has received prednisone greater than 20 mg/d at any time 28 d prior to enrollment in study.
  12. Subject has a permanent colostomy or ileostomy.
  13. Subject has aspartate aminotransferase (AST), alkaline phosphatase (ALP), or alanine transaminase (ALT) more than 2.5 times the upper limit of normal at screening.
  14. 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.
  15. 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.
  16. Subject has received an investigational agent (IA)-an agent or device not approved by FDA for marketed use in any indication-within 90 d (or 5 half-lives, whichever is longer) of randomization.
  17. Subject has cardiopulmonary disease that, in the opinion of the Investigator, is either unstable or severe enough to justify exclusion from this study.
  18. Subject has any underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would make participation in the study unsafe.
  19. Subject has any underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the evaluation of the subject. Such excluding conditions might include, for example, uncontrolled infection, right heart failure, pulmonary hypertension.
  20. Subject has unstable arrhythmia.
  21. Subject is unwilling or unable to adhere to requirements of protocols.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00294112


Locations
Layout table for location information
United States, Louisiana
Osiris Clinical Site
Baton Rouge, Louisiana, United States, 70801
United States, North Carolina
Osiris Clinical Site
Charlotte, North Carolina, United States, 28105
United States, Pennsylvania
Osiris Clinical Site
Pittsburgh, Pennsylvania, United States, 15106
United States, Virginia
Osiris Clinical Site
Richmond, Virginia, United States, 23173
Sponsors and Collaborators
Mesoblast, Inc.
Investigators
Layout table for investigator information
Study Director: Mahboob Rahman, MD Mesoblast, Inc.
Layout table for additonal information
Responsible Party: Mesoblast, Inc.
ClinicalTrials.gov Identifier: NCT00294112    
Other Study ID Numbers: OSIRIS-601-602
First Posted: February 20, 2006    Key Record Dates
Last Update Posted: April 2, 2020
Last Verified: March 2020

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mesoblast, Ltd. ( Mesoblast, Inc. ):
mesenchymal stem cells
Additional relevant MeSH terms:
Layout table for MeSH terms
Crohn Disease
Inflammatory Bowel Diseases
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases
Remestemcel-l
Anti-Inflammatory Agents
Antiviral Agents
Anti-Infective Agents