Efficacy and Safety of Adult Human Mesenchymal Stem Cells to Treat Steroid Refractory Acute Graft Versus Host Disease
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| ClinicalTrials.gov Identifier: NCT00366145 |
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Recruitment Status :
Completed
First Posted : August 21, 2006
Last Update Posted : April 6, 2020
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Graft Versus Host Disease | Biological: Mesenchymal Stem Cells Biological: placebo Drug: Standard of Care for GVHD | Phase 3 |
Approximately 6300 patients receive allogeneic hematopoietic stem cell transplants in the United States each year (IBMTR, 2003). Nearly 50% (approximately 3,150) of these patients develop acute GVHD (Goker et al). A fraction of these patients (approximately 870) will progress to the severe stages of the disease, Grades III-IV. It is estimated that nearly 82% of those patients with severe acute GVHD will be steroid refractory (Przepiorka et al., 1995) and of these, only 50% of steroid-refractory patients wll respond to secondary and tertiary treatments (Greinix et al., 2000). Thus, roughly 350 patients each year face tremendous odds against survival. In addition, most patients who initially responded to secondary and tertiary treatments have a high risk of dying within the first year (Remberger et al., 2001; Anasetti et al., 1994). Development of new therapeutic agents and strategies to rescue patients with steroid refractory, acute GVHD would provide a significant benefit in an area of unmet medical need.
Patients will receive standard of care in addition to adult mesenchymal stem cells or placebo.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 260 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase III, Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Prochymal™ (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Treatment of Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD |
| Actual Study Start Date : | August 17, 2006 |
| Actual Primary Completion Date : | December 26, 2008 |
| Actual Study Completion Date : | May 28, 2009 |
| Arm | Intervention/treatment |
|---|---|
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Active Comparator: Prochymal
Patients who receive standard of care plus treatment with ex vivo cultured adult human Mesenchymal Stem Cells
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Biological: Mesenchymal Stem Cells
2 Infusions of 2 million cells/kg per week for 4 weeks
Other Name: Prochymal Drug: Standard of Care for GVHD Institutionally defined standard of care (e.g., maintenance of steroid treatment and the addition of a second-line therapy). |
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Placebo Comparator: Placebo
Patients who receive standard of care and do not receive treatment with ex vivo cultured adult human mesenchymal stem cells.
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Biological: placebo
2 Infusions per week for 4 weeks
Other Name: excipients without adult human mesenchymal stem cells Drug: Standard of Care for GVHD Institutionally defined standard of care (e.g., maintenance of steroid treatment and the addition of a second-line therapy). |
- Percentage of Participants achieving Complete Response of Greater Than or Equal to 28 Days Duration [ Time Frame: up to 100 Days post first infusion ]A complete response was defined as complete resolution of all clinical signs of Graft versus host disease (GVHD)- that had to be maintained for at least 28 consecutive days (durable complete response [DCR]) within 100 days post first infusion.
- Overall Survival at 180 days Post First Infusion [ Time Frame: Day 180 ]Percentage of participants who survived at 180 days post first infusion.
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| Ages Eligible for Study: | 6 Months to 70 Years (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Subjects must be 6 months to 70 years of age, inclusive
- Subjects who have failed to respond to steroid treatment:
Failure to respond to steroid treatment is defined as any grade B-D (IBMTR grading) of acute GVHD that shows:
No improvement after 3 days and a duration of no greater than 2 weeks while receiving treatment with Methylprednisolone (greater than or equal to 1 mg/kg/day) or equivalent.
- Subjects must be treated within 4 days of randomization . In urgent situations 2nd line therapy may be started 24 hours prior to randomization , and Prochymal must be initiated within the following 3 days.
- Subjects who have received an increase in their steroid dose treatment prior to randomization will be eligible for enrollment. An increase in steroid dose will not be considered as second line therapy.
- Subjects must have adequate renal function as defined by: Calculated Creatinine Clearance of >30 mL/min using the Cockcroft-Gault equation
- For pediatric patients: Schwartz equation: (Patient population: infants over 1 week old through adolescence (<18 years old)
- Subjects who are women of childbearing potential, must be non-pregnant, not breast-feeding, and use adequate contraception. Male subjects must use adequate contraception
- Subject must have a minimum Karnofsky Performance Level of at least 30 at the time of study entry
- Subject (or legal representative where appropriate) must be capable of providing written informed consent.
Exclusion Criteria:
- Subject has started treatment with second line therapy >24 hours prior to randomization.
- Subject has received agents other than steroids for primary treatment of acute GVHD
- Subject is participating in the CTN Protocol 0302
- 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 including uncontrolled infection, heart failure, pulmonary hypertension, etc.
- Subjects may not receive any other investigational agents (not approved by the FDA) concurrently during study participation or within 30 days of randomization.
- Subject has a known allergy to bovine or porcine products.
- Subject has received a transplant for a solid tumor disease.
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): NCT00366145
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| Study Director: | Mahboob Rahman, MD | Mesoblast, Inc. |
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Mesoblast, Inc. |
| ClinicalTrials.gov Identifier: | NCT00366145 |
| Obsolete Identifiers: | NCT00476840 |
| Other Study ID Numbers: |
280 |
| First Posted: | August 21, 2006 Key Record Dates |
| Last Update Posted: | April 6, 2020 |
| Last Verified: | March 2020 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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acute GVHD steroid refractory GVHD severe steroid refractory acute GVHD steroid refractory Steroid Refractory Acute Graft Versus Host Disease |
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Graft vs Host Disease Immune System Diseases |