NCT00366145

Efficacy and Safety of Adult Human Mesenchymal Stem Cells to Treat Steroid Refractory Acute Graft Versus Host Disease (GVHD)

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 260 (actual)
• Sponsor: Mesoblast, Inc. · Industry
• Sex: All
• Age: 6 Months – 70 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy and gather additional safety information for Prochymal® in participants who have failed to respond to steroid treatment of Grades B-D acute GVHD.

Detailed description

Approximately 6300 patients receive allogeneic hematopoietic stem cell transplants in the United States each year (International Bone Marrow Transplant Registry \[IBMTR\], 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 will 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. Participants will receive standard of care in addition to adult mesenchymal stem cells or placebo.

Conditions

• Graft Versus Host Disease

Interventions

Type	Name	Description
BIOLOGICAL	Prochymal®	2 infusions of 2 million cells/kg per week for 4 weeks
DRUG	Placebo	2 infusions per week for 4 weeks
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)

Timeline

• Start date: 2006-08-17
• Primary completion: 2008-12-26
• Completion: 2009-05-28
• First posted: 2006-08-21
• Last updated: 2022-02-10

Locations

70 sites across 6 countries: United States, Australia, Canada, Italy, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study