NCT02336230

A Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Participants Who Have Failed to Respond to Steroid Treatment for Acute Graft-Versus-Host Disease (aGVHD)

A Single-arm, Prospective Study of Remestemcel-L, Ex-vivo Culture-Expanded Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 55 (actual)
• Sponsor: Mesoblast, Inc. · Industry
• Sex: All
• Age: 2 Months – 17 Years
• Healthy volunteers: Not accepted

Summary

The study plans to treat at least 60 pediatric participants, male and female, between the ages of 2 months and 17 years inclusive with aGVHD following allogeneic hematopoietic stem cell transplant (HSCT) that has failed to respond to treatment with systemic corticosteroid therapy. Participants may have Grades C and D aGVHD involving the skin, liver and/or gastrointestinal (GI) tract or Grade B aGVHD involving the liver and/or GI tract, with or without concomitant skin disease.

Detailed description

Remestemcel-L will be evaluated in pediatric participants with aGVHD following allogeneic HSCT that has failed to respond to treatment with systemic corticosteroid therapy.

Conditions

• Grade B aGVHD
• Grade C aGVHD
• Grade D aGVHD

Interventions

Type	Name	Description
DRUG	remestemcel-L	Participants were treated with IV remestemcel-L at a dose of 2 x 10\^6 MSC/kg (actual body weight at screening) twice per week for each of 4 consecutive weeks. Infusions were administered at least 3 days apart and no more than 5 days apart for any infusion.

Timeline

• Start date: 2015-06-04
• Primary completion: 2018-04-09
• Completion: 2018-04-09
• First posted: 2015-01-12
• Last updated: 2022-03-17
• Results posted: 2022-03-17

Locations

20 sites across 1 country: United States

Regulatory

• FDA-regulated drug study