NCT04356287

Treatment With Human Umbilical Cord-derived Mesenchymal Stromal Cells in Systemic Sclerosis

Phase I/II Randomized Controlled Trial of Umbilical Cord-derived mesenChymAl stRomal cElls in Systemic Sclerosis

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Marie Hudson, MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to test the safety and efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells (UCMSC) for the treatment of Systemic Sclerosis (SSc).

Detailed description

A single-center, three-arm, randomized, double-blind, placebo-controlled trial is proposed. A total of 18 SSc patients will be enrolled in 3 successive blocks of 6 patients each. After being informed about the study and potential risks, all patients giving written informed consent will be randomized to one of two treatment arms or a placebo arm (total of 6 patients per arm). Within each block, the 6 patients will be randomized in a 2:2:2 ratio in one of the following arms: placebo, 1 infusion of UCMSC (M0), or 2 infusions of UCMSC (M0, M3). Second infusions of UCMSC will be performed only in the absence of Treatment Related Severe Adverse Events (TRSAE). Randomization into blocks 2 and 3 will be staggered, to allow the detection of TRSAE prior to inclusion of patients in a subsequent block, i.e. the second block will be randomized only in the absence of TRSAE one month after the first infusion of all 6 patients in block one, and similarly for the third block.

Conditions

• Sclerosis, Systemic
• Mesenchymal Stem Cells

Interventions

Type	Name	Description
BIOLOGICAL	UCMSC	Each infusion will consist of 1 million MSC/kg suspended in 50 mL of PlasmaLyte A.
OTHER	Placebo	Each infusion will consist of 50 mL of PlasmaLyte A.

Timeline

• Start date: 2023-01-05
• Primary completion: 2026-12-01
• Completion: 2027-12-01
• First posted: 2020-04-22
• Last updated: 2026-02-03

Locations

1 site across 1 country: Canada