NCT03828344

Safety and Tolerability of a Single Intravenous Infusion of BX-U001 in Refractory Rheumatoid Arthritis

A Phase 1 Randomized, Placebo-Controlled, Double-Blind Study of the Safety and Tolerability of a Single Intravenous Infusion of BX-U001, a Human Umbilical Cord Tissue Derived Mesenchymal Stem Cell Product, for Refractory Rheumatoid Arthritis

• Status: Not Yet Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: Baylx Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 1, randomized, placebo-controlled, double-blind, single-dose, clinical trial examining the safety and biological effects of allogeneic fresh human umbilical cord tissue-derived mesenchymal stem cell product BX-U001, given by intravenous (IV) infusion, to rheumatoid arthritis (RA) patients with moderate to severe disease activity, who are not well controlled by their current treatments. Two doses of BX-U001 will be tested in 16 patients. The subjects will receive a one-time IV infusion of BX-U001 and monitored for 52 weeks.

Detailed description

This is a phase 1, randomized, placebo-controlled, double-blind, single-dose, clinical trial examining the safety and biological effects of allogeneic fresh human umbilical cord tissue-derived mesenchymal stem cell product BX-U001, given by IV infusion, to RA patients with moderate to severe disease activity, who are not well controlled by their current treatments. Two cohorts of patients will be recruited sequentially, and each cohort includes 8 patients. In each cohort, eligible patients will be randomized to the two treatment arms at the ratio of 3:1 (BX-U001: Placebo). The patients in the first cohort will receive a single infusion of BX-U001 at dose of 0.75×10\^6 cells/kg body weight or placebo. In the second cohort, the patients will receive a single infusion of BX-U001 at dose of 1.5×10\^6 cells/kg body weight or placebo. After the one-time infusion on Day 1, patients will be monitored for 52 weeks. During the study, besides BX-U001 or placebo infusion, patients will remain treated with their previous conventional synthetic disease modifying anti-rheumatic drugs (csDMARDs) and nonsteroidal anti-inflammatory drugs (NSAIDs) (other than biologics) in the same dosage/way. At the end of Week 12, the patients will undergo efficacy evaluations to determine their response to treatment using the response rate of American College of Rheumatology 20% improvement criteria (ACR20). For patients who lack response to treatment as defined by not achieving 20% improvement on tender joint count (TJC) and swollen joint count (SJC) at Week 12, they will receive standard of care treatment including csDMARDs, biologic DMARDs (bDMARDs) and adjunctive agents such as corticosteroids, NSAIDs, and analgesics at Week 14. The overall study duration is planned to be 2 years.

Conditions

• Rheumatoid Arthritis

Interventions

Type	Name	Description
BIOLOGICAL	hUC-MSC suspension	Patients will be treated at dose of 0.75×10\^6 cells/kg of body weight (Cohort 1) or 1.5×10\^6 cells/kg of body weight (Cohort 2) via a single IV infusion using a blood transfusion kit.
BIOLOGICAL	Placebo	Placebo contains the same cell suspension as BX-U001 but without cells.

Timeline

• Start date: 2026-12-01
• Primary completion: 2027-12-31
• Completion: 2028-02-20
• First posted: 2019-02-04
• Last updated: 2025-12-23

Regulatory

• FDA-regulated drug study