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

NCT03828344 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: BXU001-RA
• Study Type: interventional
• Target: 16
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Rheumatoid Arthritis

Keywords

rheumatoid arthritis; mesenchymal stem cell; autoimmune

Outcome Measures

Primary Outcomes (1)

• Frequency of Adverse Events (AE) and Serious Adverse Events (SAE)

  Total number and rate of AEs and SAEs, related and non-related with BX-U001 infusion will be recorded as a measure of tolerability and safety.

  12 months after infusion

Secondary Outcomes (8)

• Percentage of participants achieving ACR20 from Baseline at Week 12 and Week 24

  12 weeks and 24 weeks after infusion

• Percentage of participants achieving ACR50 response from Baseline at Week 12 and Week 24

  12 weeks and 24 weeks after infusion

• Percentage of participants achieving ACR70 response from Baseline at Week 12 and Week 24

  12 weeks and 24 weeks after infusion

• Change from Baseline in the disease activity score 28-joint count using C reactive protein (DAS28-CRP) at Week 12 and Week 24

  12 weeks and 24 weeks after infusion

• Change from Baseline in the health assessment questionnaire disability index (HAQ-DI) score at Week 12 and Week 24

  12 weeks and 24 weeks after infusion

Study Arms (2)

hUC-MSC treatment

EXPERIMENTAL

BX-U001 (hUC-MSC suspension) will be tested at dose of 0.75 or 1.5×10\^6 cells/kg of body weight via a single IV infusion using a blood transfusion kit.

Biological: hUC-MSC suspension

Placebo control

PLACEBO COMPARATOR

The control arm will be given placebo which contains the same cell suspension solution but without cells. Placebo will be given the same way as BX-U001 via a single IV infusion using a blood transfusion kit.

Biological: Placebo

Interventions

hUC-MSC suspension BIOLOGICAL

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.

Also known as: BX-U001

hUC-MSC treatment

Placebo BIOLOGICAL

Placebo contains the same cell suspension as BX-U001 but without cells.

Placebo control

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, aged 18 to 70, inclusive.
• Have a diagnosis of RA in agreement with the 2010 ACR classification criteria: Sum of score equal or more than 6/10 in categories A-D including: A, Joint involvement; B, Serology; C, Acute-phase reactant; D, Duration of symptoms.
• Have established RA \> 6 months of symptoms
• Have had an inadequate response or documented intolerance to available RA therapies including csDMARDs and TNFi bDMARDs.
• Current use of csDMARD treatment for RA with at least one of the following: methotrexate (up to 25 mg daily), sulfasalazine (up to 3 g daily), hydroxychloroquine (up to 400mg daily), or leflunomide (up to 20mg daily), or any combination of these agents (with the exception of methotrexate and leflunomide) for at least 3 months, with a stable dose (including route of administration for methotrexate) for at least 6 weeks prior to the screening visit (Visit 0)
• Have SJC of 4 or more out of 28 at screening and baseline
• Have TJC of 4 or more out of 28 at screening and baseline
• CRP greater than upper limit of normal (ULN)
• Positive for RF and/or anti-CCP antibodies but without extra-articular disease or functional limitations
• Clinically stable with no significant changes in health status within 2 weeks prior to randomization
• Stated willingness to comply with all study procedures and availability for the duration of the study
• Patients must be informed of the investigational nature of this study and give written informed consent in accordance with the institutional and hospital guidelines.

You may not qualify if:

• Infections of hepatitis B, hepatitis C, active or latent tuberculosis, or positive for human immunodeficiency virus (HIV)1 or HIV2
• Any history of ongoing, significant infections or recent serious infection, i.e., requiring hospitalization and or IV antimicrobial treatment in the 3 months prior to screening.
• Any active inflammatory diseases other than RA.
• Serum aminotransferase (ALT or AST) levels \> 2x ULN
• Inadequate kidney function, defined as an estimated glomerular filtration rate (eGFR) \< 45 ml/min/1.73 m2 using Modification of Diet in Renal Disease (MDRD) 4-variable formula
• Chronic obstructive pulmonary disease or known lung disease except for mild asthma treated with bronchodilators.
• Any coexistent active major medical diagnosis of clinically significant cardiovascular, neurological psychiatric, renal, hepatic, immunological, endocrine (including uncontrolled diabetes or thyroid disease), or hematological abnormalities that are likely to interfere with patient compliance or study assessments/procedures in the investigators' opinion
• History of transient ischemic attack
• History of cerebrovascular accident (stroke)
• Clinically significant heart disease (New York Heart Association, class III and class IV).
• Surgery or trauma within 14 days.
• Pregnant, breastfeeding, or desire to become pregnant or unwilling to practice birth control during participation in the study and for twelve months after completing the study infusion, unless surgically sterilized or postmenopausal during the study.
• Washout period less than 6 months for rituximab or less than 4 weeks for other bDMARDs.
• Corticosteroid usage at a high dose (i.e., IV or IM corticosteroids or use of oral prednisone equivalent \>10 mg/day) or not at a stable dose for the treatment of RA or other diseases within 28 days prior to randomization.
• Known allergies or had a history of allergy to blood products

Sponsors & Collaborators

• Baylx Inc.: lead

MeSH Terms

Conditions

Arthritis, Rheumatoid

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 30, 2019
• First Posted: February 4, 2019
• Study Start (Estimated): December 1, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): February 20, 2028
• Last Updated: December 23, 2025

Record last verified: 2025-12