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

• Register: ClinicalTrials.gov
• Last refreshed on: 24 February 2025
• Main ID: NCT03828344
• Date of registration: 30/01/2019
• Prospective Registration: Yes
• Primary sponsor: Baylx Inc.
• Public title: Safety and Tolerability of a Single Intravenous Infusion of BX-U001 in Refractory Rheumatoid Arthritis
• Scientific title: 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
• Date of first enrolment: December 1, 2025
• Target sample size: 16
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03828344
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female, aged 18 to 70, inclusive.

2. 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.

3. Have established RA > 6 months of symptoms

4. Have had an inadequate response or documented intolerance to available RA therapies
including csDMARDs and TNFi bDMARDs.

5. 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)

6. Have SJC of 4 or more out of 28 at screening and baseline

7. Have TJC of 4 or more out of 28 at screening and baseline

8. CRP greater than upper limit of normal (ULN)

9. Positive for RF and/or anti-CCP antibodies but without extra-articular disease or
functional limitations

10. Clinically stable with no significant changes in health status within 2 weeks prior
to randomization

11. Stated willingness to comply with all study procedures and availability for the
duration of the study

12. Patients must be informed of the investigational nature of this study and give
written informed consent in accordance with the institutional and hospital
guidelines.

Exclusion Criteria:

1. Infections of hepatitis B, hepatitis C, active or latent tuberculosis, or positive
for human immunodeficiency virus (HIV)1 or HIV2

2. 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.

3. Any active inflammatory diseases other than RA.

4. Serum aminotransferase (ALT or AST) levels > 2x ULN

5. 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

6. Chronic obstructive pulmonary disease or known lung disease except for mild asthma
treated with bronchodilators.

7. 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

8. History of transient ischemic attack

9. History of cerebrovascular accident (stroke)

10. Clinically significant heart disease (New York Heart Association, class III and
class IV).

11. Surgery or trauma within 14 days.

12. 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.

13. Washout period less than 6 months for rituximab or less than 4 weeks for other
bDMARDs.

14. 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.

15. Known allergies or had a history of allergy to blood products

16. Blood product usage within 50 days (except albumin)

17. Already participating in another interventional clinical trial or participated in
another interventional clinical trial within 3 months before screening.

18. Clinical history of malignancy with the exception of adequately treated cervical
carcinoma in situ or basal cell carcinomas.


Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Rheumatoid Arthritis

Intervention(s)

Biological: hUC-MSC suspension

Biological: Placebo

Primary Outcome(s)

Frequency of Adverse Events (AE) and Serious Adverse Events (SAE) [Time Frame: 12 months after infusion]

Secondary Outcome(s)

Change from Baseline of anti-cyclic citrullinated peptide (anti-CCP) at Week 12 and Week 24 [Time Frame: 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 [Time Frame: 12 weeks and 24 weeks after infusion]

Percentage of participants achieving ACR70 response from Baseline at Week 12 and Week 24 [Time Frame: 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 [Time Frame: 12 weeks and 24 weeks after infusion]

Percentage of participants achieving ACR50 response from Baseline at Week 12 and Week 24 [Time Frame: 12 weeks and 24 weeks after infusion]

Change from Baseline of rheumatoid factor at Week 12 and Week 24 [Time Frame: 12 weeks and 24 weeks after infusion]

Percentage of participants achieving remission by Simplified Disease Activity Index (SDAI) based criteria at Week 12 and Week 24 [Time Frame: 12 weeks and 24 weeks after infusion]

Percentage of participants achieving ACR20 from Baseline at Week 12 and Week 24 [Time Frame: 12 weeks and 24 weeks after infusion]

Secondary ID(s)

BXU001-RA

Source(s) of Monetary Support

Please refer to primary and secondary sponsors