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Rheumatoid ArthritisPhase 1NCT03828344

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

A Phase 1 study testing hUC-MSC suspension in people with Rheumatoid Arthritis, run by Baylx Inc..

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Rheumatoid Arthritis
What is being tested?
hUC-MSC suspension
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
16 participants
Where
No sites listed yet
Study dates
1 Dec 2026to 20 Feb 2028
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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.

Who cannot join

  • 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.
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  • 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
  • Blood product usage within 50 days (except albumin)
  • Already participating in another interventional clinical trial or participated in another interventional clinical trial within 3 months before screening.
  • Clinical history of malignancy with the exception of adequately treated cervical carcinoma in situ or basal cell carcinomas.

About this study

Official 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

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.

Read the 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.

Being tested
hUC-MSC suspension, Placebo
Keywords
rheumatoid arthritis, mesenchymal stem cell, autoimmune
Medical subject headings
Arthritis, Rheumatoid

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • hUC-MSC suspensionBiological

    Also called BX-U001

    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.

  • PlaceboBiological

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

Study groups in NCT03828344
Study groupRoleWhat they receive
hUC-MSC treatmentExperimentalBX-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.
Receives: Biological: hUC-MSC suspension
Placebo controlPlacebo comparatorThe 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.
Receives: Biological: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 12 months after infusion

Secondary outcomes (8)

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

    The ACR20 is a composite measure defined as both improvement of 20% in the number of tender and number of swollen joints, and a 20% improvement in three of the following five criteria: patient global assessment, physician global assessment, functional ability measure [Health Assessment Questionnaire (HAQ)], visual analog pain scale, and erythrocyte sedimentation rate or C-reactive protein (CRP).

    Measured: 12 weeks and 24 weeks after infusion

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

    The ACR50 is a composite measure defined as both improvement of 50% in the number of tender and number of swollen joints, and a 50% improvement in three of the following five criteria: patient global assessment, physician global assessment, functional ability measure [Health Assessment Questionnaire (HAQ)], visual analog pain scale, and erythrocyte sedimentation rate or C-reactive protein (CRP).

    Measured: 12 weeks and 24 weeks after infusion

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

    The ACR70 is a composite measure defined as both improvement of 70% in the number of tender and number of swollen joints, and a 70% improvement in three of the following five criteria: patient global assessment, physician global assessment, functional ability measure [Health Assessment Questionnaire (HAQ)], visual analog pain scale, and erythrocyte sedimentation rate or C-reactive protein (CRP).

    Measured: 12 weeks and 24 weeks after infusion

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

    The DAS28-CRP is a composite measure of inflammation in Rheumatoid Arthritis and incorporates a tender and swollen joint count, CRP and Patient Global Assessment of Disease Activity expressed in a Gaussian distribution of variables ranging from 0 to 10. A DAS28-CRP score of <3.2 suggests a low level of disease activity, while a score of >5.1 suggests a high level of disease activity.

    Measured: 12 weeks and 24 weeks after infusion

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

    The HAQ-DI questionnaire assesses the participant's self-perception on the degree of difficulty when performing daily activities. Each question asks on a scale ranging from 0 to 3 if the categories can be performed without any difficulty (scale 0) up to cannot be done at all (scale 3).

    Measured: 12 weeks and 24 weeks after infusion

  6. Percentage of participants achieving remission by Simplified Disease Activity Index (SDAI) based criteria at Week 12 and Week 24

    The SDAI is the numerical sum of five outcome parameters: tender joint count and swollen joint count based on a 28-joint assessment, patient global health assessment and physician global health assessment (from 0=best to 10=worst), and C-reactive protein (CRP). SDAI total score= 0 to 86. SDAI <=3.3 indicates disease remission, >3.3 to 11 = low disease activity, >11 to 26 = moderate disease activity, and >26 = high disease activity.

    Measured: 12 weeks and 24 weeks after infusion

  7. Change from Baseline of rheumatoid factor at Week 12 and Week 24

    Rheumatoid factor blood test result.

    Measured: 12 weeks and 24 weeks after infusion

  8. Change from Baseline of anti-cyclic citrullinated peptide (anti-CCP) at Week 12 and Week 24

    Anti-CCP blood test result.

    Measured: 12 weeks and 24 weeks after infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Feb 2019
  2. Study start1 Dec 2026 (estimated)
  3. Primary completion31 Dec 2027 (estimated)
  4. Study completion20 Feb 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
16 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03828344
Sponsor's protocol ID
BXU001-RA
Status last verified
Dec 2025
First submitted
30 Jan 2019
Last updated
23 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing hUC-MSC suspension in people with Rheumatoid Arthritis, run by Baylx Inc..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 23 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Frequency of Adverse Events (AE) and Serious Adverse Events (SAE) (time frame: 12 months after infusion). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03828344.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Dec 2025.

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