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Rheumatoid ArthritisPhase 1/2NCT04971980

Safety and Efficacy Study of Human Umbilical Cord-Derived Mesenchymal Stem Cells(BC-U001) for Rheumatoid Arthritis

A Phase 1/2 study testing hUC-MSC infusion (BC-U001) in people with Rheumatoid Arthritis, run by Beijing Baylx Biotech Co., Ltd. at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

BC-U001 is an allogeneic fresh human umbilical cord-derived mesenchymal stem cell product, which showed therapeutic potential for rheumatoid arthritis(RA) based on its anti-inflammatory, immunomodulatory and tissue repair activities. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Rheumatoid Arthritis
What is being tested?
hUC-MSC infusion (BC-U001)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
1 siteChina
Study dates
12 Apr 2021to Nov 2025
Phase
Phase 1/2
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 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female patients aged 18 to 65 (inclusive)
  • Patients who are diagnosed with rheumatoid arthritis following the 2010 ACR/EULAR classification criteria
  • Patients who still remain moderate-to-high disease activity, i.e. DAS28>3.2 at screening and study baseline, after standard csDMARDs therapy
  • Patients who are positive for rheumatoid factors (RF) and/or anti-CCP antibody
  • Patients who are clinically stable with no significant changes in physical condition from screening to study baseline
  • Patients who are available and willing to comply with all study procedures
  • 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 upper limits of normal
  • Creatinine clearance rate (Ccr) < 45 ml/min calculated by Cockcroft-Gault formula
  • Severe chronic obstructive pulmonary disease or known lung disease except for mild asthma treated with bronchodilators
Show all 16Show fewer
  • 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 cerebrovascular accident (stroke) within 1 year before screening
  • Clinically significant heart disease (New York Heart Association, class III and class IV)
  • Surgery or trauma (e.g. contusions, abrasions, stab wounds, cutting wounds, crush injuries, impact injuries, and firearm injuries etc.) within 14 days before enrollment that are not approporiate to participate in study in investigators' opinion
  • 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
  • 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 minor molecular heparinum and human serum albumin that are likely to interfere with patient compliance or study assessments/procedures in the investigators' opinion
  • 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
  • Other situations that are not approporiate to participate in study in investigators' opinion

Trial locations and contacts

1 site in 1 country.

China1 site

  • Peking Union Medical College Hospital (Dongdan campus)

    Beijing, Beijing Municipality, 100730

Other trials in China

About this study

Official title: A Phase 1/2a, Open-Label Study to Evaluate the Safety and Efficacy of a Single Intravenous Infusion of BC-U001, a Human Umbilical Cord-Derived Mesenchymal Stem Cell Product, for Rheumatoid Arthritis

BC-U001 is an allogeneic fresh human umbilical cord-derived mesenchymal stem cell product, which showed therapeutic potential for rheumatoid arthritis(RA) based on its anti-inflammatory, immunomodulatory and tissue repair activities. The primary objective of this open-label, non-randomized, dose-escalation study is to evaluate the safety and tolerability of a single intravenous infusion of BC-U001 for RA patients using a 3+3 design.

Read the detailed description

This study will enroll RA patients who still remained moderate-to-high disease activity after conventional synthetic DMARDs (csDMARDs) therapy. All participants are informed about the study procedures and potential risks and are required to provide written informed consent prior to study begin.

A 3+3 dose escalation design will be implemented. Three ascending dose cohorts (3 participants/cohort) will be treated successively to identify the maxium tolerated dose (MTD) and/or a recommended dose for phase II study. Dose escalation will be terminated if the dose-limiting toxicities (DLT) are observed in 2 participants during the 28-day follow-up within cohort.

DLT was defined as any ≥grade 3 non-hematological toxicity or grade 4 hemtological toxicity according to CTCAE v5.0, which was related to the investigational product determined by investigator.

Being tested
hUC-MSC infusion (BC-U001)
Keywords
mesenchymal stem cell, autoimmune disease
Medical subject headings
Arthritis, Rheumatoid, Autoimmune Diseases

What participants receive

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

  • hUC-MSC infusion (BC-U001)Drug

    The participants are intravenously administered a single infusion of hUC-MSC at 0.5x10^6 cells/kg body weight, 1.0x10^6 cells/kg body weight, 1.5x10^6 cells/kg body weight for low-dose cohort, medium-dose cohort and high-dose cohort respectively.

Study groups in NCT04971980
Study groupRoleWhat they receive
hUC-MSC infusion (BC-U001)ExperimentalCohort 1: Low-dose BC-U001 Cohort 2: Medium-dose BC-U001 Cohort 3: High-dose BC-U001
Receives: Drug: hUC-MSC infusion (BC-U001)

What is being measured

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

  1. Number and frequency of adverse events (AEs)

    Adverse events are assessed by CTCAE 5.0 up to 28±3 days. The flexible change of ±3 days is set for the convenience of patients. Day 1 refers to the day the participant accept BC-U001 infusion.

    Measured: Up to day 28±3

  2. Changes of vital signs from 1 hour after infusion to day 28±3

    Measured: Up to day 28±3

  3. Changes of complete blood count (CBC) from day 1 to day 28±3

    Measured: Up to day 28±3

  4. Changes of blood biochemical from day 1 to day 28±3

    Measured: Up to day 28±3

  5. Changes of coagulation function from day 1 to day 28±3

    Measured: Up to day 28±3

  6. Routine urine analysis

    Measured: Up to day 28±3

  7. Urine pregnancy test (female only)

    Measured: 28±3 days

  8. Cardiac rate measured by twelve-lead electrocardiogram

    Measured: Up to day 28±3

Secondary outcomes (11)

  1. Percentage of participants achieving ACR20

    Measured: Up to day 28±3

  2. Percentage of participants achieving ACR50

    Measured: Up to day 28±3

  3. Percentage of participants achieving ACR70

    Measured: Up to day 28±3

  4. Change from baseline of the disease activity score based on DAS28-CRP

    Measured: Up to day 28±3

  5. Change from baseline of the disease activity score based on DAS28-ESR

    Measured: Up to day 28±3

  6. EULAR response

    Measured: Up to day 28±3

  7. Change from baseline of the health assessment questionnaire(HAQ) score

    Measured: Up to day 28±3

  8. Change from baseline of the simplified disease activity index (SDAI) score

    Measured: Up to day 28±3

  9. Change from baseline of the clinical disease activity index (CDAI) score

    Measured: Up to day 28±3

  10. Change from baseline of TNF-α level

    Measured: Up to day 28±3

  11. Change from baseline of IL-6 level

    Measured: Up to day 28±3

Timeline

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

  1. First posted22 Jul 2021
  2. Study start12 Apr 2021
  3. Primary completion10 Jul 2023
  4. Study completionNov 2025 (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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
9 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04971980
Sponsor's protocol ID
BC-U001-RA2019
Status last verified
Feb 2025
First submitted
30 Jun 2021
Last updated
19 Sep 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing hUC-MSC infusion (BC-U001) in people with Rheumatoid Arthritis, run by Beijing Baylx Biotech Co., Ltd. at 1 site in China.

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 7 inclusion and 16 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: under way, not looking for participants, as of the sponsor's last update on 19 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

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

What does this combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Number and frequency of adverse events (AEs) (time frame: Up to day 28±3). The study lists 8 primary outcomes in all. 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 NCT04971980.

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 19 Sep 2025.

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