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

NCT04971980 | Active Not Recruiting Phase 1

• 1 other identifier: BC-U001-RA2019
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Rheumatoid Arthritis

Keywords

mesenchymal stem cell; autoimmune disease

Outcome Measures

Primary Outcomes (8)

• 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.

  Up to day 28±3

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

  Up to day 28±3

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

  Up to day 28±3

• Changes of blood biochemical from day 1 to day 28±3

  Up to day 28±3

• Changes of coagulation function from day 1 to day 28±3

  Up to day 28±3

• Routine urine analysis

  Up to day 28±3

• Urine pregnancy test (female only)

  28±3 days

• Cardiac rate measured by twelve-lead electrocardiogram

  Up to day 28±3

Secondary Outcomes (11)

• Percentage of participants achieving ACR20

  Up to day 28±3

• Percentage of participants achieving ACR50

  Up to day 28±3

• Percentage of participants achieving ACR70

  Up to day 28±3

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

  Up to day 28±3

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

  Up to day 28±3

Study Arms (1)

hUC-MSC infusion (BC-U001)

EXPERIMENTAL

Cohort 1: Low-dose BC-U001 Cohort 2: Medium-dose BC-U001 Cohort 3: High-dose BC-U001

Drug: hUC-MSC infusion (BC-U001)

Interventions

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.

hUC-MSC infusion (BC-U001)

Eligibility Criteria

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

You may qualify if:

• 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

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 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
• 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

Sponsors & Collaborators

• Beijing Baylx Biotech Co., Ltd.: lead

Study Sites (1)

Peking Union Medical College Hospital (Dongdan campus)

Beijing, Beijing Municipality, 100730, China

Location

MeSH Terms

Conditions

Arthritis, Rheumatoid; Autoimmune Diseases

Condition Hierarchy (Ancestors)

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

Study Officials

• Xiaofeng Zeng, M.D., Ph.D.

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2021
• First Posted: July 22, 2021
• Study Start: April 12, 2021
• Primary Completion: July 10, 2023
• Study Completion: November 1, 2025
• Last Updated: September 19, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)