- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04971980
Safety and Efficacy Study of Human Umbilical Cord-Derived Mesenchymal Stem Cells(BC-U001) for Rheumatoid Arthritis
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
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Anticipated)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Contact
- Name: Libo Zheng
- Phone Number: +86-010-56865685-2074
- Email: zhenglibo@blswinc.com
Study Contact Backup
- Name: Keling Cheng
- Email: chengkeling@blswinc.com
Study Locations
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Beijing
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Beijing, Beijing, China, 100730
- Recruiting
- Peking Union Medical College Hospital (Dongdan campus)
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
- Other situations that are not approporiate to participate in study in investigators' opinion
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: hUC-MSC infusion (BC-U001)
Cohort 1: Low-dose BC-U001 Cohort 2: Medium-dose BC-U001 Cohort 3: High-dose BC-U001
|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number and frequency of adverse events (AEs)
Time Frame: Up to day 28±3
|
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
Time Frame: Up to day 28±3
|
Up to day 28±3
|
|
Changes of complete blood count (CBC) from day 1 to day 28±3
Time Frame: Up to day 28±3
|
Up to day 28±3
|
|
Changes of blood biochemical from day 1 to day 28±3
Time Frame: Up to day 28±3
|
Up to day 28±3
|
|
Changes of coagulation function from day 1 to day 28±3
Time Frame: Up to day 28±3
|
Up to day 28±3
|
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Routine urine analysis
Time Frame: Up to day 28±3
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Up to day 28±3
|
|
Urine pregnancy test (female only)
Time Frame: 28±3 days
|
28±3 days
|
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Cardiac rate measured by twelve-lead electrocardiogram
Time Frame: Up to day 28±3
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Up to day 28±3
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Percentage of participants achieving ACR20
Time Frame: Up to day 28±3
|
Up to day 28±3
|
Percentage of participants achieving ACR50
Time Frame: Up to day 28±3
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Up to day 28±3
|
Percentage of participants achieving ACR70
Time Frame: Up to day 28±3
|
Up to day 28±3
|
Change from baseline of the disease activity score based on DAS28-CRP
Time Frame: Up to day 28±3
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Up to day 28±3
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Change from baseline of the disease activity score based on DAS28-ESR
Time Frame: Up to day 28±3
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Up to day 28±3
|
EULAR response
Time Frame: Up to day 28±3
|
Up to day 28±3
|
Change from baseline of the health assessment questionnaire(HAQ) score
Time Frame: Up to day 28±3
|
Up to day 28±3
|
Change from baseline of the simplified disease activity index (SDAI) score
Time Frame: Up to day 28±3
|
Up to day 28±3
|
Change from baseline of the clinical disease activity index (CDAI) score
Time Frame: Up to day 28±3
|
Up to day 28±3
|
Change from baseline of TNF-α level
Time Frame: Up to day 28±3
|
Up to day 28±3
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Change from baseline of IL-6 level
Time Frame: Up to day 28±3
|
Up to day 28±3
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Xiaofeng Zeng, M.D., Ph.D., Peking Union Medical College Hospital
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- BC-U001-RA2019
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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