The goal of this clinical trial is to learn if allogeneic human bone marrow-derived mesenchymal stem cells (CG-BM1) are safe and show preliminary efficacy in treating patients with ankylosing spondylitis (AS). It will also explore the appropriate dose of CG-BM1.
The main questions it aims to answer are:
What medical problems (adverse events) do participants have when taking CG-BM1? (Safety and tolerability)
Does CG-BM1 improve disease activity, pain, and function in patients with AS? (Preliminary efficacy)
Researchers will compare CG-BM1 to a placebo (an inactive substance that looks like CG-BM1) in the second phase of the study to see if CG-BM1 works for AS.
This study has two phases:
Phase 1 (dose-escalation): Open-label, single-arm. Participants will receive one of three escalating doses of CG-BM1 weekly for 4 weeks.
Phase 2 (dose-expansion): Randomized, double-blind, placebo-controlled. Participants will receive either the recommended dose of CG-BM1 or a placebo weekly for 4 weeks, in addition to standard background therapy (celecoxib).
Participants will:
Receive CG-BM1 or placebo via intravenous infusion once a week for 4 weeks
Visit the clinic for follow-up assessments at Week 1, 4, 8, 12, and 24 after the first infusion
Undergo physical exams, laboratory tests (blood and urine), and complete questionnaires about disease activity, pain, and function (e.g., BASDAI, VAS, ASAS response criteria)
Meet the diagnostic criteria for ankylosing spondylitis (AS)
Age 18 to 40 years
Must be able to understand and communicate with the investigator, comply with study requirements, and provide signed and dated informed consent before any study assessments are performed
Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) total score ≥ 4 (0-10 scale), and total back pain measured by VAS ≥ 40 mm (0-100 mm)
Known allergy to any component of the study drug (primarily bone marrow mesenchymal stem cells; excipients include dimethyl sulfoxide, human albumin, etc.)
Current evidence of infection or malignancy as shown by chest X-ray or MRI within 3 months prior to screening
Currently using potent opioid analgesics
Received any intra-articular injection therapy (e.g., corticosteroids) within 4 weeks prior to randomization
Sequential
IV administration of CG-BM1
Biological/VaccineDose level 1: CG-BM1 1.0×10⁶ cells/kg, IV, weekly × 4 + celecoxib, 0.2g, p.o. daily Dose level 2: CG-BM1 2.0×10⁶ cells/kg, IV, weekly × 4 + celecoxib, 0.2g, p.o. daily Dose level 3: CG-BM1 4.0×10⁶ cells/kg, IV, weekly × 4 + celecoxib, 0.2g, p.o. daily
Placebo
Biological/VaccineSodium Chloride Solution, 5 ml, IV, weekly × 4+ celecoxib, 0.2g, p.o. daily
CG-BM1
Biological/VaccineCG-BM1 \[recommended dose\], IV, weekly × 4 + celecoxib, 0.2g, p.o. daily
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Incidence of Dose-Limiting Toxicities (DLTs)
Percentage of Participants Achieving ASAS20 Response
ASAS20 response is defined as an improvement of \> 20% and an absolute improvement of \> 1 unit (on a 0-10 scale) or \> 10 mm (on a 0-100 mm scale) in at least 3 of the following 4 domains: Patient Global Assessment, Physical Function (BASFI), Total Spinal Pain, and Inflammation (Morning Stiffness). Additionally, there must be no worsening in the remaining domain.
Global Assessment of Disease Activity
Evaluated using a 0-10 Visual Analog Scale (VAS), where 0 indicates "not active" and 10 indicates "very active". Higher scores represent greater disease activity. The outcome will report the change from baseline values at each subsequent visit timepoint.
Total Spinal Pain Intensity Score
Measured by a Visual Analog Scale (VAS) ranging from 0 mm (no pain) to 100 mm (severe pain), calculating the average score of overall spinal pain and nocturnal spinal pain over the past week. Higher scores represent worse pain intensity.
Bath Ankylosing Spondylitis Functional Index (BASFI)
BASFI consists of 10 items assessing the patient's ability to perform specific activities of daily living. Each item is scored on a 0-10 scale (0 = easy, 10 = impossible). The total score is calculated as the mean of the 10 scores, where a higher score indicates more severe functional impairment.
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Eighth Affiliated Hospital, Sun Yat-sen University
Lead sponsor
Guangzhou Cellgenes Biotechnology Co.,Ltd
Collaborator
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