Safety and Efficacy of Allogeneic Bone Marrow MSCs in Ankylosing Spondylitis

NCT07632599 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: MR-44-26-023756
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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)

Conditions

Ankylosing Spondylitis

Outcome Measures

Primary Outcomes (2)

• Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

  24 weeks

• Incidence of Dose-Limiting Toxicities (DLTs)

  24 weeks

Secondary Outcomes (11)

• Percentage of Participants Achieving ASAS20 Response

  Weeks 1, 4, 8, 12, and 24

• Global Assessment of Disease Activity

  Weeks 1, 4, 8, 12, and 24

• Total Spinal Pain Intensity Score

  Weeks 1, 4, 8, 12, and 24

• Bath Ankylosing Spondylitis Functional Index (BASFI)

  Weeks 1, 4, 8, 12, and 24

• Bath Ankylosing Spondylitis Disease Activity Index (BASDAI)

  Weeks 1, 4, 8, 12, and 24

Study Arms (3)

phase 1：CG-BM1 Dose Escalation Cohorts

EXPERIMENTAL

Participants will be sequentially assigned to one of three dose cohorts using a "3+3" design: low-dose (1\*10\^6 cells/kg), medium-dose (2.0\*10\^6 cells/kg), or high-dose 4.0\*10\^6 cells/kg). In each cohort, CG-BM1 will be administered via intravenous infusion once weekly for a total of 4 doses on Days 0, 7, 14, and 21. All participants across all cohorts will concurrently receive standard background therapy consisting of celecoxib 0.2g orally once daily.

Biological: IV administration of CG-BM1

phase 2 ：placebo Cohorts

PLACEBO COMPARATOR

Participants will be randomized to receive the placebo (Compound Electrolyte Injection) via intravenous (IV) infusion once weekly for a total of 4 doses, mimicking the regimen of the experimental group. All participants will simultaneously receive a background therapy of celecoxib 0.2g orally once daily.

Biological: Placebo

phase 2 ：CG-BM1 Cohorts

EXPERIMENTAL

Participants will be randomized to receive CG-BM1 at the clinically recommended dose (determined by the Safety Review Committee based on Phase 1 results) via intravenous (IV) infusion once weekly for a total of 4 doses. All participants will simultaneously receive a background therapy of celecoxib 0.2g orally once daily.

Biological: CG-BM1

Interventions

IV administration of CG-BM1 BIOLOGICAL

Dose 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

phase 1：CG-BM1 Dose Escalation Cohorts

Placebo BIOLOGICAL

Sodium Chloride Solution, 5 ml, IV, weekly × 4+ celecoxib, 0.2g, p.o. daily

phase 2 ：placebo Cohorts

CG-BM1 BIOLOGICAL

CG-BM1 \[recommended dose\], IV, weekly × 4 + celecoxib, 0.2g, p.o. daily

phase 2 ：CG-BM1 Cohorts

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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)
• CRP or ESR elevated ≥ 1.5 times the upper limit of normal
• Patients taking methotrexate (≤ 25 mg/week) or sulfasalazine (≤ 3 g/day) are permitted to continue these medications, provided they have been used for at least 3 months and maintained at a stable dose for at least 4 weeks prior to randomization. Patients taking methotrexate must maintain stable folic acid supplementation prior to randomization
• Patients taking DMARDs other than methotrexate and sulfasalazine must discontinue them at least 4 weeks prior to randomization
• No prior use of any form of biologics within 6 months
• Spinal X-ray must rule out complete rigid ankylosis

You may not qualify if:

• 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
• Received any intramuscular corticosteroid injection within 2 weeks prior to randomization
• Received traditional Chinese medicine treatment for AS within 4 weeks prior to randomization
• Pregnant or breastfeeding women
• Presence of underlying metabolic, hematologic, renal, hepatic, pulmonary, neurologic, endocrine, cardiac, infectious, or gastrointestinal disease that, in the investigator's opinion, would place the patient at unacceptable risk if treated with immunomodulatory agents
• Significant health problem or disease including (but not limited to): uncontrolled hypertension (≥ 160/95 mmHg), congestive heart failure, uncontrolled diabetes, or extremely poor functional status rendering the patient unable to care for themselves
• History of renal impairment, glomerulonephritis, or a single kidney, or serum creatinine level \> 1.5 mg/dL
• Active systemic infection within 2 weeks prior to randomization (common cold excluded)
• Current infection or history of chronic, recurrent infectious disease, or clinical test suggesting tuberculosis (including latent tuberculosis)
• Known HIV infection, hepatitis B, or hepatitis C at screening or randomization
• History or evidence of alcohol or drug abuse within 6 months prior to randomization
• History of lymphoproliferative disease or known malignancy of any organ system within the past 5 years

Sponsors & Collaborators

• Guangzhou Cellgenes Biotechnology Co.,Ltd: collaborator
• Eighth Affiliated Hospital, Sun Yat-sen University: lead

Study Sites (1)

The Eighth Affiliated Hospital, Sun Yat-sen University

Shenzhen, Guangdong, 518033, China

Location

MeSH Terms

Conditions

Spondylitis, Ankylosing

Condition Hierarchy (Ancestors)

Axial Spondyloarthritis; Spondylarthropathies; Spondylarthritis; Spondylitis; Spinal Diseases; Bone Diseases; Musculoskeletal Diseases; Ankylosis; Joint Diseases; Arthritis

Central Study Contacts

Prof.Wang

CONTACT

86+138 2602 4785; wangp57@mail.sysu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2026
• First Posted: June 8, 2026
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: June 8, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)