JointStem injection for grade 3 knee osteoarthritis

A 48-Weeks, Phase 2b/3a, Double-Blind, Randomized, Placebo Controlled, Multi-center, Superiority Study to Evaluate the Efficacy and Safety of JointStem, Autologous Adipose Tissue Derived Mesenchymal Stem Cells in Patients Diagnosed as Knee Osteoarthritis

This will test whether JointStem—stem cells taken from your own fat—can reduce pain and improve function in adults with grade 3 knee osteoarthritis.

Quick facts

• Phase: Phase2; Phase3
• Study type: Interventional
• Enrollment: 140 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Nature Cell Co. Ltd. (Industry-sponsored)
• Drugs / interventions: prednisone
• Locations: 9 sites (El Cajon, California and 8 other locations)
• Trial ID: NCT04368806 on ClinicalTrials.gov

What this trial studies

This is a double-blind, randomized, placebo-controlled Phase 2b/3a study that gives either an intra-articular injection of autologous adipose-derived mesenchymal stem cells (JointStem) or a placebo. About 140 adults with radiographic grade 3 knee osteoarthritis will be randomized 1:1 after lipoaspiration and followed with visits through 48 weeks. Key clinical measures include pain and function scores (WOMAC and VAS), with safety monitoring throughout. Blinding will be maintained until the database is locked for statistical analysis.

Who should consider this trial

Good fit: Adults (18+) with radiographic grade 3 knee osteoarthritis, high baseline pain (≥70 mm VAS) and functional impairment (WOMAC function ≥45), varus angle ≤5°, at least 12 weeks of persistent symptoms, and willing to stop most OA pain meds are ideal candidates.

Not a fit: Patients with milder (grade 1–2) or end-stage (grade 4) knee osteoarthritis, varus deformity >5°, inflammatory arthritides, or who cannot stop prohibited medications are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, JointStem could provide meaningful pain relief and improved knee function using a patient’s own cells, potentially delaying the need for surgery.

How similar studies have performed: Some small early-phase and limited randomized studies of adipose-derived MSC injections have reported symptomatic improvements and acceptable safety, but larger confirmatory trials remain limited.

Eligibility criteria

\<Inclusion Criteria\>

1. Male or female of any race, adult aged 18 years or older
2. Subject who had osteoarthritis of knee diagnosed (according to the clinical and American College of Rheumatology Criteria)
3. Subject who has ≥ 45 on WOMAC function score at Screening and Baseline
4. Subject who has knee pain ≥ 70 mm for the study knee out of 100 mm on VAS (Visual Analog Scale) at Screening and Baseline
5. Subject who has radiographic evidence of grade 3 osteoarthritis in the study knee based on the Kellgren and Lawrence radiographic criteria
6. Subject who has a varus angle of 5 degrees or less confirmed through radiography
7. Subjects whose knee pain persists for at least 12 weeks (about 3 months) prior to Screening and does not improve symptoms with non-operative treatment options
8. Subject who is willing to discontinue all pain medications for osteoarthritis except rescue medication (acetaminophen \< 3.25 g per day) at least 72 hours prior to Screening and throughout the duration of study
9. Subject who is willing and able to give written informed consent for participation in the study

\<Exclusion Criteria\>

1. Subject who has Body Mass Index (BMI) \> 35 kg/m2
2. Subject judged by the investigator to have a history of clinically significant disease(s) (i.e., uncontrolled comorbid disease, kidney diseases, liver diseases, endocrine diseases, etc., but not limited to these diseases) that may affect the patient's participation in the study.
3. Subject who has any of following clinically significant diseases:

   * Autoimmune diseases
   * Paget's disease, ochronosis, acromegaly, hemochromatosis, or Wilson's disease
   * Genetic diseases (hyperkinesia, collagen gene abnormality, etc.)
   * Inflammatory joint disorders (e.g., rheumatoid inflammation)
   * Infectious joint disorders (e.g., septic arthritis)
   * Other joint disorders (e.g., gout, recurrent pseudogout, articular fracture, primary osteochondrosis, villonodular synovitis)
4. Subject who has any history of cancer and/or currently receiving treatment for a current cancer diagnosis.
5. Subject who is positive in pathogenic test (HIV, viral hepatitis, or syphilis)
6. Subject who has heart diseases (myocardial infarction, coronary artery bypass graft surgery, arrhythmia, or other serious heart diseases) or has history of heart diseases within 6 months prior to Screening
7. Subject who has received any intra-articular therapy in any joint within 6 months prior to Screening, or surgery on the relevant knee including articular endoscopic procedures within 6 months prior to Screening
8. Subject who has history of prolotherapy, or platelet rich plasma injection within 6 months prior to Screening
9. Subject who has received long-acting hyaluronic acid injection (e.g., Synvisc-One®, etc.) within 6 months prior to Screening
10. Subject who has history of stem cell therapy
11. Subject who has significant lab abnormalities for the following parameters (If the value is within 10% of the listed laboratory exclusion criterion value and the value is considered not to be clinically significant by the investigator, the subject can be considered for enrollment):

    * Serum ALT and AST \> 2 x upper limit of normal
    * Serum creatinine out of normal range
    * PT/INR out of normal range
    * Hemoglobin \< 10 g/dL for female subject and hemoglobin \< 11 g/dL for male subject
    * Platelets out of normal range
12. Subject for whom the investigator judges the lipoaspiration can cause any problem
13. Subject who has history of local anesthetic allergy
14. Subject who has taken anti-inflammatory drugs (prescription and non-prescription NSAIDs), symptomatic slow acting drugs (glucosamine, chondroitin sulfate, diacerhein etc.), or oral steroids (prednisone etc.) within 14 days prior to Screening (however, those who undergo a 14-day wash-out period can participate.)
15. Subject who is an active drug/alcohol abuser
16. Pregnant or breast-feeding women, or women or men who are not using appropriate method of contraception (appropriate method includes hormones, bilateral tubal ligation, and barrier method with spermicide, and intra-uterine device for women and vasectomy and barrier method with spermicide for men; subjects should agree to use appropriate method)
17. Subject who is enrolled in any other clinical trials within 3 months from screening
18. Subject who received the COVID-19 vaccines within 1 week from Visit 2 (Lipoaspiration, baseline)
19. Subject who the principal investigator considers inappropriate for the study due to any other reasons than those listed above

Where this trial is running

El Cajon, California and 8 other locations

• TriWest Research Associates — El Cajon, California, United States (Recruiting)
• BioSolutions Clinical Research Center — La Mesa, California, United States (Recruiting)
• Neurovations Research — Napa, California, United States (Recruiting)
• Newport Therapeutics — Newport Beach, California, United States (Recruiting)
• Source Healthcare — Santa Monica, California, United States (Recruiting)
• Keimyung University Dongsan Hospital — Daegu, Korea, South Korea (Recruiting)
• Kyung Hee University Medical Center — Seoul, Korea, South Korea (Recruiting)
• Kyung Hee University Hospital At Gangdong — Seoul, Korea, South Korea (Recruiting)
• Smg-Snu Boramae Medical Center — Seoul, Korea, South Korea (Recruiting)

Study contacts

• Study coordinator: Hugh Lee

Conditions Osteoarthritis, Knee