A Phase 2b/3a Study to Evaluate the Efficacy and Safety of JointStem in Patients Diagnosed as Knee Osteoarthritis

NCT04368806 | Recruiting Phase 2 FDA Drug

• 1 other identifier: JS-OAP3-US01
• Study Type: interventional
• Target: 140
• Locations: 2 countries
• Sites: 9

Brief Summary

This study is a double-blind, randomized, placebo controlled, multi-center, superiority study to evaluate the efficacy and safety of JointStem, autologous adipose tissue derived mesenchymal stem cells (AdMSC), in patients diagnosed as knee osteoarthritis. Following a 2-week screening period, approximately 140 subjects will be randomly assigned into one of the following two arms in a 1:1 ratio (1 JointStem : 1 Placebo). After each subject completes 48-Weeks visit (Visit 8) and the data management team confirms all individual data have no issue, the individual database will be locked and the blinding will be open for the statistical analysis.

Conditions

Osteoarthritis, Knee

Outcome Measures

Primary Outcomes (2)

• Western Ontario and McMaster Universities Arthritis Index (WOMAC) function Score

  Change from baseline in WOMAC function score at Week 48

  Baseline and Week 48

• Visual Analog Scale (VAS) score

  Change from baseline on Visual Analog Scale (VAS) score at Week 48

  Baseline and Week 48

Secondary Outcomes (8)

• Western Ontario and McMaster Universities Arthritis Index (WOMAC) subscale (Function)

  Week 12, 24 and 36

• Western Ontario and McMaster Universities Arthritis Index (WOMAC) subscale (Pain)

  Week 12, 24, 36, and 48

• Total Western Ontario and McMaster Universities Arthritis Index (WOMAC) score

  Week 12, 24, 36, and 48

• Visual Analog Scale (VAS) score

  Week 12, 24 and 36

• International Knee Documentation Committee (IKDC) score

  Week 12, 24, 36, and 48

Study Arms (2)

JointStem

EXPERIMENTAL

Autologous Adipose tissue derived Mesenchymal Stem Cells(AdMSC)

Drug: JointStem

Placebo

PLACEBO COMPARATOR

Normal Saline with Autologous Serum

Other: Placebo Control

Interventions

JointStem DRUG

Autologous Adipose tissue derived Mesenchymal Stem Cells (AdMSC)

JointStem

Placebo Control OTHER

Normal Saline with autologous Serum

Placebo

Eligibility Criteria

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

You may qualify if:

• Male or female of any race, adult aged 18 years or older
• Subject who had osteoarthritis of knee diagnosed (according to the clinical and American College of Rheumatology Criteria)
• Subject who has ≥ 45 on WOMAC function score at Screening and Baseline
• Subject who has knee pain ≥ 70 mm for the study knee out of 100 mm on VAS (Visual Analog Scale) at Screening and Baseline
• Subject who has radiographic evidence of grade 3 osteoarthritis in the study knee based on the Kellgren and Lawrence radiographic criteria
• Subject who has a varus angle of 5 degrees or less confirmed through radiography
• 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
• 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
• Subject who is willing and able to give written informed consent for participation in the study

You may not qualify if:

• Subject who has Body Mass Index (BMI) \> 35 kg/m2
• 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.
• 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)
• Subject who has any history of cancer and/or currently receiving treatment for a current cancer diagnosis.
• Subject who is positive in pathogenic test (HIV, viral hepatitis, or syphilis)
• 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
• 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
• Subject who has history of prolotherapy, or platelet rich plasma injection within 6 months prior to Screening
• Subject who has received long-acting hyaluronic acid injection (e.g., Synvisc-One®, etc.) within 6 months prior to Screening

Sponsors & Collaborators

• Nature Cell Co. Ltd.: lead

Study Sites (9)

TriWest Research Associates

El Cajon, California, 92020, United States

RECRUITING

BioSolutions Clinical Research Center

La Mesa, California, 91942, United States

RECRUITING

Neurovations Research

Napa, California, 94558, United States

RECRUITING

Newport Therapeutics

Newport Beach, California, 92660, United States

RECRUITING

Source Healthcare

Santa Monica, California, 90403, United States

RECRUITING

Keimyung University Dongsan Hospital

Daegu, Korea, Republic of, 42601, South Korea

RECRUITING

Kyung Hee University Medical Center

Seoul, Korea, Republic of, 02447, South Korea

RECRUITING

Kyung Hee University Hospital At Gangdong

Seoul, Korea, Republic of, 05278, South Korea

RECRUITING

Smg-Snu Boramae Medical Center

Seoul, Korea, Republic of, 07061, South Korea

RECRUITING

MeSH Terms

Conditions

Osteoarthritis, Knee

Condition Hierarchy (Ancestors)

Osteoarthritis; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Central Study Contacts

Hugh Lee

CONTACT

301-540-2600; hughlee@kcrnresearch.com

Christine Lee

CONTACT

301-540-2600; christinelee@kcrnresearch.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 21, 2020
• First Posted: April 30, 2020
• Study Start: May 26, 2021
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: July 24, 2025

Record last verified: 2025-07

Locations

US (5); KR (4)