Phase 3 Pivotal Trial Comparing CARTISTEM® and Surgical Comparator for Knee Cartilage Lesions and Osteoarthritis

NCT07339111 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: MPA-CARTISTEM-2023-01
• Study Type: interventional
• Target: 300
• Locations: 1 country
• Sites: 16

Brief Summary

To establish the efficacy and safety of CARTISTEM®, a combination product composed of allogeneic human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs) formulated with a cross-linked sodium hyaluronate (HA) hydrogel, CARTISTEM® compared to the surgical comparator of debridement in Subjects with knee cartilage lesions and osteoarthritis. This trial is intended to provide evidence of the superiority of CARTISTEM® to reduce knee pain and improve knee function compared to debridement for Subjects with knee cartilage lesions and osteoarthritis at 2-years post-treatment. Additionally, this trial is intended to intended to explore whether CARTISTEM® may have disease-modifying effects on osteoarthritis progression through the use of semi-quantitative MRI assessment.

Conditions

Knee Cartilage Defects

Keywords

symptomatic knee cartilage defects; knee cartilage; cartilage repair; osteoarthritis

Outcome Measures

Primary Outcomes (2)

• Visual Analog Scale (VAS) Pain Score

  Change from Baseline Visual Analog Scale (VAS) Pain score (0-100 point scale, 0 being no pain at one end and 100 being the worst imaginable pain at the other) in index knee comparing CARTISTEM® to surgical control at 24-months post-treatment.

  From enrollment to end of follow-up at 24 months

• Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC®) Function score

  Change from Baseline Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC®) Function score (0-100 point scale, 0 being the best functioning and 100 being the worst functioning) in index knee comparing CARTISTEM® to surgical control at 24-months post-treatment.

  From start of treatment to the end of follow-up at 24 months

Secondary Outcomes (2)

• Modified Magnetic Resonance Observation of Cartilage Repair Tissue (MOCART) Score

  enrollment to follow-up at 24 months.

• Change in MRI Osteoarthritis Knee Score (MOAKS) cartilage morphology

  enrollment to follow up at 24 months

Other Outcomes (10)

• Change in Western Ontario and McMaster Universities OA Index (WOMAC®) Total score (0-100 point scale, 0 being no osteoarthritis symptoms and 100 being the worst symptoms) in index knee comparing CARTISTEM® to surgical control at 24-months post-treatment.

  enrollment to follow up at 24 months

• Change in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC®) Stiffness score (0-100 point scale, 0 being the no stiffness, 100 being the most) in index knee comparing CARTISTEM® to surgical control at 24-months post-treatment.

  enrollment to follow up at 24 months

• Change in EuroQoL 5 Dimensions, 5 Levels (5D-5L, with 0 being the worst and 100 being the best) comparing CARTISTEM® to surgical control at 24-months post-treatment.

  enrollment at follow up at 24 months

Study Arms (2)

CARTISTEM + Debridement

EXPERIMENTAL

Participants in Group A will receive CARTISTEM + Debridement in the target knee.

Biological: CARTISTEM® + Debridement

Debridement

ACTIVE COMPARATOR

Participants in Group B will receive debridement procedure.

Procedure: Debridement

Interventions

Debridement PROCEDURE

Debridement of unstable cartilage in and around cartilage lesion(s) in the knee.

Debridement

CARTISTEM® + Debridement BIOLOGICAL

CARTISTEM® + Debridement CARTISTEM® consists of approximately 7.5 x 106 hUCB-MSCs (human umbilical cord blood-derived mesenchymal stem/stromal cells) suspended in 1.5 ml medium in one vial, and 60 mg sodium hyaluronate provided as a lyophilized powder in a separate vial. A viscous and malleable gel matrix is formed by mixing hUCB-MSCs (main component) and 60mg of sodium hyaluronate at a 4% concentration (excipient).

CARTISTEM + Debridement

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 and ≤ 75 years old.
• Knee Osteoarthritis diagnosed in the index knee according to the clinical definition of American College of Rheumatology (ACR) guidelines at screening.
• Failed conservative knee osteoarthritis treatment for at least 3 months prior to consent (weight reduction, physical therapy, injections, pain medications, etc.).
• Kellgren-Lawrence (KL) in the index knee of grade 2 to 3 as determined by independent radiologist review of study X-Ray.
• Clinically relevant femoral cartilage defects classified using modified Outerbridge Grade 3 or 4 in index knee with combined lesion area ≥ 2 cm2 and ≤ 9 cm2 and at least a single femoral lesion ≥ 2 cm2 as determined by independent radiologist review of study MRI.
• Body Mass Index (BMI) ≤ 35 kg / m2 .
• VAS PAIN index knee score ≥ 40 to ≤ 90 based on 100-point scale at screening.
• VAS PAIN contralateral knee score ≤ 30 based on 100-point scale at screening.
• WOMAC® FUNCTION index knee mean score ≥ 25 to ≤ 90 based on 100-point scale at screening.
• WOMAC® Pain index knee mean score ≥ 40 to ≤ 90 based on 100-point VAS (visual analogue scale) at screening.
• Subjects must be willing to abstain from treatments in the index knee from the time the informed consent is signed until after the last study follow up visit.
• Subjects must be willing to discontinue analgesics except:
• Rescue medication (acetaminophen) allowed for break-through pain throughout the study.
• Subjects must be willing to discontinue use of all pain medications at least 48 hours prior to baseline assessment and each follow up visit.
• Limited postoperative NSAID use is permissible per IND clinical guidance and site manual, provided washout before scheduled efficacy assessments.

You may not qualify if:

• Subject received an IA injection of Hyaluronic Acid (HA), Platelet Rich Plasma (PRP), Bone Marrow Aspirate / Concentrate (BMA / BMAC), long-acting / conventional steroid, or investigational drug in either knee within 3 months of signing informed consent. Note: injections of the contralateral knee are allowed during the trial.
• Bone marrow lesion (BML) grade 3 on femur, tibia, or patella except at femoral lesion area (BML grade 3 allowed) in index knee as determined by independent radiologist review of study MRI.
• KL index knee grade 0, 1 or 4 as determined by independent radiologist review of study X-Ray.
• KL Contralateral knee severity grade 4 as determined by independent radiologist review of study X-Ray.
• Anatomical axis varus or valgus malalignment ≥ 6° in either knee as determined by independent radiologist.
• Joint ligament instability in index knee ≥ Grade 3 or ligament instability that would require surgical intervention as determined by investigator or designee.
• Complete meniscal deficiency, meniscal root tears and / or severe meniscal extrusion as determined by independent radiologist.
• Kissing bipolar lesions (tibial or patellar) Outerbridge 4 exceeding 25% of the femoral lesion area in size and/or penetrating through the subchondral bone as determined by independent radiologist.
• Surgery of the index knee joint within 6 months of signing the informed consent. Investigator confirms that Subject has recovered from any prior surgical intervention to enable treatment within this study protocol. No radiation therapy of the index knee as determined by documented medical history.
• Chronic inflammatory articular diseases such as rheumatoid arthritis, gout, pseudogout, gouty arthritis, or fibromyalgia as determined by documented medical history.
• Joint disorders of the index knee including infection, osteomyelitis, soft tissue tumors, and bone marrow infiltration as determined by independent radiologist review of study MRI or X-ray.
• Diseases or disorders including Paget's disease, ochronosis, acromegaly, haemochromatosis, Wilson's disease, genetic disease (hyperkinesia, etc.), or genetic collagen disorder as determined by documented medical history.
• Subject is taking anticoagulant medication (e.g., warfarin, (Coumadin), apixaban (Eliquis), rivaroxaban (Xarelto), clopidogrel (Plavix) or equivalent) but not excluding 81mg aspirin.
• Uncontrolled diabetes with HbA1c \> 8% as determined by documented medical history.
• Infectious disease requiring parenteral administration of antibiotics.

Sponsors & Collaborators

• Medipost, Inc.: lead

Study Sites (16)

Flourish Research Birmingham

Birmingham, Alabama, 35205, United States

NOT YET RECRUITING

AZ Orthopedic

Tempe, Arizona, 85224, United States

RECRUITING

TriWest Research Associates

El Cajon, California, 91910, United States

NOT YET RECRUITING

Horizon Clinical Research

La Mesa, California, 91924, United States

NOT YET RECRUITING

Source Healthcare

Santa Monica, California, 90403, United States

RECRUITING

Orthopedic Centers of Colorado

Parker, Colorado, 80134, United States

RECRUITING

Steamboat Orthopaedic and Spine Institute

Steamboat Springs, Colorado, 80487, United States

RECRUITING

Curalta Clinical Trials

Westwood, New Jersey, 07662, United States

RECRUITING

Atrium Health Mercy

Charlotte, North Carolina, 28207, United States

NOT YET RECRUITING

West Clinical Research

Morehead City, North Carolina, 28557, United States

RECRUITING

University of Cincinnati Department of Orthopaedic Surgery

Cincinnati, Ohio, 45267, United States

NOT YET RECRUITING

Zenos Clinical Research

Dallas, Texas, 75230, United States

RECRUITING

Memorial Hermann Center

Houston, Texas, 77043, United States

RECRUITING

HD Research Corp.

Houston, Texas, 77401, United States

RECRUITING

Flourish Research San Antonio

San Antonio, Texas, 78229, United States

RECRUITING

JBR Clinical Research

Salt Lake City, Utah, 84107, United States

RECRUITING

MeSH Terms

Conditions

Osteoarthritis

Interventions

Debridement

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Intervention Hierarchy (Ancestors)

Surgical Procedures, Operative

Central Study Contacts

Adrian Orr, VP Clinical Development

CONTACT

617-575-2203; cartistem2023-01@medi-post.com

Luis Toro, Medical Monitor for IQVIA, MD

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 9, 2026
• First Posted: January 14, 2026
• Study Start: March 11, 2026
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): June 30, 2029
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (16)