A Study of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee

NCT03588975 | Recruiting Phase 3 FDA Drug

• 1 other identifier: 55-1702-1
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 12

Brief Summary

The objective of this study is to compare the efficacy and safety of MACI® vs arthroscopic microfracture in the treatment of participants aged 10 to 17 years with symptomatic articular chondral or osteochondral defects of the knee.

Conditions

Chondral Defect; Osteochondritis Dissecans (OCD); Articular Cartilage Defect; Articular Cartilage Disorder of Knee

Keywords

OCD; cartilage; cartilage defect

Outcome Measures

Primary Outcomes (1)

• Proportion of Participants Who Achieve ≥ 10-point Improvement From Baseline in KOOS-Child Pain and Function (Sports and Recreational Activities) Subscale Scores

  A responder is defined as a participant with ≥ 10-point change (improvement) in both the KOOS-Child Pain and Function (Sports and Recreational activities) scores from Baseline scores. The Knee and Osteoarthritis Outcome Score for Children (KOOS-Child) is a validated knee-specific instrument developed to assess the participant's opinion of their knee and associated problems. KOOS consists of 5 subscales: Pain, Function in sports and recreational activities, other Symptoms, Function in activities of daily living (ADL), and knee related Quality of life (QOL).

  Baseline and Week 104

Secondary Outcomes (2)

• Change from Baseline in KOOS-Child subscores

  Baseline and Week 104

• Change from Baseline in KOOS-Child subscores

  Baseline and Week 52

Other Outcomes (1)

• Treatment Emergent Adverse Events

  Week 104

Study Arms (2)

MACI

EXPERIMENTAL

autologous cultured chondrocytes on porcine collagen membrane

Biological: MACI

microfracture

OTHER

surgical procedure

Procedure: microfracture

Interventions

MACI BIOLOGICAL

autologous cultured chondrocytes on porcine collagen membrane

MACI

microfracture PROCEDURE

Arthroscopic microfracture treatment

microfracture

Eligibility Criteria

• Age: 10 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Symptomatic cartilage or osteochondral defects
• One or more International Cartilage Repair Society (ICRS) Grade III or IV chondral or unsalvageable osteochondral defects located on the femoral condyles and/or trochlea amenable to treatment with the surgical procedure determined at randomization (MACI or microfracture).
• Concurrent ICRS Grade I and II defects are acceptable on the tibia if they remain untreated (or are treated with debridement only) at the time of study treatment.

You may not qualify if:

• At least 1 defect size ≥1.5 cm2 on the patella, femoral condyles and/or the trochlea; defects include OCD lesions with a bone lesion depth of ≤6 mm and does not require a bone graft.
• Salvageable OCD lesions must have failed prior conservative and a reparative treatment, and the participant still experiences pain associated with the osteochondral lesion. Participants with defects as a result of trauma or unknown etiology are eligible regardless of prior treatments.
• Stable target knee (i.e., anterior and posterior cruciate ligaments should be free of laxity as well as stable and intact). Ligament repair or reconstruction procedures are allowed prior to screening arthroscopy.
• Intact meniscus or partial meniscus (at least 50% of functional meniscus remaining) in the target knee. Meniscal repair or resection might be performed either staged or concurrent with the cartilage repair procedure provided that the surgeon was able to confirm that at least 50% of functional meniscus would remain after the corrective meniscal treatment.
• Written informed consent
• Any surgery on the target knee joint within 6 months prior to Screening (not including diagnostic arthroscopy)
• Previous investigational drug or device use within 3 months prior to Screening
• Avascular necrosis of the target knee (other than potential OCD lesion to be treated)
• Participants with uninvolved open growth plates where the surgery necessitates crossing the growth plate, or where the depth of the lesion crosses the growth plate
• ICRS Grade III or IV chondral or unsalvageable osteochondral defects located on the tibia or any lesion that is bipolar to the index lesion
• Symptomatic musculoskeletal conditions in the lower limbs that could impede measurement of efficacy for the target knee joint
• Concomitant inflammatory disease or other conditions that affects the joints (eg, rheumatoid arthritis, metabolic bone disease, psoriasis, symptomatic chondrocalcinosis)
• Known history of septic arthritis in the index knee joint
• Current malignancy or treatment for malignancy within the past 5 years
• Known history of hypersensitivity to gentamicin, other aminoglycosides, or products of porcine or bovine origin

Sponsors & Collaborators

• Vericel Corporation: lead

Study Sites (12)

Stanford University

Palo Alto, California, 94304, United States

RECRUITING

Shriner's Hospital for Children Northern California

Sacramento, California, 95817, United States

RECRUITING

University of California Davis Health

Sacramento, California, 95817, United States

RECRUITING

Ochsner Sports Medicine Institute

New Orleans, Louisiana, 70121, United States

RECRUITING

Johns Hopkins Outpatient Center

Baltimore, Maryland, 21224, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48106, United States

RECRUITING

Akron Children's Hospital

Akron, Ohio, 44308, United States

RECRUITING

The Ohio State University Jameson Crane Sports Medicine Institute

Columbus, Ohio, 43202, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Penn Sports Medicine Center

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77094, United States

WITHDRAWN

Medical College of Wisconsin

Milwaukee, Wisconsin, 53201, United States

RECRUITING

Related Links

• Vericel Clinical Trial for Cartilage Repair

MeSH Terms

Conditions

Osteochondritis Dissecans

Interventions

Arthroplasty, Subchondral

Condition Hierarchy (Ancestors)

Osteochondritis; Bone Diseases; Musculoskeletal Diseases

Intervention Hierarchy (Ancestors)

Arthroplasty; Orthopedic Procedures; Surgical Procedures, Operative; Plastic Surgery Procedures

Study Officials

• Jonathan Hopper, BSc (Hons), MB ChB, FRCSEd

  Vericel Corporation

  STUDY DIRECTOR

Central Study Contacts

Vericel Clinical Affairs

CONTACT

978-347-2876; clinicalhotline@vcel.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2018
• First Posted: July 17, 2018
• Study Start: October 24, 2018
• Primary Completion (Estimated): February 1, 2030
• Study Completion (Estimated): February 1, 2030
• Last Updated: June 24, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (12)