A Study of MACI in Patients Aged 17 to 65 Years With Symptomatic Chondral or Osteochondral Defects of the Ankle

NCT06915233 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: 55-2410-01
• Study Type: interventional
• Target: 309
• Locations: 1 country
• Sites: 4

Brief Summary

The objective of this study is to demonstrate the superiority of MACI (autologous cultured chondrocytes on porcine collagen membrane) versus Bone Marrow Stimulation in the treatment of patients aged 17 to 65 years with symptomatic articular chondral or osteochondral defects of the talus.

Conditions

Chondral Defect; Articular Cartilage Defect

Keywords

Bone Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Osteochondritis; Cartilage Diseases

Outcome Measures

Primary Outcomes (1)

• Change from Baseline to Week 104 in the FAOS Pain and Function (SRA) subscale scores

  The FAOS is a 42-item, patient-reported outcome measure that assesses foot and ankle health in the past week. There are five FAOS subscales: Pain, Symptoms, Function (Daily Living), Function (SRA), and Foot and Ankle Related Quality of Life. The FAOS has been validated for a variety of foot and ankle conditions, including OLT. Responses to each question on the FAOS are recorded on a 5-point Likert scale. A normalized score out of 100 is calculated for each subscale, with 0 indicating extreme symptoms and 100 no symptoms. Selection of this composite primary endpoint is consistent with FDA guidance recommending selection of primary endpoints that evaluate changes in Pain and Function (SRA) subscale scores as they represent clinically meaningful outcomes for patients who receive products intended to repair or replace damaged cartilage in the knee.

  Baseline to Week 104

Secondary Outcomes (2)

• Percentage of subjects who respond to study treatment after 2 years, i.e., subjects who have ≥ 10-point improvement on both the FAOS Pain and Function (SRA) subscale scores from Baseline to Week 104.

  Baseline to Week 104

• Change from Baseline to Week 104 in the remaining FAOS subscales scores [Symptoms, Function (Daily Living) and Quality of Life]

  Baseline to Week 104

Study Arms (2)

MACI

EXPERIMENTAL

autologous cultured chondrocytes on porcine collagen membrane

Biological: autologous cultured chondrocytes on porcine collagen membrane

Bone Marrow Stimulation

ACTIVE COMPARATOR

Surgical procedure

Procedure: Bone Marrow Stimulation

Interventions

autologous cultured chondrocytes on porcine collagen membrane BIOLOGICAL

An autologous cellularized scaffold product being studied for the repair of symptomatic, single or multiple full-thickness cartilage defects of the talus with or without bone involvement in adults.

MACI

Bone Marrow Stimulation PROCEDURE

A surgical awl or microdrill will be used to make multiple holes in the exposed base of the lesion. The microfracture procedure should result in holes that are approximately 3 to 4 mm apart. When fat droplets can be seen coming from the marrow cavity, the appropriate depth (approximately 2 to 4 mm) has been reached. The released marrow elements (including mesenchymal stem cells, growth factors, and other healing proteins) form a surgically induced clot that provides an enriched environment for new tissue formation.

Bone Marrow Stimulation

Eligibility Criteria

• Age: 17 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 17 to 65 at the time of planned randomization visit (Visit 2).
• One or more symptomatic chondral or osteochondral lesion/s as defined by FAOS Pain score ≤ 50 and FAOS Function (SRA) score ≤ 50.
• International Cartilage Repair Society (ICRS) Score Grade 3 or 4 chondral or osteochondral lesion/s located on the talus with or without cysts, including shoulder lesions (lesions on the talar neck), and amenable to treatment with the surgical procedure specified at randomization.
• At least 1 lesion ≥ 1.2 cm².
• Written informed consent and assent (as applicable) per Institutional Review Board (IRB) requirements.
• Subject will refrain from using Non-steroidal Anti-inflammatory Drugs (NSAIDS) for 12 weeks post-study treatment. (Post-surgical use of low-dose aspirin for clot prevention is acceptable).
• Subject will restrict pain medication to over-the-counter analgesics (NSAIDs or acetaminophen/paracetamol) after 12 weeks post-study treatment.
• Subject must have Hematocrit ≥30.0%; White Blood Cell count ≤14,000 cells/μL; Platelet Count ≥50,000 platelets/μL; Creatinine ≤2.0 mg/dL; and International Normalized Ratio (INR) ≤1.6.

You may not qualify if:

• Lesions with an underlying bony defect depth of \> 5 mm.
• Any surgery on the target joint within 24 weeks prior to Visit 1 (not including diagnostic ankle arthroscopy).
• Previous investigational drug, biologic or device use within 12 weeks prior to Visit.
• Avascular necrosis of the target ankle.
• Symptomatic musculoskeletal conditions in the lower limbs that could impede measurement of efficacy for the target ankle joint.
• Subjects with "kissing lesions" (bipolar lesions, involving both the tibia and talus), or with bilateral lesions in both ankles.
• Subjects with lesions that require an osteotomy procedure to allow for MACI implantation as determined at the time of ankle arthroscopy (Visit 2).
• Concomitant systemic inflammatory diseases or other conditions that affect the joints (e.g., rheumatoid arthritis, metabolic bone disease, psoriasis, symptomatic chondrocalcinosis).
• History of advanced or severe osteoarthritis of the ankle as determined by prior surgical history confirming no joint space (i.e., "bone on bone") or radiographic evidence of Modified Kellgren-Lawrence Grade 4 arthritis (i.e., osteophytes on lateral and medial malleoli and at the tibiotalar joint margins, narrowing of the joint space width \> 50%, and tibiotalar sclerosis), Van Dijk Grade III (i.e., total or subtotal destruction of the joint space) or the equivalent.
• Known history of septic arthritis in the target ankle joint within 1 year prior to Visit 1.
• Current malignancy or treatment for malignancy within the past 5 years prior to Visit 1, excluding non-melanoma skin cancer.
• Known history of hypersensitivity to gentamicin, other aminoglycosides, or products of porcine or bovine origin.
• Females who are pregnant or lactating.
• Any condition that has potential for negatively impacting intra- or postoperative course.
• Conditions that limit compliance with rehabilitation program.

Sponsors & Collaborators

• Vericel Corporation: lead

Study Sites (4)

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

RECRUITING

NextStage Clinical Research Wichita - Kansas Joint and Spine specialists

Wichita, Kansas, 67226, United States

RECRUITING

NextStage Clinical Research Houston - All American Orthopedic and Sports Medicine

Houston, Texas, 77058, United States

RECRUITING

NextStage Clinical Research San Antonio - San Antonino Podiatry Associates

San Antonio, Texas, 78251, United States

RECRUITING

MeSH Terms

Conditions

Bone Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Osteochondritis; Cartilage Diseases

Condition Hierarchy (Ancestors)

Skin and Connective Tissue Diseases

Study Officials

• Jonathan Hopper BSc (Hons), MB ChB, FRCSEd, DIMC RCSEd., MBA, BSc (Hons), MB

  Vericel Corporation

  STUDY CHAIR

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 1, 2025
• First Posted: April 8, 2025
• Study Start: March 1, 2026
• Primary Completion (Estimated): July 1, 2030
• Study Completion (Estimated): July 1, 2030
• Last Updated: March 6, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)