Clinical trial NCT03588975: A Study of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee
Official title: A Prospective, Open-label, Randomized, Concurrent Active-controlled, Longitudinal, Multicenter, Phase 3 Clinical Study of the Safety and Efficacy of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Chondral Defect
Osteochondritis Dissecans (OCD)
Articular Cartilage Defect
Articular Cartilage Disorder of Knee
Keywords: OCD
cartilage
cartilage defect
Lead sponsor: Vericel Corporation
Sponsor class: INDUSTRY
First posted: 2018-07-17
Enrollment: 45
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed description: This is a 2-year prospective, multicenter, randomized, open-label, parallel group clinical trial; a total of 45 patients, ages 10 to 17 years, will be randomized to receive a 1-time treatment with MACI or microfracture (2:1, 30 MACI:15 microfracture).

After meeting screening criteria at the initial visit, all patients will have a screening arthroscopy to further assess study eligibility. During the screening arthroscopy, patients will be further evaluated against entry criteria. Cartilage lesion size will be measured prior to any cartilage repair procedure and randomization. All patients who meet the eligibility criteria and are considered suitable for treatment in the study will have a cartilage biopsy taken prior to randomization to study treatment. Eligible patients will be randomized during the screening arthroscopy procedure to receive either MACI or microfracture treatment. Patients randomized to microfracture will undergo the procedure during the screening arthroscopy.

All biopsied tissue will be sent to the Vericel manufacturing facility in Cambridge, Massachusetts, where the sample will be processed to isolate the autologous chondrocytes. Cells from patients randomized to the MACI group will be used in the preparation of the MACI implant; cells from patients randomized to the microfracture group will be cryopreserved.

Patients randomized to treatment with MACI will return within 12 weeks of the screening arthroscopy to undergo the chondrocyte implantation procedure via arthrotomy. Patients are to follow a recommended postoperative rehabilitation program and compliance with the rehabilitation schedule will be monitored.

Patients will be followed post-study treatment for 2 years (104 Weeks).

Eligibility criteria: Inclusion Criteria:

* 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).
* At least 1 defect size ≥1.5 cm2 on the 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.
* 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.

Exclusion Criteria:

* Any surgery on the target knee joint within 6 months prior to Screening (not including diagnostic arthroscopy)
* ICRS Grade III or IV chondral or unsalvageable osteochondral defects located on the patella or tibia or any lesion that is bipolar to the index lesion
* 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
* Known history of hypersensitivity to gentamicin, other aminoglycosides, or products of porcine or bovine origin
* Females who are pregnant or lactating

Primary outcomes:
measure: Proportion of Patients Who Achieve at Least a 10-point Improvement From Baseline in KOOS-Child Pain and Function (Sports and Recreational Activities) Scores
timeFrame: Baseline and Week 104
Secondary outcomes:
measure: Change from Baseline in KOOS-Child subscores
timeFrame: Baseline and Week 104
measure: Change from Baseline in KOOS-Child subscores
timeFrame: Baseline and Week 52
Other outcomes:
measure: Treatment Emergent Adverse Events
timeFrame: Week 104
Locations:
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Sacramento
country: United States
facility: Shriner's Hospital for Children Northern California
state: California
city: Sacramento
country: United States
facility: University of California Davis Health
state: California
city: New Orleans
country: United States
facility: Ochsner Sports Medicine Institute
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins Outpatient Center
state: Maryland
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Jameson Crane Sports Medicine Institute
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: Penn Sports Medicine Center
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: Vericel Corporation
name: Jonathan Hopper
role: STUDY_DIRECTOR
References: