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Extension Study for Participants of MACI00206 Study of MACI® for the Treatment of Symptomatic Articular Cartilage Defects of the Knee (Extension)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Vericel Corporation
ClinicalTrials.gov Identifier:
NCT01251588
First received: November 30, 2010
Last updated: May 8, 2017
Last verified: May 2017
  Purpose
To examine the 5-year efficacy and safety of MACI implant, compared with arthroscopic microfracture, in participants who received study treatment in the SUMMIT study for treatment of symptomatic articular cartilage defects of the knee.

Condition Intervention
Articular Cartilage Defects Biological: autologous cultured chondrocytes on porcine collagen membrane Procedure: Microfracture

Study Type: Observational
Study Design: Observational Model: Other
Time Perspective: Prospective
Official Title: An Extension Protocol for Participants of Genzyme-Sponsored Prospective, Randomized, Open-Label, Parallel-Group, Multicenter Study of MACI® for the Treatment of Symptomatic Articular Cartilage Defects of the Femoral Condyle Including the Trochlea

Further study details as provided by Vericel Corporation:

Primary Outcome Measures:
  • Change From MACI00206 Baseline for the Participant's Knee Injury and Osteoarthritis Outcome (KOOS) Pain and Function (Sports and Recreational Activities) Scores. [ Time Frame: MACI00206 Baseline to Week 156 ]
    The KOOS is a validated knee-specific instrument developed to assess the patients' 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). A 5-point Likert scale was used to record the response to each item ranging from 0 (no problems) to 4 (extreme problems). Within each subscale, items were added up and normalized to a value between 0 (extreme problems) and 100 (no problems). Subscales are not combined to calculate a total score.


Secondary Outcome Measures:
  • Change From MACI00206 Baseline for the Participant's KOOS Pain and Function (Sports and Recreational Activities) Scores [ Time Frame: MACI00206 Baseline and Week 260 ]
    The KOOS is a validated knee-specific instrument developed to assess the patients' 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). A 5-point Likert scale was used to record the response to each item ranging from 0 (no problems) to 4 (extreme problems). Within each subscale, items were added up and normalized to a value between 0 (extreme problems) and 100 (no problems). Subscales are not combined to calculate a total score.

  • Magnetic Resonance Imaging (MRI) Assessments of Degree of Defect Fill [ Time Frame: Week 260 ]
    MRI was assessed by the independent blinded evaluators by means of consensus. The number of participants with a degree of defect fill of >50% is reported.

  • Proportion of Patients Who Achieve at Least a 10-point Improvement From MACI00206 Baseline in KOOS Pain and Function (Sports and Recreational Activities) Scores [ Time Frame: Up to week 260 ]
    A responder is defined as a participant with at least a 10-point improvement in both the KOOS Pain and Function (Sports and Recreational activities) scores from MACI00206 Baseline scores.

  • The Proportion of Patients in Each Treatment Group Assessed as Treatment Failures [ Time Frame: Years 2 through 5 post treatment (MACI or microfracture) ]

    Patients were considered as a treatment failure if all of the following 5 criteria were met:

    1. Patient's global assessment of their knee joint compared to Baseline was the same or worse
    2. Physician's global assessment of the patient's knee joint compared to Baseline was the same, worse, or significantly worse.
    3. Percent improvement from Baseline in KOOS Pain score was less than 10%.
    4. Physician diagnostic evaluation of failure excluded etiologies (eg, meniscal tear) other than failed treatment of the index lesion.
    5. The physician decided that surgical re-treatment of the index lesion(s) was required that involved either extensive debridement for lesion expansion, violation of the subchondral bone, or ACI.

  • Average Time to Treatment Failure [ Time Frame: Up to 260 weeks ]

    ANALYSIS NOT DONE: The planned analyses concerning time to treatment failure were not conducted due to the small number of per protocol treatment failure cases. The number of per protocol treatment failures in each treatment group are reported here.

    Patients were considered as a treatment failure if all of the following 5 criteria were met:

    1. Patient's global assessment of their knee joint compared to Baseline was the same or worse
    2. Physician's global assessment of the patient's knee joint compared to Baseline was the same, worse, or significantly worse.
    3. Percent improvement from Baseline in KOOS Pain score was less than 10%.
    4. Physician diagnostic evaluation of failure excluded etiologies (eg, meniscal tear) other than failed treatment of the index lesion.
    5. The physician decided that surgical re-treatment of the index lesion(s) was required that involved either extensive debridement for lesion expansion, violation of the subchondral bone, or ACI.

  • Change From MACI00206 Baseline in the Remaining 3 Subscales (Activities of Daily Living, Quality of Life, and Other Symptoms) of KOOS [ Time Frame: MACI00206 Baseline and Week 260 ]
    The KOOS is a validated knee-specific instrument developed to assess the patients' 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). A 5-point Likert scale was used to record the response to each item ranging from 0 (no problems) to 4 (extreme problems). Within each subscale, items were added up and normalized to a value between 0 (extreme problems) and 100 (no problems). Subscales are not combined to calculate a total score.

  • Change From MACI00206 Baseline in the Patient's Evaluation of Overall Knee Condition Using the International Knee Documentation Committee (IKDC) Subjective Knee Evaluation Form [ Time Frame: MACI00206 Baseline and Week 260 ]

    The IKDC Subjective Knee Evaluation Form is a validated knee-specific measure of symptoms, function, and sports activity that is appropriate for patients with a wide variety of knee problems. The form consists of 18 items covering the domains of symptoms, functioning during activities of daily living and sports, and current function of the knee.

    The IKDC Subjective Knee Evaluation Form is scored by summing the scores for the individual items and then transforming the score to a scale that ranges from 0 to 100. The transformed score is interpreted as a measure of function such that higher scores represent higher levels of function and lower levels of symptoms. A score of 100 is interpreted to mean no limitation with activities of daily living or sports activities and the absence of symptoms.


  • Change From MACI00206 Baseline in the Patient's Evaluation of Overall Knee Condition Using the Modified Cincinnati Knee Rating System [ Time Frame: MACI00206 Baseline and Week 260 ]
    The Modified Cincinnati Knee Rating System is a self-assessment of the intensity of sports participation, functional limitations, and the ability to participate in different types of sports. The Modified Cincinnati Knee Rating System overall knee condition score ranges from 1 (poor) to 10 (excellent).

  • Change From MACI00206 Baseline in the 12-Item Short-Form Health Survey (SF-12) Physical and Mental Component Scores [ Time Frame: MACI00206 Baseline and Week 260 ]

    The SF-12 is a subset of the 36-Item Short-Form Health Survey (SF-36) and includes 8 subscales (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional, and mental health) that are used to calculate the physical (PCS) and mental (MCS) summary component scores.

    MCS and PCS are summarized as Z-scores using standard SF-12 scoring and a US population means. The Z-score indicates how many standard deviations a score is from the population mean. Higher values reflect better health. Changes from Baseline are reported.


  • Change From MACI00206 Baseline in the European Quality of Life 5 Dimensions (EQ-5D) Visual Analog Scale (VAS) Score [ Time Frame: MACI00206 Baseline and Week 260 ]
    The EQ-5D is a standardized instrument for use as a measure of health outcome (see the EuroQOL Website for details: www.euroqol.org). Applicable to a wide range of health conditions and treatments, it provides a simple descriptive profile and a single index value for health status. The EQ Visual Analogue Scale (VAS) was used to record the respondents' self-rated health status on a vertical graduated (0-100) VAS where 0 is 'the worst health you can imagine' and 100 is 'the best health you can imagine'.

  • Number of Participants Reporting Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Years 2 through 5 post treatment (MACI or microfracture) ]
  • Number of Participants Reporting Serious Adverse Events (SAEs) [ Time Frame: Years 2 through 5 post treatment (MACI or microfracture) ]
  • Number of Participants Having Subsequent Surgical Procedures (SSPs) in Target Knee [ Time Frame: Years 2 through 5 post treatment (MACI or microfracture) ]

Enrollment: 128
Study Start Date: December 2010
Study Completion Date: March 2015
Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
MACI
autologous cultured chondrocytes on porcine collagen membrane implant received in previous MACI00206 study
Biological: autologous cultured chondrocytes on porcine collagen membrane
Implantation received in the previous MACI00206 study
Other Names:
  • MACI
  • matrix applied characterized autologous cultured chondrocytes
Microfracture
Microfracture treatment received in previous MACI00206 study
Procedure: Microfracture
Arthroscopic Microfracture treatment received in the previous MACI00206 study

Detailed Description:

This study was an open-label, multicenter extension to the SUMMIT study designed to examine the 5-year efficacy and safety of MACI compared to arthroscopic microfracture in the treatment of articular cartilage defects of the knee in participants who received study treatment in the SUMMIT study. Follow-up to 2 years post-study treatment was completed as part of the SUMMIT study. Follow-up from 3 to 5 years post-study treatment in SUMMIT was completed in this extension study.

All 144 participants who received study treatment in the SUMMIT study were eligible for enrollment into this extension study. Participants had until the end of the visit window for the last visit of this extension study (ie, Week 260 + 6 weeks) to consent to enter this extension study.

Efficacy and safety assessments were performed at scheduled visits 3, 4, and 5 years following treatment in SUMMIT (ie, at Weeks 156, 208, and 260 postarthrotomy for patients treated with MACI implant or at Weeks 156, 208, and 260 postarthroscopy for patients treated with microfracture).

  Eligibility

Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Randomized and received study treatment (MACI or microfracture) in the SUMMIT study.
Criteria
Randomized and received study treatment (MACI or microfracture) in the SUMMIT study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01251588

Locations
Czechia
Urazova nemocnice v Brne
Brno, Czechia, 662 50
University Hospital Na Bulovce- Department of Orthopaedic Surgery Postgraduate Medical Institute
Praha 8, Czechia, 180 81
France
Polyclinique Saint-Roch
Montpellier Cedex 2, France, 34967
Clinique Maussins-Nollet
Paris, France, 75019
Hôpital d'instruction des Armées Robert Picqué
VILLENAVE d'ORNON cedex, France, 33882
Netherlands
Academisch Ziekenhuis Maastricht
Maastricht, Netherlands, 6202 AZ
St. Elisabeth Ziekenhuis
Tilburg, Netherlands, 5022 GC
University Medical Centre of Utrecht, Department of Orthopaedics
Utrecht, Netherlands, 3508 GA
Norway
St Olavs Hospital
Trondheim, Norway, 7006
Poland
Orthopaedic & Traumatology of Motor Organs, Independent Public Clinic Hospital
Bydgoszcz, Poland, 85-094
Regional Centre of Knee Surgery & Arthroscopy, Regional Hospital of Traumatologic Surgery
Piekary Slaskie, Poland, 41-940
Lekmed Medical Center
Warsow, Poland, 01-480
Sweden
Kungsbacka Närsjukhus
Kungsbacka, Sweden, 434 80
United Kingdom
University of Oxford, Nuffield Dept. of Orthopaedic Surgery
Oxford, United Kingdom, OX3 7LD
Sponsors and Collaborators
Vericel Corporation
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Vericel Corporation
ClinicalTrials.gov Identifier: NCT01251588     History of Changes
Other Study ID Numbers: MACI00809
2009-016970-33 ( EudraCT Number )
Study First Received: November 30, 2010
Results First Received: January 12, 2017
Last Updated: May 8, 2017
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by Vericel Corporation:
Cartilage legions
Symptomatic focal cartilage defects
Microfracture
MACI
Autologous chondrocyte

Additional relevant MeSH terms:
Cartilage Diseases
Musculoskeletal Diseases
Connective Tissue Diseases

ClinicalTrials.gov processed this record on June 28, 2017