Treatment of Pectus Excavatum Deformity Using Macrolane Filler

Study Overview
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Brief Summary
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This is a prospective, open, non-comparative and baseline-controlled study to evaluate efficacy and safety of Macrolane VRF20 treatment in 40 subjects with pectus excavatum deformity. Each subject participating in the study will be treated with approximately 50-150 ml of Macrolane VRF20. The amount of study product used will be individually determined in order to achieve an optimal correction of the deformity in each subject.

Official Title
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An Open Non Comparative Multicenter Prospective Study to Evaluate Efficacy and Safety of Macrolane VRF20 in Treatment of Pectus Excavatum

Conditions
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Pectus Excavatum Deformity

Intervention / Treatment
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* Device: Macrolane VRF20


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Give verbal and written informed consent to participate in the study. Be a healthy male of 18 years or more. Have a pectus excavatum deformity without functional problems; score 4 in items 10, 11 and 12 of the PEEQ, indicating there is no functional impairment due to the pectus excavatum deformation. Present normal cardiac function as assessed by ECG and echocardiogram. Present normal pulmonary function as assessed by pulmonary function test. Present a chest X-ray taken within 12 months prior to the baseline visit, without clinically significant defects to heart, lungs, skeleton, ribs, sternum or spinal cord except for the pectus excavatum defect, in the opinion of the Investigator. Have the ability to understand and comply with the requirements of the study. Exclusion Criteria: Score 1, 2 or 3 in either of items 10, 11 and 12 of the PEEQ, indicative of functional problems due to the pectus excavatum deformity. Previous treatment for the same indication. Known or suspected hypersensitivity to hyaluronic acid based products. BMI < 20. A history of severe allergies manifested by a history of anaphylaxis, or a history or presence of multiple severe allergies (as judged by the Investigator). Known allergy to any anesthesia planned during the study. Presence of autoimmune disease or other chronic disease that in the opinion of the Investigator may interfere with the outcome of the study. Subjects with bleeding disorders or subjects who are taking thrombolytics or anticoagulants, or have taken inhibitors of platelet aggregation, including non-steroidal anti-inflammatory agents and acetylsalicylic acid, two weeks before treatment. Subjects on immunomodulatory therapy (suppressive or stimulatory). Subjects with contraindications for MRI, such as presence of pacemaker, clips or splinter, or tendency for claustrophobia. Any condition which in the opinion of the Investigator makes the subject unsuitable for inclusion (e.g., subjects not likely to participate for the duration of the study). Use of any investigational drugs or devices within 30 days prior to baseline. Subjects who are study site staff for this study, or close relatives of the study site staff, as well as subjects who are employed by the Sponsor company, or close relatives of employees at the Sponsor company.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Macrolane VRF20<br>All subjects will receive treatment with Macrolane VRF20 to correct pectus excvatum deformity. | Device: Macrolane VRF20<br>* Injection treatment with Macrolane VRF20<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| PEEQ | To evaluate improvement from baseline in general self-esteem and emotion at 1, 3, 6, 12 and 24 months after treatment with Macrolane VRF20, using a modified version of the pectus excavatum evaluation questionnaire (PEEQ) before and after treatment. | Jul 2016 | 
| Subject satisfaction | Evaluate subject satisfaction at 1, 3, 6, 12 and 24 months after treatment. | Jul 2016 | 
| Duration | Estimate duration of Macrolane VRF20 calculated using MRI images at 12 months (and 24 months for a subset of patients). | Jul 2016 | 
| Placement | Assess placement using MRI at 1 and 12 months post treatment. | Jul 2016 | 
| Adverse event | To study safety throughout the study period, i.e. up to 24 months after treatment, based on evaluation of reported Adverse Events. | Jul 2016 | 
| Downtime | Evaluate recovery time after treatment using 14-days subject diary. | Jul 2016 | 
| Downtime 2 | Evaluate days hospitalized or on sick leave after treatment. | Jul 2016 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pectus excavatum, funnel chest, sunken chest