Study of the AeriSeal System Treatment in Patients With Advanced Non-Upper Lobe Predominant Heterogeneous Emphysema

The purpose of this study is to prospectively evaluate the safety and efficacy of the AeriSeal System in patients with advanced Non-Upper Lobe Predominant Heterogeneous Emphysema.

Study Overview

• Status: Withdrawn
• Conditions: Emphysema; Chronic Obstructive Pulmonary Disease (COPD)
• Intervention / Treatment: Device: AeriSeal Emphysematous Lung Sealant Syst

Detailed Description

This is a prospective single arm study to evaluate the safety and efficacy of the AeriSeal System in patients with advanced Non-Upper Lobe Predominant Heterogeneous Emphysema.

• Study Type: Interventional
• Phase: Phase 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Willing and able to provide informed consent and to participate in the study
• Age > or = 40 years at the time of the screening
• Advanced lower or lower and upper lobe predominant heterogeneous emphysema by CT scan
• Minimum of 2 subsegments appropriate for treatment
• MRCD questionnaire score of 2 or greater at screening
• Failure of medical therapy to provide relief of symptoms

• Spirometry 15 minutes after administration of bronchodilator (BOTH):

  • FEV1 < 50 % predicted.
  • FEV1/FVC ratio <70 %

• Lung volumes by plethysmography (BOTH):

  • Total Lung Capacity (TLC) > 100 % predicted
  • Residual Volume (RV) > 150 % predicted
• Diffusing Capacity of Carbon Monoxide(DLco) > = 20 and < = 60 percent predicted
• Oxygen saturation (SpO2) > 90 % on < or = 4 L/min supplemental O2, at rest
• Six-Minute Walk Test distance > or = 150 m
• Abstinence from smoking for at least 16 weeks prior to screening

Exclusion Criteria:

• Prior lung volume reduction surgery, prior lobectomy or pneumonectomy, or prior lung transplantation
• Requirement for ventilator support (invasive or non-invasive)
• Three (3) or more COPD exacerbations requiring hospitalization within 1 year of Screening visit or a COPD exacerbation requiring hospitalization within 8 weeks of Screening visit

• Pulmonary hypertension, defined as:

  • Echocardiogram with estimated peak systolic pressure > 45 mmHg in the presence of tricuspid valve regurgitation stated in the echocardiogram report
  • If the echocardiogram shows peak systolic pressure > 45 mmHg, right heart catheterization is required to rule out pulmonary hypertension, defined as peak systolic pressure > 45 mmHg or mean pressure > 35 mmHg
• Clinically significant asthma (reversible airway obstruction) or bronchiectasis

• CT scan: Presence of the following radiologic abnormalities:

  • Pulmonary nodule on CT scan greater that 1.0 cm in diameter (Does not apply if present for 2 years or more without increase in size or if proven benign by biopsy/PET)
  • Radiologic picture consistent with active pulmonary infection, e.g., unexplained parenchymal infiltrate
  • Significant interstitial lung disease
  • Significant pleural disease
  • Giant bullous disease (a predominant bulla > 10 cm in all dimensions >1 / 3 of the hemithorax)
• Use of systemic steroids > 20 mg/day or equivalent, immunosuppressive agents, heparins, oral anticoagulants (e.g., warfarin, dicumarol; note: antiplatelet drugs including aspirin and clopidogrel are permitted)
• Allergy or sensitivity to medications required to safely undergo AeriSeal System treatment
• Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to the screening visit
• Body mass index < 15 kg/m2 or > 35 kg/m2
• Female patient pregnant or breast-feeding or planning to be pregnant in the next year

• Significant comorbidity that carries prohibitive risks or is associated with less than 2-year expected survival, including any of the following:

  • HIV/AIDS
  • Active malignancy
  • Stroke or Transient Ischemia Attack (TIA) within 12 months of screening
  • Myocardial infarction within 12 months of screening
  • Congestive heart failure within 12 months of screening defined at clinical evidence of right or left hear failure or left ventricular ejection fraction < 45 % on echocardiogram
• Any condition that the Investigator believes would interfere with the intent of the study or would make participation not in the best interest of the patient such as alcoholism, high risk for drug abuse or noncompliance in returning for follow-up visits

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AeriSeal Emphysematous Lung Sealant Syst; This is a prospective, open label, single-arm, multicenter, investigational study. Patients will receive either unilateral or bilateral AeriSeal System therapy as appropriate utilizing 20 mL/subsegment dosing at 2 to 4 subsegments.	Device: AeriSeal Emphysematous Lung Sealant Syst; This is a prospective, open label, single-arm, multicenter, investigational study. Patients will receive either unilateral or bilateral AeriSeal System therapy as appropriate utilizing 20 mL/subsegment dosing at 2 to 4 subsegments.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Volume change by CT	Group mean normalized change from baseline in the volume of the treated lung lobes measured by quantitative CT at 24 weeks post-treatment (defined as the sum of volume change in all treated lobes/number of treatments).	28 - 30 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Fraction of patients showing clinically significant improvements from baseline in one or more of the following outcomes at 24 weeks post treatment: Forced Expiratory Volume in 1 second (FEV1), Forced Vital Capacity (FVC), Medical Research Council Dyspnea	Group mean change in baseline to week 24 in:Forced Expiratory Volume in 1 second (FEV1),Forced Vital Capacity (FVC),Medical Research Council Dyspnea (MRCD), 6 Six-Minute Walk Test (6MWT), Saint George Respiratory Questionnaire (SGRQ)	28 - 30 weeks

Collaborators and Investigators

• Sponsor: Aeris Therapeutics
• Investigators: Principal Investigator: Felix Herth, Prof. Dr med, Thoraxklinik am Uniklinikum Heidelberg; Principal Investigator: Wolfgang Gesierich, Dr med, Asklepios Fachkliniken Muenchen - Gauting; Principal Investigator: Manfred Wagner, Dr med, Klinikum Nuernberg Nord; Principal Investigator: Mordechai Kramer, Prof, Rabin Medical Center; Principal Investigator: Martin Hetzel, Dr med, Krankenhaus vom Roten Kreuz - Stuttgart

Study record dates

Study Major Dates

• Study Start: September 1, 2013
• Primary Completion (Anticipated): November 1, 2013
• Study Completion (Actual): November 1, 2013

Study Registration Dates

• First Submitted: July 18, 2013
• First Submitted That Met QC Criteria: July 24, 2013
• First Posted (Estimate): July 26, 2013

Study Record Updates

• Last Update Posted (Estimate): November 14, 2013
• Last Update Submitted That Met QC Criteria: November 13, 2013
• Last Verified: November 1, 2013

More Information

Terms related to this study

• Keywords: Advanced Emphysema; Heterogeneous Lower Lobe Predominant Emphysema; Heterogeneous Upper and Lower Lobe Predominant
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Lung Diseases, Obstructive; Pulmonary Disease, Chronic Obstructive; Pulmonary Emphysema; Emphysema
• Other Study ID Numbers: 03-C13-001PLV