Clinical trial NCT01908933: Study of the AeriSeal System Treatment in Patients With Advanced Non-Upper Lobe Predominant Heterogeneous Emphysema
Official title: Study of the AeriSeal System Treatment in Patients With Advanced Non-Upper Lobe Predominant Heterogeneous Emphysema
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE3
Conditions: Emphysema
Chronic Obstructive Pulmonary Disease (COPD)
Keywords: Advanced Emphysema
Heterogeneous Lower Lobe Predominant Emphysema
Heterogeneous Upper and Lower Lobe Predominant
Lead sponsor: Aeris Therapeutics
Sponsor class: INDUSTRY
First posted: 2013-07-26
Enrollment: 0
Sex: ALL
Minimum age (years): 40
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Volume change by CT
timeFrame: 28 - 30 weeks
Secondary outcomes:
measure: 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
timeFrame: 28 - 30 weeks
Other outcomes:
Locations:
Study officials:
affiliation: Thoraxklinik am Uniklinikum Heidelberg
name: Felix Herth, Prof. Dr med
role: PRINCIPAL_INVESTIGATOR
affiliation: Asklepios Fachkliniken Muenchen - Gauting
name: Wolfgang Gesierich, Dr med
role: PRINCIPAL_INVESTIGATOR
affiliation: Klinikum Nuernberg Nord
name: Manfred Wagner, Dr med
role: PRINCIPAL_INVESTIGATOR
affiliation: Rabin Medical Center
name: Mordechai Kramer, Prof
role: PRINCIPAL_INVESTIGATOR
affiliation: Krankenhaus vom Roten Kreuz - Stuttgart
name: Martin Hetzel, Dr med
role: PRINCIPAL_INVESTIGATOR
References: