Clinical trial NCT01520740: Collateral Ventilation Effects on Response to AeriSeal System Treatment in Upper Lobe Predominant Emphysema
Official title: Collateral Ventilation Effects on Response to AeriSeal System Treatment in Upper Lobe Predominant Emphysema
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Pulmonary Emphysema
Keywords: Gold Stage III
Gold Stage IV
Chronic Obstructive Pulmonary Disease
COPD
Lung Volume Reduction Surgery
LVRS
Bronchoscopic Lung Volume Reduction
BLVR
Upper Lobe Predominant
ULP
Heterogeneous
Homogeneous
Lead sponsor: Aeris Therapeutics
Sponsor class: INDUSTRY
First posted: 2012-01-30
Enrollment: 100
Sex: ALL
Minimum age (years): 40
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will evaluate the effects of baseline collateral ventilation status on outcomes following treatment with AeriSeal System in patients with advanced upper-lobe predominant heterogenous 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 upper lobe predominant emphysema by CT scan
* Two (2) subsegments appropriate for treatment based upon CT scan in 2 different upper lobe segments in each lung (total 4 available subsegments)
* 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):

  * TLC \> 100% predicted
  * RV \> 150% predicted
* DLco \> or = 20 and \< or = 60% 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, prior lung transplantation, prior airway stent placement, prior pleurodesis, or prior endobronchial lung volume reduction therapy of any type
* 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
* α-1 antitrypsin serum level of \<80 mg/dl (immunodiffusion) or \<11µmol/L (nephelometry) at Screening visit in the absence of enzyme replacement therapy. Patients with documented α-1 antitrypsin deficiency requiring replacement therapy are excluded from the study participation.
* 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 diameter)
* 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) and investigational medications within 4 weeks of screening
* 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 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: Change from baseline in group mean post bronchodilator Forced Exhalation Volume in one second (FEV1)
timeFrame: 24 Weeks
Secondary outcomes:
measure: Change from Baseline in group mean 6 Minute Walk Test (6MWT) distance
timeFrame: 24 Weeks
measure: Change from baseline in group mean health related quality of life
timeFrame: 24 Weeks
Other outcomes:
Locations:
city: Bad Berka
country: Germany
facility: Zentralklinik Bad Berka GmbH
city: Berlin
country: Germany
facility: Charite Campus Virchow-Klinikum
city: Coburg
country: Germany
facility: Klinikum Coburg
city: Donaustauf
country: Germany
facility: Klinikum Donaustauf
city: Gauting
country: Germany
facility: Asklepios Fachkliniken Muenchen - Gauting
city: Halle
country: Germany
facility: Universitatsklinikum Halle
city: Hamburg
country: Germany
facility: Asklepios Klinik Hamburg-Harburg
city: Heidelberg
country: Germany
facility: Thoraxklinik am Uniklinikum Heidelberg
city: Luebeck
country: Germany
facility: Sana Kliniken Luebeck
city: Munchen
country: Germany
facility: Medizinische Klinik und Poliklinik Grosshadern
city: Jerusalem
country: Israel
facility: Hadassah - Hebrew University Medical Center
city: Petach-Tikva
country: Israel
facility: Carmel Medical Center
city: Tel Aviv
country: Israel
facility: Chaim Sheba Medical Center
Study officials:
affiliation: Aeris Therapeutics
name: Janine McDermott, MS CCRP
role: STUDY_DIRECTOR
References: