Clinical trial NCT00515164: US Biologic Lung Volume Reduction (BLVR) Phase 2 Emphysema Study
Official title: Phase 2 Study of the 20 mL Biologic Lung Volume Reduction System (BLVR) in Patients With Advanced Upper Lobe Predominant Emphysema
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Pulmonary Emphysema
Chronic Obstructive Pulmonary Disease
Keywords: lung volume reduction
emphysema
chronic obstructive pulmonary disease
Lead sponsor: Aeris Therapeutics
Sponsor class: INDUSTRY
First posted: 2007-08-13
Enrollment: 22
Sex: ALL
Minimum age (years): 40
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study it to evaluate the efficacy and safety of the 20 mL BLVR System in patients with advanced upper lobe predominant emphysema.

Detailed description: Background:

Patients with emphysema currently have limited treatment choices. Many patients are treated with steroids and inhaled medications, which often provide little or no benefit. In recent years, lung volume reduction surgery has become an accepted therapy for advanced emphysema. Lung volume reduction surgery involves the removal of diseased portions of the lung in order to enable the remaining, healthier portions of the lung to function better. This procedure, although effective for many patients, is complicated and is accompanied by substantial morbidity and mortality risk.

Aeris Therapeutics has developed the Biologic Lung Volume Reduction (BLVR) System which is intended to achieve lung volume reduction without surgery and its attendant risks. Patients are treated using a bronchoscope to direct treatment to the most damaged areas of the lung. The treatment delivers a precisely proportioned proprietary mixture of drugs and biologics which, when combined at the treatment site, form a biodegradable hydrogel. The hydrogel acts to reduce lung volume by permanently collapsing and sealing the diseased areas of the lung. This provides room within the chest to allow the remaining healthier portions of the lung to function better.

Aeris' BLVR development program has been granted Fast Track designation by the U.S. FDA, and is the subject of ongoing clinical trials designed to investigate the safety and efficacy of the BLVR System as a treatment for patients with advanced heterogeneous emphysema. Fast Track designation is reserved for drug and biologic development programs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

Eligibility criteria: Inclusion Criteria:

* clinical diagnosis of advanced upper lobe emphysema
* age \>/= 40 years
* clinically significant dyspnea
* failure of standard medical therapy to relieve symptoms (inhaled beta agonist \& inhaled anticholinergic)
* pulmonary function tests within protocol-specified ranges (post bronchodilator FEV1 \< 45% predicted \& experiencing \< 30% or 300 mL improvement using bronchodilator; total lung capacity \> 110% predicted; residual volume \> 150% predicted)
* 6 Minute Walk Distance \>/= 150 m

Exclusion Criteria:

* alpha-1 protease inhibitor deficiency
* homogeneous emphysema
* tobacco use within 4 months of initial visit
* body mass index \< 15 kg/m2 or\> 35 kg/m2
* clinically significant asthma, chronic bronchitis or bronchiectasis
* allergy or sensitivity to procedural components
* pregnant, lactating or unwilling to use birth control if required
* prior lung volume reduction surgery, lobectomy, pneumonectomy, lung transplant, endotracheal valve placement, airway stent placement or pleurodesis
* comorbid condition that could adversely influence outcomes
* inability to tolerate bronchoscopy under conscious sedation (or anesthesia)
* history of renal infarction or renal failure

Primary outcomes:
measure: Reduction in gas trapping
timeFrame: 12 weeks post treatment
measure: SAEs - Safety of treatment and the procedure
timeFrame: 2 years post treatment
Secondary outcomes:
measure: Improvement in exercise capacity
timeFrame: 12 weeks post treatment
measure: Improvement in vital capacity
timeFrame: 12 weeks post treatment
measure: Improvement in expiratory flow
timeFrame: 12 weeks post treatment
measure: Improvement in inspiratory flow
timeFrame: 12 weeks post treatment
measure: Improvement in dyspnea symptoms (breathlessness)
timeFrame: 12 weeks post treatment
measure: Improvement in respiratory quality of life
timeFrame: 12 weeks post treatment
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Pulmonary Associates
state: Arizona
city: Iowa City
country: United States
facility: University of Iowa Hospitals & Clinics
state: Iowa
city: Topeka
country: United States
facility: Veritas Clinical Specialties, Ltd
state: Kansas
city: Towson
country: United States
facility: St Joseph's Medical Center
state: Maryland
city: Akron
country: United States
facility: Akron Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Pulmonary, Allergy & Critical Care Medicine, Cleveland Clinic Foundation
state: Ohio
city: Philadelphia
country: United States
facility: Temple University Lung Center
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
Study officials:
affiliation: University of Iowa Hospitals & Clinics, Iowa City, IA
name: Geoffrey McLennan, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Medical University of South Carolina
name: Charlie Strange, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Pulmonary Associates, Phoenix, AZ
name: Mark Gotfried, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: St Joseph's Medical Center, Towson, MD
name: Mark Krasna, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Akron Medical Center, Akron, OH
name: Sanjiv Tewari, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Temple University Lung Center, Philadelphia, PA
name: Gerard Criner, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Veritas Clinical Specialties, Topeka, KS
name: William Leeds, DO
role: PRINCIPAL_INVESTIGATOR
affiliation: Pulmonary, Allergy & Critical Care Medicine, Cleveland Clinic, Cleveland, OH
name: Thomas Gildea, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Ingenito EP, Berger RL, Henderson AC, Reilly JJ, Tsai L, Hoffman A. Bronchoscopic lung volume reduction using tissue engineering principles. Am J Respir Crit Care Med. 2003 Mar 1;167(5):771-8. doi: 10.1164/rccm.200208-842OC. Epub 2002 Oct 11.
citation: Reilly J, Washko G, Pinto-Plata V, Velez E, Kenney L, Berger R, Celli B. Biological lung volume reduction: a new bronchoscopic therapy for advanced emphysema. Chest. 2007 Apr;131(4):1108-13. doi: 10.1378/chest.06-1754.
citation: Criner GJ, Pinto-Plata V, Strange C, Dransfield M, Gotfried M, Leeds W, McLennan G, Refaely Y, Tewari S, Krasna M, Celli B. Biologic lung volume reduction in advanced upper lobe emphysema: phase 2 results. Am J Respir Crit Care Med. 2009 May 1;179(9):791-8. doi: 10.1164/rccm.200810-1639OC. Epub 2009 Jan 29.