US Biologic Lung Volume Reduction (BLVR) Phase 2 Emphysema Study

Phase 2 Study of the 20 mL Biologic Lung Volume Reduction System (BLVR) in Patients With Advanced Upper Lobe Predominant Emphysema

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.

Study Overview

• Status: Completed
• Conditions: Chronic Obstructive Pulmonary Disease; Pulmonary Emphysema
• Intervention / Treatment: Drug: Biologic Lung Volume Reduction (BLVR) - 20 mL Hydrogel

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.

• Study Type: Interventional
• Enrollment (Actual): 22
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Pulmonary Associates
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals & Clinics
  • Kansas
    • Topeka, Kansas, United States, 66606
      • Veritas Clinical Specialties, Ltd
  • Maryland
    • Towson, Maryland, United States, 21204
      • St Joseph's Medical Center
  • Ohio
    • Akron, Ohio, United States, 44302
      • Akron Medical Center
    • Cleveland, Ohio, United States, 44195
      • Pulmonary, Allergy & Critical Care Medicine, Cleveland Clinic Foundation
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19140
      • Temple University Lung Center
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina

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:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Group 1; Treatment will be administered in 2 treatment sessions.	Drug: Biologic Lung Volume Reduction (BLVR) - 20 mL Hydrogel; 20 mL Hydrogel
EXPERIMENTAL: Group 2; Treatment will be administered in a single treatment session.	Drug: Biologic Lung Volume Reduction (BLVR) - 20 mL Hydrogel; 20 mL Hydrogel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Reduction in gas trapping	12 weeks post treatment
SAEs - Safety of treatment and the procedure	2 years post treatment

Secondary Outcome Measures

Outcome Measure	Time Frame
Improvement in exercise capacity	12 weeks post treatment
Improvement in vital capacity	12 weeks post treatment
Improvement in expiratory flow	12 weeks post treatment
Improvement in inspiratory flow	12 weeks post treatment
Improvement in dyspnea symptoms (breathlessness)	12 weeks post treatment
Improvement in respiratory quality of life	12 weeks post treatment

Collaborators and Investigators

• Sponsor: Aeris Therapeutics
• Investigators: Principal Investigator: Mark Krasna, MD, St Joseph's Medical Center, Towson, MD; Principal Investigator: Thomas Gildea, MD, Pulmonary, Allergy & Critical Care Medicine, Cleveland Clinic, Cleveland, OH

Publications and helpful links

General Publications

• 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.
• 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.
• 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.

Study record dates

Study Major Dates

• Study Start: August 1, 2007
• Primary Completion (ACTUAL): April 1, 2008
• Study Completion (ACTUAL): December 1, 2009

Study Registration Dates

• First Submitted: August 9, 2007
• First Submitted That Met QC Criteria: August 10, 2007
• First Posted (ESTIMATE): August 13, 2007

Study Record Updates

• Last Update Posted (ESTIMATE): October 24, 2011
• Last Update Submitted That Met QC Criteria: October 21, 2011
• Last Verified: October 1, 2011

More Information

Terms related to this study

• Keywords: emphysema; chronic obstructive pulmonary disease; lung volume reduction
• 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: 01-C07-001