Effect of Lucinactant on Mucus Clearance in Cystic Fibrosis Lung Disease

• ClinicalTrials.gov Identifier: NCT00934362
• Recruitment Status: Completed
• First Posted: July 8, 2009
• Results First Posted: February 20, 2013
• Last Update Posted: March 13, 2017
• Sponsor: University of North Carolina, Chapel Hill
• Collaborators: Cystic Fibrosis Foundation; Windtree Therapeutics
• Information provided by (Responsible Party): Scott Donaldson, MD, University of North Carolina, Chapel Hill

Study Description

Brief Summary:

Mucus clearance is impaired in cystic fibrosis. Inhaled surfactants may reduce adhesive forces between mucus and airway surfaces and improve mucus clearance. This in turn my improve lung health. The investigators propose to measure mucus clearance before and after lucinactant or vehicle administration in patients with cystic fibrosis.

Condition or disease	Intervention/treatment	Phase
Cystic Fibrosis	Drug: Lucinactant first; Drug: Placebo first	Phase 2

Detailed Description:

This single-center pilot study is designed as a double-blind, randomized, cross-over clinical trial to evaluate the effects of inhaled lucinactant, an investigational peptide-containing synthetic surfactant (6 ml of 20 mg total phospholipid (TPL)/mL solution x 5 doses) in patients with mild to moderate CF lung disease. Lucinactant and vehicle will be delivered via a 510k approved vibrating mesh nebulizer, the Pari eFlowTM. The study duration corresponds to a 2-10 day screening phase, followed by a 20 day post-randomization phase that consists of two treatment periods (3 days each) and a washout period (14 days). A total of 16 patients will be enrolled and randomly assigned to one of two treatment sequences (Lucinactant followed by vehicle or vehicle followed by lucinactant). The primary outcome will be the rate of MC, as assessed via gamma scintigraphy, post-lucinactant and post vehicle. Secondary outcomes will include the rate of cough clearance (CC), lung clearance index (LCI), absolute change from baseline in FEV1 after 5 doses of study medication, CF-specific quality of life score (via CFQ-R instrument), in vitro assessments of sputum rheology, and various safety parameters.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 16 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Double Blind, Cross-Over Study Comparing Aerosolized Lucinactant and Vehicle on Mucociliary Clearance for Cystic Fibrosis Lung Disease
• Study Start Date: October 2008
• Actual Primary Completion Date: August 2010
• Actual Study Completion Date: August 2010

Arms and Interventions

Arm	Intervention/treatment
Lucinactant first, then placebo; Active treatment first, then washout period, then placebo treatment	Drug: Lucinactant first; lucinactant 120 mg (20 mg/ml) x 5 doses over 24 hours, then washout period x 14 days, then vehicle x 5 doses over 24 hrs; Other Name: KL-4 surfactant, lucinactant, aerosurf
Placebo treatment first, then lucinactant treatment; 0.9% NaCl vehicle treatment first, then washout period, then lucinactant treatment	Drug: Placebo first; 6 mL normal saline x 5 doses over 24 hours, then washout period x 14 days, then lucinactant x 5 doses over 24 hours; Other Names: normal saline; 0.9% NaCl

Outcome Measures

Primary Outcome Measures :

1. Change in Mucociliary Clearance [ Time Frame: 1 hour after final treatment (5th dose) minus baseline ]
   Clearance of radiolabeled particles, following inhalation, are followed over time. Average clearance rate through 60 minutes post inhaled isotope deposition is calculated. Absolute difference between baseline and post-treatment (e.g. <60 minutes after the last dose of lucinactant or placebo) reported.

Secondary Outcome Measures :

1. Spirometry [ Time Frame: after 5 doses ]
   Percent change (relative) in FEV1 between pre-treatment baseline and following 5 doses of study treatment. Post treatment values obtained 3 and approximately 22 hours after 5th dose were averaged to determine the treatment effect.

Eligibility Criteria

• Ages Eligible for Study: 14 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Cystic fibrosis
• FEV1>40%

Exclusion Criteria:

• Unstable lung disease
• Unable or unwilling to stop hypertonic saline and dornase alfa for 3 days prior to each study period
• Relevant drug allergy or intolerance
• Recent investigational drug use (30 days)

Contacts and Locations

Locations

United States, North Carolina

University of North Carolina

Chapel Hill, North Carolina, United States, 27599

Sponsors and Collaborators

University of North Carolina, Chapel Hill

Cystic Fibrosis Foundation

Windtree Therapeutics

Investigators

• Principal Investigator: Scott H Donaldson, MD; University of North Carolina, Chapel Hill

More Information

• Responsible Party: Scott Donaldson, MD, MD, University of North Carolina, Chapel Hill
• ClinicalTrials.gov Identifier: NCT00934362
• Other Study ID Numbers: 08-0795; KL4-CF-01; DONALD04A0
• First Posted: July 8, 2009
• Results First Posted: February 20, 2013
• Last Update Posted: March 13, 2017
• Last Verified: January 2013

Additional relevant MeSH terms:

Cystic Fibrosis; Lung Diseases; Pulmonary Fibrosis; Fibrosis; Pathologic Processes; Pancreatic Diseases; Digestive System Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Pulmonary Surfactants; Respiratory System Agents