Effect of Lucinactant on Mucus Clearance in Cystic Fibrosis Lung Disease

NCT ID: NCT00934362

Last Updated: 2017-03-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-10-31
• Study Completion Date: 2010-08-31

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.

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.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Lucinactant first, then placebo

Active treatment first, then washout period, then placebo treatment

Group Type: OTHER

Lucinactant first

Intervention Type: DRUG

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

Placebo treatment first, then lucinactant treatment

0.9% NaCl vehicle treatment first, then washout period, then lucinactant treatment

Group Type: OTHER

Placebo first

Intervention Type: DRUG

6 mL normal saline x 5 doses over 24 hours, then washout period x 14 days, then lucinactant x 5 doses over 24 hours

Interventions

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

Intervention Type: 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

Intervention Type: DRUG

Other Intervention Names

KL-4 surfactant, lucinactant, aerosurf; normal saline; 0.9% NaCl

Eligibility 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)

• Minimum Eligible Age: 14 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cystic Fibrosis Foundation

OTHER

Sponsor Role: collaborator

Windtree Therapeutics

INDUSTRY

Sponsor Role: collaborator

University of North Carolina, Chapel Hill

OTHER

Sponsor Role: lead

Responsible Party

Scott Donaldson, MD

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Scott H Donaldson, MD

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina, Chapel Hill

Locations

University of North Carolina

Chapel Hill, North Carolina, United States

Countries

United States

Other Identifiers

KL4-CF-01

Identifier Type: -

Identifier Source: secondary_id

DONALD04A0

Identifier Type: -

Identifier Source: secondary_id

08-0795

Identifier Type: -

Identifier Source: org_study_id