A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis

Genentech

Status and phase: Completed; Phase 4

Conditions: Cystic Fibrosis

Treatments: Drug: dornase alfa [Pulmozyme®]

Study type: Interventional

Funder types: Industry

Identifiers

NCT01712334

ML28249

Details and patient eligibility

About

This phase IV, multicenter, randomized, open-label, two-period crossover study will evaluate the comparable efficacy and safety of Pulmozyme (dornase alfa) delivered by the eRapid nebulizer system in patients with cystic fibrosis. Patients who have been receiving Pulmozyme once daily chronically for at least 6 months will continue to receive Pulmozyme once daily for a run-in period of 2 weeks using the Pari LC Plus nebulizer. Patients will then be randomized to receive in a crossover design Pulmozyme once daily for two treatment periods of 2 weeks each using either the Pari LC Plus or the eRapid nebulizer. Anticipated time on study treatment is 6 weeks.

Enrollment: 99 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male and female patients, >/= 6 years of age
• Confirmed diagnosis of cystic fibrosis (CF)
• Receiving Pulmozyme once daily chronically for treatment of CF for at least 6 months prior to screening
• Percent predicted FEV1 >/= 40% at screening based on the Wang (males < 18 years, females < 16 years) or Hankinson (males >/= 18 years, females >/= 16 years) standardized equations
• Able to reproducibly perform spirometry testing and comply with study assessments

Exclusion criteria

• An acute respiratory infection or pulmonary exacerbation within 4 weeks prior to randomization
• Initiation of any new chronic therapy (e.g. inhaled corticosteroids, inhaled oral antibiotics, high-dose ibuprofen, hypertonic saline, ivacaftor) for respiratory disease within 4 weeks prior to randomization
• Changes in chest physiotherapy schedule within 4 weeks prior to randomization
• Hospitalization within 4 weeks prior to randomization
• Planned hospitalization during the 6-week study
• History of organ transplantation
• Participation in an investigational drug or device study within 30 day prior to screening

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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99 participants in 2 patient groups

eRapid Nebulizer

Experimental group

Description:

Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.

Treatment:

Drug: dornase alfa [Pulmozyme®]

Drug: dornase alfa [Pulmozyme®]

Jet Nebulizer

Active Comparator group

Description:

Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.

Treatment:

Drug: dornase alfa [Pulmozyme®]

Drug: dornase alfa [Pulmozyme®]