A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis

NCT00680316 | Terminated Phase 4 Results

• 1 other identifier: Z4240g
• Study Type: interventional
• Target: 3
• Locations: 0 countries
• Sites: N/A

Brief Summary

This was a Phase IV, multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate the effect of Pulmozyme on pulmonary function, health-related quality of life (HRQOL), and respiratory symptoms in 3- to 5-year-old children with cystic fibrosis (CF). Approximately 40 patients were planned to be enrolled in this study. However, only 3 patients were eligible for random allocation and received treatment: 1 patient in the Pulmozyme group and 2 patients in the placebo group. All 3 patients completed the study assessments but did not have usable pulmonary function test (PFT) data.

Conditions

Cystic Fibrosis

Keywords

Pulmozyme; CF

Outcome Measures

Primary Outcomes (1)

• Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment).

  The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.

  from Visit 2 to Visit 3 (16 +/- 2 days)

Secondary Outcomes (3)

• Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)

  from Visit 2 to Visit 3 (16 +/- 2 days)

• Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10)

  from Visit 2 to Visit 3 (16 +/- 2 days)

• Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers

  from Visit 2 to Visit 3 (16 +/- 2 days)

Study Arms (2)

Dornase alfa

EXPERIMENTAL

Drug: Dornase alfa

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Dornase alfa DRUG

2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days

Also known as: Pulmozyme

Dornase alfa

Placebo DRUG

2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days

Placebo

Eligibility Criteria

• Age: 3 Years - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Signed Informed Consent Form
• Aged 3-5 years
• Diagnosis of cystic fibrosis

You may not qualify if:

• Children taking scheduled inhaled Pulmozyme or hypertonic saline within 56 days prior to Visit 1 or any Pulmozyme in the 28 days before Visit 1
• Involvement in a clinical intervention trial within the 4 weeks prior to Visit 1
• Use of an investigational drug or device within 28 days prior to Visit 1
• Any other condition that might increase the risk of participation to the patient in the judgement of the investigator

Sponsors & Collaborators

• Genentech, Inc.: lead

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

dornase alfa

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Limitations and Caveats

Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.

Results Point of Contact

• Title: Medical Communications
• Organization: Genentech, Inc.

800-821-8590

Study Officials

• Michelle Freemer, M.D.

  Genentech, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2008
• First Posted: May 20, 2008
• Study Start: June 1, 2008
• Primary Completion: May 1, 2009
• Study Completion: May 1, 2009
• Last Updated: January 12, 2017
• Results First Posted: June 17, 2011

Record last verified: 2016-11