Effectiveness of Pulmozyme in Infants With Cystic Fibrosis

NCT00179998 | Completed Phase 2 Results

• 1 other identifier: Z2910s
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.

Conditions

Cystic Fibrosis

Keywords

Cystic fibrosis; Infants; Children; Pulmozyme; Pulmonary function; Computed tomography

Outcome Measures

Primary Outcomes (2)

• Chest CT (High Resolution Computed Tomography (HRCT) Score)

  Change in Total HRCT Score from initiation of intervention to 6 months Modified Maffessanti HRCT Scoring System Airways 1. Bronchial Wall Thickening:1 = mild, 2 = moderate, 3 = severe 2. Bronchiectasis:1 = mild, 2 = moderate, 3 = severe 3. Axial extent of 1 or 2: 1 = central/middle, 2 = also periphery 4. Regional extent of 1 or 2: x 1 if \< 50 %, x 2 if \> 50 % 5. Gas trapping score:0 if 1 sub-segment, 1 if \< 25 %, 2 if 25 - 50 %, 3 if 50 - 75 %, 4 if \> 75 % Multiply (# 1 + # 2 + # 3) by # 4 then add # 5 Parenchyma 1. Airspace disease: 0 = none, 1 = present 2. Ground glass opacity: 0 = none, 1 = present 3. Mucous Plugging: 0 = none, 1 = present Total Score = Airway + Parenchymal Scores for RUL, LUL, RLL, and LLL Sections. The Total Score ranges from 12 to 92, with higher scores indicating greater impairment. Maximum Score = 4 x 23 = 92

  6 months

• Infant Pulmonary Function Tests (FEV0.5)

  Change in FEV0.5 from initiation of intervention to 6 months

  6 months

Secondary Outcomes (1)

• Antibiotic Treatment Days

  per 6 month interval

Study Arms (2)

Recombinant Human DNase (Pulmozyme) then Placebo

ACTIVE COMPARATOR

once daily nebulized rhDNAse

Drug: Recombinant Human DNase (Pulmozyme); Drug: Placebos

Placebo (Nebulized Saline) then rhDNase

PLACEBO COMPARATOR

once daily nebulized vehicle

Drug: Recombinant Human DNase (Pulmozyme); Drug: Placebos

Interventions

Recombinant Human DNase (Pulmozyme) DRUG

2.5 mg in 3 ml diluent delivered by nebulization given daily for 6 months with 3 ml diluent placebo delivered by nebulization given daily for 6 months

Also known as: Pulmozyme

Placebo (Nebulized Saline) then rhDNase; Recombinant Human DNase (Pulmozyme) then Placebo

Placebos DRUG

2.5 ml sterile solution (8.77 mg/ml sodium chloride, 0.15 mg/ml calcium chloride, pH 7.0 +/- 2.0) delivered daily by nebulization for 6 months, either preceding or following 6 months of Pulmozyme depending on randomization of the subject

Placebo (Nebulized Saline) then rhDNase; Recombinant Human DNase (Pulmozyme) then Placebo

Eligibility Criteria

• Age: 1 Month - 30 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age \< 30 months
• Diagnosis of CF based on clinical features consistent with CF as well as 1 of the 2 following criteria: a) two sweat chlorides \>60 mEq/L (by quantitative pilocarpine iontophoresis), b) genotype with 2 identifiable mutations consistent with CF.
• Informed consent by parent or legal guardian

You may not qualify if:

• Previous treatment with Pulmozyme
• Hospitalization or treatment with IV antibiotics with 14 days of initial study visit
• Acute intercurrent respiratory infection, defined as any of the following symptoms within the preceding 48 hours: 1) fever \> 38 degrees C, 2) new onset of coryza or other upper respiratory symptoms, 3) increase in cough, wheezing, or respiratory rate
• History of adverse reaction to sedation
• Oxyhemoglobin saturation \<90% on room air
• Severe upper airway obstruction as determined by site PI (severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnea)
• Hemodynamically significant congenital heart disease or diagnosed arrhythmias
• History of hemoptysis
• History of previous pulmonary air leak (pneumothorax)
• Use of Investigational drug(s) within 60 days or 5 half-lives of enrollment in this study.
• Known allergy to Chinese Hamster Ovary-derived biological products or any component of the placebo or active drug formulations.

Sponsors & Collaborators

• Nationwide Children's Hospital: lead
• Genentech, Inc.: collaborator

Study Sites (1)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

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

Dr. Castile is retired and no longer is associated with Nationwide Children's Hospital and no one associated with the trial works at NCH any longer. We are unable to further update this study in clinicaltrials.gov.

Results Point of Contact

• Title: Dr. Robert Castile
• Organization: Nationwide Children's Hospital

robert.castile@nationwidechildrens.org

614-355-6670

Study Officials

• Robert G Castile, MD

  The Research Institute at Nationwide Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2005
• First Posted: September 16, 2005
• Study Start: January 1, 2005
• Primary Completion: October 1, 2008
• Study Completion: July 1, 2016
• Last Updated: May 9, 2019
• Results First Posted: May 9, 2019

Record last verified: 2019-04

Locations

US (1)