Effectiveness of Pulmozyme in Infants With Cystic Fibrosis
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Study type: Interventional
Phase: Phase 2

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.
    

Detailed description: 
      Pulmozyme is given using a nebulizer and is now widely used in older children and adults with
      cystic fibrosis. In adults and older children, studies have shown that daily use of Pulmozyme
      improves lung function and decreases the number of lung infections requiring hospital
      treatment. Pulmozyme has been approved by the Food and Drug Administration for use in
      children over 5 years old and adults with cystic fibrosis. Pulmozyme has also been approved
      by the FDA for use in children with cystic fibrosis less than 5 years old based upon studies
      showing that it is safe in this age group and that it does get into the airway tubes as well
      in infants and toddlers as it does in older children and adults. Currently Pulmozyme is not
      widely used in children with cystic fibrosis younger than 5 years because no study has
      clearly shown that inhaling Pulmozyme daily improves lung function or improves clearance of
      mucus from the airway tubes in very young children. This study will measure whether Pulmozyme
      improves lung function and mucous clearance from the lungs in children with cystic fibrosis
      less than 3 ½ years of age.

      This study will compare Pulmozyme to a placebo. During the study infants and young children
      with cystic fibrosis will be treated with Pulmozyme for 6 months and placebo for 6 months.
      The study medicines will be inhaled at home once a day from a nebulizer for a period of one
      year. Half of the children will be treated with Pulmozyme for the first 6 months of the study
      and half will receive the placebo. At the 6 month point the group receiving Pulmozyme will be
      changed to the placebo and the group receiving placebo will be changed to Pulmozyme. The
      order of the 6 month treatment periods is randomized. This study is blinded. The study doctor
      and his staff will not know who is receiving Pulmozyme or placebo at any time during the
      study.

      Whether Pulmozyme works will be measured using infant lung function tests and by doing a
      special 3-D x-ray of the child's chest (a high resolution CT or HRCT) at the beginning of the
      study, at 6 months and at 12 month after starting study. The study will not change the
      regular clinical care.
    

Intervention name(s): Recombinant Human DNase (Pulmozyme); Placebos
Intervention type(s): Drug; Drug
Intervention description(s): 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; 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
Other intervention name(s): Pulmozyme
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Cystic fibrosis; Infants; Children; Pulmozyme; Pulmonary function; Computed tomography