Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
Condition: Cystic Fibrosis; Pancreatic Insufficiency
MeSH terms: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Fibrosis
Study type: Interventional
Phase: Phase 3

Summary: 
      This is a Phase III, multicenter, open-label study, that will evaluate the improvement of
      nutrient absorption when participants will receive Ultrase® MT20. This study is sponsored by
      Aptalis (formerly Axcan). This study is performed in children from 7 to 11 years old.
    

Detailed description: 
      This is a Phase III, multicenter, open-label study, which will quantify the improvement of
      nutrient absorption when participants are receiving Ultrase® MT20. The improvement will be
      demonstrated by comparing the CFA percent (%) and CNA% obtained during a washout of enzyme
      with the CFA% and CNA% obtained during a period of treatment with Ultrase® MT20. The study is
      also designed to obtain safety data in CF children suffering also from PI taking Ultrase®
      MT20. The total duration for the participation of children in this study will be
      approximately up to 38 days and will include 3 phases: screening phase, the washout phase and
      treatment phase.

      Screening phase: this phase will last 15 days and all participants will take Ultrase® MT20 as
      per investigator's discretion during this period. During the last 4 days, participants will
      be stabilized on a high fat diet and with Ultrase® MT20. The individual 'stabilized dose' of
      Ultrase® MT20 capsules will be determined for each participant based on the average number of
      capsules of Ultrase® MT20 taken during last 4 days.

      Washout phase: this phase will last 6 to 7 days. The participants will continue the high-fat
      diet but will refrain from taking Ultrase® MT20 or any other enzymes. A 72-hour stool
      collection will be performed and all food consumed by the participants will be recorded to
      assess the CFA% and CNA%.

      Treatment phase: this phase will last 7 to 11 days. The participants will continue the
      high-fat diet and will take the 'stabilized dose' of Ultrase® MT20 established during
      screening. Another 72-hour stool collection will be performed and all food consumed by the
      participants will be recorded to assess the CFA% and CNA%.
    

Intervention name(s): Ultrase® MT20
Intervention type(s): Drug
Intervention description(s): Ultrase® MT20 capsules will be administered orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants will receive high-fat diet and Ultrase® MT20 dose will be adjusted depending on symptoms of steatorrhea. This will be followed by a washout phase of 6 to 7 days, in which participants will receive only high-fat diet; then stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) will be administered orally for 7 to 11 days during treatment phase. The stabilized dose should not to exceed 2500 lipase units per kilogram body weight per meal (lipase units/kg/meal).
Intervention MeSH terms: Pancrelipase; Pancreatin
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Cystic Fibrosis; Pancreatic insufficiency; Steatorrhea; Children 7 to 11 years; Pancreatic enzyme