A Multi-Center, Placebo-Controlled, Double-Blind, Randomized Study Evaluating the Role of Oral Glutathione on Growth Parameters in Children with Cystic Fibrosis.
Study ID: STU 082016-087
This will be a multi-center, placebo-controlled, double-blind, randomized, Phase ii clinical trial. approximately 60 pancreatic insufficient (Pi) subjects with CF, [GreaterThanorequalTo] 2 and [Less Than] 11 years of age, will be enrolled to participate in this study. For each subject, the total study duration may be 32 weeks. each subject will come to clinic for four study visits (see Figure 1). Screening: Visit 1 (Screening, Day -42), Visit 2 (Baseline, Day 0), Visit 3 (Week 12, Day 84) and Visit 4 (Week 24, Day 168). at Visit 2 (Baseline), subjects will be randomized to receive GSH 65 mg/kg/day or placebo (lactose 65mg/kg/day) for 24 weeks. Visit 1 and 2 may be combined if subject meets eligibility requirements and a fecal specimen is collected prior to dosing. Safety and clinical outcomes will be assessed throughout the study. assessment of inflammatory markers in blood and fecal will be performed at Visits 2 and 4. Subjects will be contacted by phone 4 and 8 weeks after Visit 2, as well as 4 and 8 weeks after Visit 3 and 2 weeks after Visit 4, to assess safety. if a subject had a clinically significant abnormal lab result or on-going treatment-related ae at Visit 4, the subject will be asked to return for a follow-up visit two weeks after Visit 4 instead of the call (Visit 5). Subjects will be on study for up to 32 weeks: Screening: up to 42 days Treatment: 24 weeks Follow-up: Phone call/visit 2 weeks after end of treatment
Inclusion Criteria: 1. Male or female >= 2 and < 11 years of age at Visit 1 2. Documentation of a CF diagnosis as evidenced by the following criteria: * Sweat chloride >= 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT) AND * Two well-characterized mutations in the cystic fibrosis transmembrane conductive regulator (CFTR) gene 3. Weight-for-age between the 10th and 50th percentiles at Screening (Visit 1) (using the Center for Disease Control (CDC) reference equations) 4. Current chronic use, greater than 8 weeks before Day 0, of pancreatic enzyme replacement therapy (PERT) for management of pancreatic insufficiency 5. Written informed consent (and assent when applicable) obtained from subject or subject[Single Quote]s legal representative and ability to comply with the requirements of the study 6. Clinically stable with no significant changes in health status within 2 weeks prior to Day 0