The Role of Oral Glutathione on Growth Parameters in Children With Cystic Fibrosis

Cystic Fibrosis Clinical Trial

— GROW  

Official title:

A Multi Center Placebo Controlled Double Blind Randomized Study Evaluating the Role of Oral Glutathione on Growth Parameters in Children With Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03020719
• Other study ID #: GROW-IP-16
• Status: Completed
• Phase: Phase 2
• Start date: June 14, 2017
• Est. completion date: December 12, 2018

Study information

• Verified date: December 2019
• Source: University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this randomized, placebo-controlled (Phase II) study will be to further evaluate the effects of oral glutathione on growth in children with CF.

Description:

a prospective, multi-center, randomized, placebocontrolled, double-blind, Phase II clinical trial. Approximately sixty pancreatic insufficient (PI) subjects with CF who are ≥ 2 and < 11 years of age, will be enrolled to receive either L-Glutathione Reduced (GSH) or placebo given orally (tid) for 24 weeks. Each subject will be seen for four study visits: Visit 1 (Screening), Visit 2 (Baseline/Randomization, Day 0), Visit 3 (Week 12) and Visit 4 (Week 24). At Visit 2, subjects will be randomized to receive either active treatment or placebo. 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 and other bio-markers in blood and fecal specimens will be performed at Visits 2 and 4

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: December 12, 2018
• Est. primary completion date: December 12, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 11 Years

Eligibility

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'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

Exclusion Criteria:

• 1. Intestinal obstruction or gastrointestinal surgery within the 6 months prior to Day 0 2. History of diabetes, Crohn's disease, celiac disease, or bowel resection 3. Use of either oral or inhaled GSH or N-acetyl cysteine within the 4 months prior to Screening (Visit 1) 4. Known hypersensitivity to oral glutathione or lactose 5. Initiation of any new chronic therapy (e.g., ibuprofen, hypertonic saline, azithromycin, Pulmozyme, Cayston TOBI Kalydeco,Orkambi, Proton Pump Inhibitor, Histamine H-2 Blocker [PPI/H2-blocker], Miralax® , PERT, dietary supplementation, probiotics) within the 4 weeks prior to Day 0 6. Changes in the amount of proprietary dietary supplement formulas (e.g., Scandishakes, Boost, Pediasure, or homemade formula) given (oral or gastrostomy tube) within the 4 weeks prior to Day 0 7. Use of antibiotics (oral, IV, or inhaled) for acute symptoms within the 2 weeks prior to Day 0 8. Use of oral steroids within the 4 weeks prior to Day 0 9. Active treatment for nontuberculous mycobacteria (NTM) at Day 0 10. Active treatment for allergic bronchopulmonary aspergillosis (ABPA) at Day 0 11. Administration of any investigational drug within the 30 days prior to Day 0 12. Sibling who received study drug as part of this study 13. Presence of a condition or abnormality that in the opinion of the investigator would compromise the safety of the patient or the quality of the data

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Oral Glutathione
Oral Glutathione oral powder
• Placebo
Placebo oral powder

Locations

Country	Name	City	State
United States	Emory University Hospital	Atlanta	Georgia
United States	Childrens Hospital Colorado	Aurora	Colorado
United States	Women and Childrens Hospital of Buffalo	Buffalo	New York
United States	The University of Vermont Inc.	Burlington	Vermont
United States	University of Virginia Health System	Charlottesville	Virginia
United States	UT Southwestern Medical Center	Dallas	Texas
United States	Childrens Hospital of Michigan	Detroit	Michigan
United States	Helen DeVos Women and Childrens Center	Grand Rapids	Michigan
United States	Connecticut Childrens Medical Center	Hartford	Connecticut
United States	Baylor College of Medicine	Houston	Texas
United States	Riley Children's Hospital	Indianapolis	Indiana
United States	Nemours Childrens Clinic	Jacksonville	Florida
United States	The Childrens Mercy Hospital	Kansas City	Missouri
United States	Childrens Hospital of Milwaukee	Milwaukee	Wisconsin
United States	University of Minnesota Medical Center	Minneapolis	Minnesota
United States	Childrens Hospital of New York/Columbia University Medical Center	New York	New York
United States	Nemours Children's Clinic	Pensacola	Florida
United States	Oregon Health & Science University	Portland	Oregon

Sponsors (2)

Lead Sponsor	Collaborator
University of Minnesota	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Weight-for-age Z-score	Difference between the oral glutathione and placebo groups in the 24-week change from baseline in weight-for-age Z-score. Weight-for-age Z-scores are derived from the 2000 Centers for Disease Control and Prevention growth charts for U.S. children. The reference population in these growth charts is children surveyed by the National Center for Health Statistics from 1963-65 to 1988-94. The Z-score indicates the number of standard deviations away from the mean of the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.	Baseline to 24 weeks
Secondary	Change in Height-for-age Z-score	Difference between the oral glutathione and placebo groups in the 24-week change from baseline in height-for-age Z-score. Height-for-age Z-scores are derived from the 2000 Centers for Disease Control and Prevention growth charts for U.S. children. The reference population in these growth charts is children surveyed by the National Center for Health Statistics from 1963-65 to 1988-94. The Z-score indicates the number of standard deviations away from the mean of the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.	Baseline to 24 weeks
Secondary	Change in BMI-for-age Z-score	Difference between the oral glutathione and placebo groups in the 24-week change from baseline in BMI-for-age Z-score. BMI-for-age Z-scores are derived from the 2000 Centers for Disease Control and Prevention growth charts for U.S. children. The reference population in these growth charts is children surveyed by the National Center for Health Statistics from 1963-65 to 1988-94. The Z-score indicates the number of standard deviations away from the mean of the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.	Baseline to 24 weeks
Secondary	Change in Fecal Calprotectin	Difference between the oral glutathione and placebo groups in the 24-week change from baseline in fecal calprotectin.	Baseline to 24 weeks
Secondary	Change in High-sensitivity C-reactive Protein (Hs-CRP)	Difference between the oral glutathione and placebo groups in the 24-week change from baseline in hs-CRP.	Baseline to 24 weeks
Secondary	Adverse Events (AEs) and Serious Adverse Events (SAEs)	The number and percentage of participants with at least one event over the 24 week follow-up period.	Baseline to 24 weeks
Secondary	Rate of Adverse Events (AEs) and Serious Adverse Events (SAEs)	Rate is defined as the number of events per participant follow-up month.	Baseline to 24 weeks