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

NCT ID: NCT03020719

Last Updated: 2020-01-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 60 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-06-14
• Study Completion Date: 2018-12-12

Brief 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.

Detailed 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

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: TRIPLE

Study Groups

Oral Glutathione

Oral Glutathione oral powder at 65mg/kg/day

Group Type: EXPERIMENTAL

Oral Glutathione

Intervention Type: DRUG

Oral Glutathione oral powder

Placebo

Placebo oral powder at 65mg/kg/day

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo oral powder

Interventions

Oral Glutathione

Oral Glutathione oral powder

Intervention Type: DRUG

Placebo

Placebo oral powder

Intervention Type: DRUG

Other Intervention Names

GSH; Inactive powder

Eligibility Criteria

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

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 11 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cystic Fibrosis Foundation

OTHER

Sponsor Role: collaborator

University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sarah J Schwarzenberg, MD

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota

Molly Bozic, MD

Role: PRINCIPAL_INVESTIGATOR

Indiana University School of Medicine Riley Hospital

Locations

Childrens Hospital Colorado

Aurora, Colorado, United States

Connecticut Childrens Medical Center

Hartford, Connecticut, United States

Nemours Childrens Clinic

Jacksonville, Florida, United States

Nemours Children's Clinic

Pensacola, Florida, United States

Emory University Hospital

Atlanta, Georgia, United States

Riley Children's Hospital

Indianapolis, Indiana, United States

Childrens Hospital of Michigan

Detroit, Michigan, United States

Helen DeVos Women and Childrens Center

Grand Rapids, Michigan, United States

University of Minnesota Medical Center

Minneapolis, Minnesota, United States

The Childrens Mercy Hospital

Kansas City, Missouri, United States

Women and Childrens Hospital of Buffalo

Buffalo, New York, United States

Childrens Hospital of New York/Columbia University Medical Center

New York, New York, United States

Oregon Health & Science University

Portland, Oregon, United States

UT Southwestern Medical Center

Dallas, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

The University of Vermont Inc.

Burlington, Vermont, United States

University of Virginia Health System

Charlottesville, Virginia, United States

Childrens Hospital of Milwaukee

Milwaukee, Wisconsin, United States

Countries

United States

References

Bozic M, Goss CH, Tirouvanziam RM, Baines A, Kloster M, Antoine L, Borowitz D, Schwarzenberg SJ; GROW study group. Oral Glutathione and Growth in Cystic Fibrosis: A Multicenter, Randomized, Placebo-controlled, Double-blind Trial. J Pediatr Gastroenterol Nutr. 2020 Dec;71(6):771-777. doi: 10.1097/MPG.0000000000002948.

Reference Type: DERIVED

PMID: 32960827 (View on PubMed)

Provided Documents

Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

GROW-IP-16

Identifier Type: -

Identifier Source: org_study_id