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

NCT03020719 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: GROW-IP-16
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 18

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.

Conditions

Cystic Fibrosis

Keywords

Growth Parameters; Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• Change in Height-for-age Z-score

  Baseline to 24 weeks

• Change in BMI-for-age Z-score

  Baseline to 24 weeks

• Change in Fecal Calprotectin

  Baseline to 24 weeks

• Change in High-sensitivity C-reactive Protein (Hs-CRP)

  Baseline to 24 weeks

• Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Baseline to 24 weeks

Study Arms (2)

Oral Glutathione

EXPERIMENTAL

Oral Glutathione oral powder at 65mg/kg/day

Drug: Oral Glutathione

Placebo

PLACEBO COMPARATOR

Placebo oral powder at 65mg/kg/day

Drug: Placebo

Interventions

Oral Glutathione DRUG

Oral Glutathione oral powder

Also known as: GSH

Oral Glutathione

Placebo DRUG

Placebo oral powder

Also known as: Inactive powder

Placebo

Eligibility Criteria

• Age: 2 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female ≥ 2 and \< 11 years of age at Visit 1
• 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
• Weight-for-age between the 10th and 50th percentiles at Screening (Visit 1) (using the Center for Disease Control (CDC) reference equations)
• Current chronic use, greater than 8 weeks before Day 0, of pancreatic enzyme replacement therapy (PERT) for management of pancreatic insufficiency
• 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
• Clinically stable with no significant changes in health status within 2 weeks prior to Day 0

You may not qualify if:

• \. 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

Sponsors & Collaborators

• University of Minnesota: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (18)

Childrens Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Connecticut Childrens Medical Center

Hartford, Connecticut, 06107, United States

Location

Nemours Childrens Clinic

Jacksonville, Florida, 32207, United States

Location

Nemours Children's Clinic

Pensacola, Florida, 32504, United States

Location

Emory University Hospital

Atlanta, Georgia, 30324, United States

Location

Riley Children's Hospital

Indianapolis, Indiana, 46202, United States

Location

Childrens Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Helen DeVos Women and Childrens Center

Grand Rapids, Michigan, 49503, United States

Location

University of Minnesota Medical Center

Minneapolis, Minnesota, 55414, United States

Location

The Childrens Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Women and Childrens Hospital of Buffalo

Buffalo, New York, 14222, United States

Location

Childrens Hospital of New York/Columbia University Medical Center

New York, New York, 10032, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

The University of Vermont Inc.

Burlington, Vermont, 05401, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

Childrens Hospital of Milwaukee

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (1)

• 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.

  PMID: 32960827 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Glutathione

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Oligopeptides; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Arthur Baines
• Organization: Seattle Children's Hospital

Arthur.Baines@seattlechildrens.org

2068841431

Study Officials

• Sarah J Schwarzenberg, MD

  University of Minnesota

  PRINCIPAL INVESTIGATOR

• Molly Bozic, MD

  Indiana University School of Medicine Riley Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 11, 2017
• First Posted: January 13, 2017
• Study Start: June 14, 2017
• Primary Completion: December 12, 2018
• Study Completion: December 12, 2018
• Last Updated: January 9, 2020
• Results First Posted: January 9, 2020

Record last verified: 2019-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (18)