Trial Outcomes & Findings for The Role of Oral Glutathione on Growth Parameters in Children With Cystic Fibrosis (NCT NCT03020719)
NCT ID: NCT03020719
Last Updated: 2020-01-09
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
60 participants
Primary outcome timeframe
Baseline to 24 weeks
Results posted on
2020-01-09
Participant Flow
Participant milestones
| Measure |
Oral Glutathione
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Overall Study
STARTED
|
30
|
30
|
|
Overall Study
COMPLETED
|
30
|
28
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
The Role of Oral Glutathione on Growth Parameters in Children With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Oral Glutathione
n=30 Participants
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=30 Participants
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
Total
n=60 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
6.1 years
STANDARD_DEVIATION 2.43 • n=99 Participants
|
6 years
STANDARD_DEVIATION 2.87 • n=107 Participants
|
6.1 years
STANDARD_DEVIATION 2.64 • n=206 Participants
|
|
Age, Customized
Age Distribution · < 6 years
|
16 Participants
n=99 Participants
|
15 Participants
n=107 Participants
|
31 Participants
n=206 Participants
|
|
Age, Customized
Age Distribution · ≥ 6 years
|
14 Participants
n=99 Participants
|
15 Participants
n=107 Participants
|
29 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
18 Participants
n=99 Participants
|
17 Participants
n=107 Participants
|
35 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
29 Participants
n=99 Participants
|
27 Participants
n=107 Participants
|
56 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
29 Participants
n=99 Participants
|
30 Participants
n=107 Participants
|
59 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=99 Participants
|
30 participants
n=107 Participants
|
60 participants
n=206 Participants
|
|
Cystic Fibrosis (CF) Genotype
Delta F508 Homozygous
|
18 Participants
n=99 Participants
|
14 Participants
n=107 Participants
|
32 Participants
n=206 Participants
|
|
Cystic Fibrosis (CF) Genotype
Delta F508 Heterozygous
|
9 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
22 Participants
n=206 Participants
|
|
Cystic Fibrosis (CF) Genotype
Other
|
3 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Cystic Fibrosis (CF) Genotype
Unidentified
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Cystic Fibrosis (CF) Genotype
Not Available
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Weight (Z-score)
|
-0.57 z-score
STANDARD_DEVIATION 0.33 • n=99 Participants
|
-0.53 z-score
STANDARD_DEVIATION 0.35 • n=107 Participants
|
-0.55 z-score
STANDARD_DEVIATION 0.34 • n=206 Participants
|
|
Weight-for-age Z-score Distribution
< -0.52 (equiv. to < 30th percentile)
|
13 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
26 Participants
n=206 Participants
|
|
Weight-for-age Z-score Distribution
≥ -0.52 (equiv. to ≥ 30th percentile)
|
17 Participants
n=99 Participants
|
17 Participants
n=107 Participants
|
34 Participants
n=206 Participants
|
|
Fecal Elastase
< 200 mg/g
|
30 Participants
n=99 Participants
|
30 Participants
n=107 Participants
|
60 Participants
n=206 Participants
|
|
Fecal Elastase
≥ 200 mg/g
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Fecal Elastase
Not Available
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline to 24 weeksPopulation: Include participants with weight-for-age Z-score measurements at Baseline, Week 12 and Week 24
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.
Outcome measures
| Measure |
Oral Glutathione
n=30 Participants
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=28 Participants
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Change in Weight-for-age Z-score
|
-0.018 Z-score
Interval -0.124 to 0.087
|
0.045 Z-score
Interval -0.073 to 0.163
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksDifference 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.
Outcome measures
| Measure |
Oral Glutathione
n=30 Participants
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=28 Participants
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Change in Height-for-age Z-score
|
-0.04 Z-score
Standard Deviation 0.21
|
0.03 Z-score
Standard Deviation 0.16
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksDifference 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.
Outcome measures
| Measure |
Oral Glutathione
n=30 Participants
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=28 Participants
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Change in BMI-for-age Z-score
|
-0.01 Z-score
Standard Deviation 0.48
|
0.04 Z-score
Standard Deviation 0.44
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksPopulation: Include participants with fecal calprotectin measurements at Baseline and Week 24.
Difference between the oral glutathione and placebo groups in the 24-week change from baseline in fecal calprotectin.
Outcome measures
| Measure |
Oral Glutathione
n=29 Participants
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=26 Participants
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Change in Fecal Calprotectin
|
0.05 log base 10 transformation of ug/g
Standard Deviation 0.55
|
-0.05 log base 10 transformation of ug/g
Standard Deviation 0.53
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksDifference between the oral glutathione and placebo groups in the 24-week change from baseline in hs-CRP.
Outcome measures
| Measure |
Oral Glutathione
n=29 Participants
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=25 Participants
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Change in High-sensitivity C-reactive Protein (Hs-CRP)
|
-0.45 mg/L
Standard Deviation 3.4
|
-0.04 mg/L
Standard Deviation 1.6
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksThe number and percentage of participants with at least one event over the 24 week follow-up period.
Outcome measures
| Measure |
Oral Glutathione
n=30 Participants
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=30 Participants
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Adverse Events
|
29 Participants
|
29 Participants
|
|
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Serious Adverse Events
|
1 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksRate is defined as the number of events per participant follow-up month.
Outcome measures
| Measure |
Oral Glutathione
n=30 Participants
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=30 Participants
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Rate of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Adverse Events
|
0.89 number of events/total follow-up months
|
0.74 number of events/total follow-up months
|
|
Rate of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Serious Adverse Events
|
0.01 number of events/total follow-up months
|
0.05 number of events/total follow-up months
|
Adverse Events
Oral Glutathione
Serious events: 1 serious events
Other events: 29 other events
Deaths: 0 deaths
Placebo
Serious events: 5 serious events
Other events: 29 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Oral Glutathione
n=30 participants at risk
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=30 participants at risk
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
0.00%
0/30 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Infections and infestations
Cellulitis
|
0.00%
0/30 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Infections and infestations
Pneumonia
|
0.00%
0/30 • 24 weeks
|
3.3%
1/30 • Number of events 2 • 24 weeks
|
|
Investigations
Weight decreased
|
0.00%
0/30 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.00%
0/30 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Infective pulmonary exacerbation of cystic fibrosis
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
Other adverse events
| Measure |
Oral Glutathione
n=30 participants at risk
Oral Glutathione oral powder at 65mg/kg/day
Oral Glutathione: Oral Glutathione oral powder
|
Placebo
n=30 participants at risk
Placebo oral powder at 65mg/kg/day
Placebo: Placebo oral powder
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
13.3%
4/30 • Number of events 4 • 24 weeks
|
6.7%
2/30 • Number of events 3 • 24 weeks
|
|
Gastrointestinal disorders
Abdominal pain upper
|
6.7%
2/30 • Number of events 4 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Gastrointestinal disorders
Constipation
|
10.0%
3/30 • Number of events 3 • 24 weeks
|
13.3%
4/30 • Number of events 4 • 24 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
13.3%
4/30 • Number of events 4 • 24 weeks
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/30 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Gastrointestinal disorders
Steatorrhoea
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Gastrointestinal disorders
Vomiting
|
26.7%
8/30 • Number of events 9 • 24 weeks
|
20.0%
6/30 • Number of events 6 • 24 weeks
|
|
General disorders
Pyrexia
|
26.7%
8/30 • Number of events 12 • 24 weeks
|
16.7%
5/30 • Number of events 6 • 24 weeks
|
|
Infections and infestations
Gastroenteritis
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Infections and infestations
Influenza
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Infections and infestations
Otitis media
|
20.0%
6/30 • Number of events 6 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Infections and infestations
Sinusitis
|
20.0%
6/30 • Number of events 7 • 24 weeks
|
10.0%
3/30 • Number of events 3 • 24 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
13.3%
4/30 • Number of events 5 • 24 weeks
|
20.0%
6/30 • Number of events 8 • 24 weeks
|
|
Infections and infestations
Viral upper respiratory tract infection
|
10.0%
3/30 • Number of events 3 • 24 weeks
|
0.00%
0/30 • 24 weeks
|
|
Investigations
Alanine aminotransferase increased
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Investigations
Pulmonary function test decreased
|
0.00%
0/30 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Investigations
Weight decreased
|
16.7%
5/30 • Number of events 5 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Metabolism and nutrition disorders
Decreased appetite
|
6.7%
2/30 • Number of events 3 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Musculoskeletal and connective tissue disorders
Clubbing
|
0.00%
0/30 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Nervous system disorders
Headache
|
10.0%
3/30 • Number of events 3 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
43.3%
13/30 • Number of events 24 • 24 weeks
|
40.0%
12/30 • Number of events 14 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Infective pulmonary exacerbation of cystic fibrosis
|
33.3%
10/30 • Number of events 14 • 24 weeks
|
30.0%
9/30 • Number of events 11 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
13.3%
4/30 • Number of events 5 • 24 weeks
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
6.7%
2/30 • Number of events 5 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
0.00%
0/30 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/30 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
10.0%
3/30 • Number of events 4 • 24 weeks
|
6.7%
2/30 • Number of events 2 • 24 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place