Trial Outcomes & Findings for Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children (NCT NCT01529268)
NCT ID: NCT01529268
Last Updated: 2021-06-10
Results Overview
Centrally scored and masked assessment of histologic improvement in Nonalcholic Fatty Liver Disease (NAFLD) between the baseline liver biopsy and follow-up biopsy after 52 weeks of treatment, where improvement is defined as: (1) decrease in the NAFLD Activity Score (NAS) of 2 or more and (2) no worsening of fibrosis.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
169 participants
Primary outcome timeframe
52 weeks
Results posted on
2021-06-10
Participant Flow
Patients were enrolled at 10 NASH CRN clinical centers from June 2012 to January 2014.
Participant milestones
| Measure |
DR Cysteamine Bitartrate Capsule
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Overall Study
STARTED
|
88
|
81
|
|
Overall Study
Completed 52-week Biopsy
|
71
|
75
|
|
Overall Study
COMPLETED
|
76
|
78
|
|
Overall Study
NOT COMPLETED
|
12
|
3
|
Reasons for withdrawal
| Measure |
DR Cysteamine Bitartrate Capsule
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
9
|
3
|
|
Overall Study
Moved
|
3
|
0
|
Baseline Characteristics
Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children
Baseline characteristics by cohort
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
Total
n=169 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
13.8 years
STANDARD_DEVIATION 2.9 • n=5 Participants
|
13.6 years
STANDARD_DEVIATION 2.5 • n=7 Participants
|
13.7 years
STANDARD_DEVIATION 2.7 • n=5 Participants
|
|
Sex: Female, Male
Female
|
25 Participants
n=5 Participants
|
25 Participants
n=7 Participants
|
50 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
63 Participants
n=5 Participants
|
56 Participants
n=7 Participants
|
119 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian/Alaska Native
|
5 participants
n=5 Participants
|
6 participants
n=7 Participants
|
11 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African-American
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
56 participants
n=5 Participants
|
46 participants
n=7 Participants
|
102 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
More than one race
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Refusal/not stated
|
21 participants
n=5 Participants
|
23 participants
n=7 Participants
|
44 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
88 participants
n=5 Participants
|
81 participants
n=7 Participants
|
169 participants
n=5 Participants
|
|
Alanine aminotransferase
|
140 U/L
STANDARD_DEVIATION 118 • n=5 Participants
|
103 U/L
STANDARD_DEVIATION 76 • n=7 Participants
|
123 U/L
STANDARD_DEVIATION 101 • n=5 Participants
|
|
Aspartate aminotransferase
|
82 U/L
STANDARD_DEVIATION 71 • n=5 Participants
|
59 U/L
STANDARD_DEVIATION 38 • n=7 Participants
|
71 U/L
STANDARD_DEVIATION 59 • n=5 Participants
|
|
Weight group
Less than or equal to 65 kg
|
24 participants
n=5 Participants
|
23 participants
n=7 Participants
|
47 participants
n=5 Participants
|
|
Weight group
>65-80 kg
|
14 participants
n=5 Participants
|
10 participants
n=7 Participants
|
24 participants
n=5 Participants
|
|
Weight group
>80 kg
|
50 participants
n=5 Participants
|
48 participants
n=7 Participants
|
98 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 52 weeksPopulation: Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement.
Centrally scored and masked assessment of histologic improvement in Nonalcholic Fatty Liver Disease (NAFLD) between the baseline liver biopsy and follow-up biopsy after 52 weeks of treatment, where improvement is defined as: (1) decrease in the NAFLD Activity Score (NAS) of 2 or more and (2) no worsening of fibrosis.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Improvement in Nonalcoholic Fatty Liver Disease (NAFLD)
|
25 participants
|
18 participants
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis).
Change from baseline in the NAFLD Activity Score (NAS), which is a composite score equal to the sum of the steatosis grade (0-3), lobular inflammation grade (0-3), and hepatocellular ballooning grade (0-2), from centralized pathologist scoring of liver biopsies. The overall scale of the NAS is 0-8, with higher scores indicating more severe disease. The outcome measure, change from baseline in NAFLD Activity Score (NAS), has a possible range from -8 to +8, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome. Components of the NAS are scored as follows: Steatosis grade 0=\<5% steatosis, 1=5-33% steatosis, 2=34-66% steatosis, 3=\>66% steatosis. Lobular inflammation grade=amount of lobular inflammation (combines mononuclear, fat granulomas, and polymorphonuclear (pmn) foci): 0=0, 1=\<2 under 20x magnification, 2=2-4 under 20x magnification, 3=\>4 under 20x magnification. Hepatocellular ballooning 0=none, 1=mild, 2=more than mild.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=71 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=75 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Nonalcoholic Fatty Liver Disease (NAFLD) Activity Score (NAS)
|
-0.8 units on a scale
Standard Deviation 1.8
|
-0.8 units on a scale
Standard Deviation 1.8
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement.
Improvement in steatosis defined as any decrease in steatosis grade comparing 52-week biopsy to baseline.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Steatosis: Patients With Improvement
|
26 participants
|
33 participants
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis).
Change from baseline in steatosis score. Steatosis score is based on central pathologist grading of liver biopsies: 0=\<5% steatosis; 1=5-33% steatosis, 2=34-66% steatosis, 3=\>66% steatosis. Change in steatosis score has a possible range of -3 to +3, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement).
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=71 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=75 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Steatosis: Change in Score
|
-0.3 units on a scale
Standard Deviation 0.9
|
-0.4 units on a scale
Standard Deviation 0.9
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement.
Improvement in lobular inflammation defined as any decrease in lobular inflammation grade comparing 52-week biopsy to baseline.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Lobular Inflammation: Patients With Improvement
|
32 participants
|
17 participants
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis).
Change from baseline in lobular inflammation score. The amount of lobular inflammation is based on central pathologist grading of liver biopsies, and combines mononuclear, fat granulomas, and polymorphonuclear (pmn) foci: 0=none; 1=\<2 under 20x magnification, 2=2-4 under 20x magnification, 3=\>4 under 20x magnification. Change in lobular inflammation score has a possible range of -3 to +3, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement).
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=71 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=75 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Lobular Inflammation: Change in Score
|
-0.4 units on a scale
Standard Deviation 0.8
|
-0.1 units on a scale
Standard Deviation 0.8
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement.
Improvement in hepatocellular ballooning defined as any decrease in hepatocellular ballooning score comparing 52-week biopsy to baseline.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Hepatocellular Ballooning: Patients With Improvement
|
17 participants
|
21 participants
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis).
Change from baseline in hepatocellular ballooning score. The amount of hepatocellular ballooning is based on central pathologist grading of liver biopsies: 0=none; 1=few ballooned hepatocytes, 2=many ballooned hepatocytes. Change in hepatocellular ballooning score has a possible range of -2 to +2, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement).
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=71 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=75 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Hepatocellular Ballooning: Change in Score
|
-0.1 units on a scale
Standard Deviation 0.7
|
-0.3 units on a scale
Standard Deviation 0.8
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement.
Improvement in portal inflammation defined as any decrease in portal inflammation score comparing 52-week biopsy to baseline.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Portal Inflammation: Patients With Improvement
|
18 participants
|
14 participants
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis).
Change from baseline in portal inflammation score. The amount of portal inflammation is based on central pathologist grading of liver biopsies: 0=none; 1=mild, 2=more than mild. Change in portal inflammation score has a possible range of -2 to +2, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement).
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=71 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=75 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Portal Inflammation: Change in Score
|
-0.1 units on a scale
Standard Deviation 0.6
|
-0.1 units on a scale
Standard Deviation 0.6
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement.
Improvement in fibrosis stage defined as any decrease in fibrosis stage comparing 52-week biopsy to baseline.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Fibrosis: Patients With Improvement
|
25 participants
|
23 participants
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis).
Change from baseline in fibrosis stage. The amount of fibrosis is based on central pathologist grading of liver biopsies: 0=none; 1a=mild, zone 3 perisinusoidal, 1b=moderate, zone 3, perisinusoidal, 1c=portal/periportal only, 2=zone 3 and periportal, any combination, 3=bridging, 4=cirrhosis. Fibrosis stages 1a, 1b, 1c recoded as 1, so the possible range of values for fibrosis stage was 0-4. Change in fibrosis stage has a possible range of -4 to +4, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement).
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=71 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=75 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Fibrosis: Change in Stage
|
-0.3 units on a scale
Standard Deviation 0.9
|
-0.1 units on a scale
Standard Deviation 1.0
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: Analysis was limited to patients with a diagnosis of definite NASH at baseline.
Patients with a change from a histological diagnosis of definite NASH or indeterminate for NASH to not NASH at end of treatment
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=24 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=23 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Resolution of NASH
|
4 participants
|
2 participants
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: The smaller number of participants analyzed is due to missing 52-week laboratory data.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Serum Aminotransferase and Gamma-glutamyl Transpeptidase
Alanine aminotransferase
|
-53 U/L
Standard Deviation 88
|
-8 U/L
Standard Deviation 77
|
|
Change in Serum Aminotransferase and Gamma-glutamyl Transpeptidase
Aspartate aminotransferase
|
-31 U/L
Standard Deviation 52
|
-4 U/L
Standard Deviation 36
|
|
Change in Serum Aminotransferase and Gamma-glutamyl Transpeptidase
Gamma-glutamyl transpeptidase
|
-10 U/L
Standard Deviation 23
|
-1 U/L
Standard Deviation 16
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: Smaller number of patients analyzed due to missing 52-week weight measurement.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Weight (kg)
|
6.3 kg
Standard Deviation 9.3
|
7.8 kg
Standard Deviation 6.6
|
SECONDARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Body-mass Index
|
0.8 kg/m^2
Standard Deviation 2.8
|
1.1 kg/m^2
Standard Deviation 2.2
|
SECONDARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Body-mass Index Z-score
|
-0.1 SD
Standard Deviation 0.3
|
0 SD
Standard Deviation 0.2
|
SECONDARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Waist Circumference
|
2.5 cm
Standard Deviation 7.7
|
2.3 cm
Standard Deviation 7.5
|
SECONDARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Fasting Serum Glucose
|
1 mg/dL
Standard Deviation 12
|
5 mg/dL
Standard Deviation 27
|
SECONDARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Fasting Insulin
|
6 μU/mL
Standard Deviation 36
|
10 μU/mL
Standard Deviation 40
|
SECONDARY outcome
Timeframe: 52 weeks(Glucose (mmol/L) x insulin (pmol/L))/22.5
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in HOMA-IR
|
1.4 (10E-15 mol^2)/L^2
Standard Deviation 9.2
|
3.6 (10E-15 mol^2)/L^2
Standard Deviation 12.5
|
SECONDARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Systolic Blood Pressure
|
3 mmHg
Standard Deviation 12
|
2 mmHg
Standard Deviation 12
|
SECONDARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Diastolic Blood Pressure
|
-1 mmHg
Standard Deviation 9
|
1 mmHg
Standard Deviation 9
|
SECONDARY outcome
Timeframe: 52 weeksPediatric Quality of Life Inventory (PedsQL) version 4.0 is completed by both the child and parent/caregiver, and is composed of 23 items comprising 4 dimensions: Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Scores are transformed on a scale from 0 to 100, with higher scores indicating better health-related quality of life. Physical Health Summary Score =Physical Functioning Scale Score. Psychosocial Health Summary Score = Sum of items over the number of items answered in the Emotional, Social, and School Functioning Scales.
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=75 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=77 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Change in Pediatric Quality of Life Inventory (PedsQL) Score
Self-reported physical health
|
4 units on a scale
Standard Deviation 17
|
5 units on a scale
Standard Deviation 16
|
|
Change in Pediatric Quality of Life Inventory (PedsQL) Score
Self-reported psychosocial health
|
4 units on a scale
Standard Deviation 15
|
5 units on a scale
Standard Deviation 14
|
|
Change in Pediatric Quality of Life Inventory (PedsQL) Score
Parent/guardian-reported physical health
|
4 units on a scale
Standard Deviation 27
|
5 units on a scale
Standard Deviation 24
|
|
Change in Pediatric Quality of Life Inventory (PedsQL) Score
Parent/guardian-reported psychosocial health
|
5 units on a scale
Standard Deviation 18
|
6 units on a scale
Standard Deviation 24
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: The smaller number of observations is because MRI was an optional procedure. This is the number with MRI exams at both baseline and 52 weeks.
Change from baseline in MRI Proton Density Fat Fraction (PDFF) (%).
Outcome measures
| Measure |
DR Cysteamine Bitartrate Capsule
n=38 Participants
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=46 Participants
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Reduction in MRI-determined Hepatic Fat Fraction
|
-5.3 percentage of PDFF
Standard Deviation 7.1
|
-2.6 percentage of PDFF
Standard Deviation 7.6
|
Adverse Events
DR Cysteamine Bitartrate Capsule
Serious events: 5 serious events
Other events: 62 other events
Deaths: 0 deaths
DR Cysteamine Bitartrate Placebo
Serious events: 4 serious events
Other events: 55 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 participants at risk
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 participants at risk
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Hepatobiliary disorders
Pain - Gallbladder
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection - Appendix
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Psychiatric disorders
Mood alteration - depression
|
1.1%
1/88 • Number of events 2 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Endocrine disorders
Diabetes
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Hepatobiliary disorders
Cholecystitis
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Soft tissue necrosis
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Pain - Abdomen NOS
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Psychiatric disorders
Personality/behavioral
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
Other adverse events
| Measure |
DR Cysteamine Bitartrate Capsule
n=88 participants at risk
Active DR cysteamine bitartrate capsule
DR cysteamine bitartrate capsule: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
DR Cysteamine Bitartrate Placebo
n=81 participants at risk
Placebo DR cysteamine bitartrate capsule
DR cysteamine bitartrate placebo: ◦ 600 mg/day (four 75 mg capsules twice daily) for patients ≤ 65 kg at baseline
* 750 mg/day (five 75 mg capsules twice daily) for patients \>65 - 80 kg at baseline
* 900 mg/day (six 75 mg capsules twice daily) for patients \>80 kg at baseline
|
|---|---|---|
|
Gastrointestinal disorders
Pain - abdomen NOS
|
12.5%
11/88 • Number of events 11 • 52 weeks
|
9.9%
8/81 • Number of events 9 • 52 weeks
|
|
Eye disorders
Ocular - other
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Immune system disorders
Allergic reaction
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Immune system disorders
Rhinitis
|
3.4%
3/88 • Number of events 3 • 52 weeks
|
6.2%
5/81 • Number of events 5 • 52 weeks
|
|
Immune system disorders
Allergy - other
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Psychiatric disorders
Mood alteration - anxiety
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Fracture
|
4.5%
4/88 • Number of events 4 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Constipation
|
4.5%
4/88 • Number of events 4 • 52 weeks
|
4.9%
4/81 • Number of events 5 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.8%
6/88 • Number of events 7 • 52 weeks
|
9.9%
8/81 • Number of events 10 • 52 weeks
|
|
Renal and urinary disorders
Cystitis
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Gastrointestinal disorders
Teeth
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Endocrine disorders
Diabetes
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
13.6%
12/88 • Number of events 12 • 52 weeks
|
13.6%
11/81 • Number of events 13 • 52 weeks
|
|
Nervous system disorders
Dizziness
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/88 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Nervous system disorders
Pain - head/headache
|
10.2%
9/88 • Number of events 9 • 52 weeks
|
7.4%
6/81 • Number of events 6 • 52 weeks
|
|
Gastrointestinal disorders
Vomiting
|
6.8%
6/88 • Number of events 7 • 52 weeks
|
11.1%
9/81 • Number of events 10 • 52 weeks
|
|
Gastrointestinal disorders
Pain - stomach
|
4.5%
4/88 • Number of events 4 • 52 weeks
|
4.9%
4/81 • Number of events 4 • 52 weeks
|
|
Gastrointestinal disorders
Nausea
|
5.7%
5/88 • Number of events 5 • 52 weeks
|
6.2%
5/81 • Number of events 6 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
3.4%
3/88 • Number of events 3 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.5%
4/88 • Number of events 5 • 52 weeks
|
1.2%
1/81 • Number of events 2 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Striae
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Infection - skin
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Reproductive system and breast disorders
Pain - testicle
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Vascular disorders
Vasculitis
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection - urinary tract NOS
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Psychiatric disorders
Mood alteration - not specified
|
0.00%
0/88 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Psychiatric disorders
Mood alteration - depression
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Psychiatric disorders
Mood alteration - irritability
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection - ungual
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
General disorders
Hemorrhage - pulmonary - nose
|
3.4%
3/88 • Number of events 5 • 52 weeks
|
4.9%
4/81 • Number of events 8 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain - joint
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pain - chest/thorax NOS
|
4.5%
4/88 • Number of events 4 • 52 weeks
|
1.2%
1/81 • Number of events 2 • 52 weeks
|
|
Reproductive system and breast disorders
Pain - breast
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Hepatobiliary disorders
Metabolic/lab - other
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Bone growth - spine
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Hemorrhage, GI - rectum
|
0.00%
0/88 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Reproductive system and breast disorders
Hemorrhage, GU - vaginal
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Cardiac disorders
Hypertension
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
3.7%
3/81 • Number of events 3 • 52 weeks
|
|
Reproductive system and breast disorders
Gynecomastia
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Gastrointestinal disorders
Heartburn
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
3.7%
3/81 • Number of events 5 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Hyperpigmentation
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Hypopigmentation
|
0.00%
0/88 • 52 weeks
|
2.5%
2/81 • Number of events 3 • 52 weeks
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
General disorders
Fatigue
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Extremity - upper
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Fever
|
3.4%
3/88 • Number of events 3 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Endocrine disorders
Metabolic/lab - other
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection - pharynx
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Infection - larynx
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Infection - stomach
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
General disorders
Insomnia
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Reproductive system and breast disorders
Irregular menses
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Joint - function
|
0.00%
0/88 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Lumbar spine ROM
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal/paranasal reactions
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection - middle ear (otitis media)
|
4.5%
4/88 • Number of events 5 • 52 weeks
|
3.7%
3/81 • Number of events 3 • 52 weeks
|
|
Ear and labyrinth disorders
Pain - middle ear
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Pain - skin
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pain - chest wall
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain (extremity - limb)
|
3.4%
3/88 • Number of events 3 • 52 weeks
|
3.7%
3/81 • Number of events 4 • 52 weeks
|
|
Injury, poisoning and procedural complications
Pain - bone
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Cardiac disorders
Palpitations
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Eye disorders
Ocular surface disease
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Ulceration
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Infection (middle ear) with unknown ANC
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection (ungual) with unknown ANC
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Infection (sinus) with unknown ANC
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection - upper airway NOS
|
3.4%
3/88 • Number of events 4 • 52 weeks
|
3.7%
3/81 • Number of events 3 • 52 weeks
|
|
Surgical and medical procedures
Sexual - other (orchiopexy)
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary - other (cold)
|
3.4%
3/88 • Number of events 3 • 52 weeks
|
3.7%
3/81 • Number of events 3 • 52 weeks
|
|
Infections and infestations
Infection - bronchus
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatology - other
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
General disorders
Flu-like syndrome
|
3.4%
3/88 • Number of events 3 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatology - other (warts, HPV)
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection - sinus
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Infection (documented clinically) - pharynx
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Infections and infestations
Infection - lung (pneumonia)
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Gastrointestinal disorders
Enteritis
|
0.00%
0/88 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary - other
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Neuropathy - sensory
|
2.3%
2/88 • Number of events 2 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Injury, poisoning and procedural complications
Neurology - other (concussion)
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Injury, poisoning and procedural complications
Intraop injury - liver
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pain - throat/pharynx/larynx
|
0.00%
0/88 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Injury, poisoning and procedural complications
Dermatology - other
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Musculoskeletal - other (ankle sprain)
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Injury, poisoning and procedural complications
Musculoskeletal - other (wrist sprain)
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Musculoskeletal - other (finger sprain)
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Cervical spine ROM
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Musculoskletal - other (spondylosis)
|
1.1%
1/88 • Number of events 1 • 52 weeks
|
0.00%
0/81 • 52 weeks
|
|
Surgical and medical procedures
Tonsillectomy
|
0.00%
0/88 • 52 weeks
|
2.5%
2/81 • Number of events 2 • 52 weeks
|
|
Eye disorders
Ocular - other (eye redness, allergies)
|
0.00%
0/88 • 52 weeks
|
1.2%
1/81 • Number of events 1 • 52 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place