Trial Outcomes & Findings for A Study to Evaluate Setanaxib in Patients With Alport Syndrome (NCT NCT06274489)
NCT ID: NCT06274489
Last Updated: 2026-04-13
Results Overview
Count and percentage of patients experiencing SAEs
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
20 participants
Primary outcome timeframe
From informed consent up to 30 days post-final dose, up to 32 weeks
Results posted on
2026-04-13
Participant Flow
Overall, 34 participants were screened. 20 of them were randomized: 13 to the setanaxib group and 7 to the placebo group. 2 participants in the placebo group discontinued early from the study: * 1 participant in the placebo group did not start the study treatment. * 1 participant in the placebo group who received study drug discontinued early due to pregnancy.
Participant milestones
| Measure |
Setanaxib
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
7
|
|
Overall Study
COMPLETED
|
13
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Setanaxib
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Overall Study
Pregnancy
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
A Study to Evaluate Setanaxib in Patients With Alport Syndrome
Baseline characteristics by cohort
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=7 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
1 Participants
n=386 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
1 Participants
n=386 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=193 Participants
|
1 Participants
n=193 Participants
|
2 Participants
n=386 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=193 Participants
|
4 Participants
n=193 Participants
|
14 Participants
n=386 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=193 Participants
|
2 Participants
n=193 Participants
|
2 Participants
n=386 Participants
|
|
Age, Continuous
|
29.7 years
STANDARD_DEVIATION 8.54 • n=193 Participants
|
29 years
STANDARD_DEVIATION 12.40 • n=193 Participants
|
29.5 years
STANDARD_DEVIATION 9.73 • n=386 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=193 Participants
|
3 Participants
n=193 Participants
|
11 Participants
n=386 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=193 Participants
|
4 Participants
n=193 Participants
|
9 Participants
n=386 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
1 Participants
n=386 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=193 Participants
|
7 Participants
n=193 Participants
|
19 Participants
n=386 Participants
|
PRIMARY outcome
Timeframe: From informed consent up to 30 days post-final dose, up to 32 weeksPopulation: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Count and percentage of patients experiencing SAEs
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Serious Adverse Events (SAEs)
|
1 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: From informed consent up to 30 days post-final dosePopulation: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Count and percentage of patients experiencing treatment-emergent AEISIs. CTCAE Grade ≥2 anemia will be considered an AESI. During the course of the study, additional AESIs may be identified by the Sponsor
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Treatment-emergent Adverse Events of Special Interest (AESIs)
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: At baseline and week 24Population: Full Analysis Set: All randomized patients.
The ratio of UPCR at 24 weeks compared to baseline
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=7 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
The Ratio of Urine Protein to Creatinine Ratio (UPCR) Analysed in 24-hour Urine Sample
|
1.06 Ratio of UPCR
Interval 0.87 to 1.3
|
1.25 Ratio of UPCR
Interval 0.93 to 1.68
|
SECONDARY outcome
Timeframe: At baseline and week 24Population: Full Analysis Set: All randomized patients.
The proportions of patients achieving a 25% reduction in UPCR at 24 weeks from baseline in the setanaxib group will be compared to placebo group
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=7 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With a 25% Reduction in UPCR Analysed in 24-hour Urine Sample
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: At baseline and Week 24.Population: Full Analysis Set: All randomized patients.
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=7 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
The Ratio of Estimated Glomerular Filtration Rate (eGFR) at 24 Weeks Compared to Baseline.
|
0.91 Ratio of eGFR
Interval 0.84 to 0.98
|
0.96 Ratio of eGFR
Interval 0.86 to 1.08
|
SECONDARY outcome
Timeframe: within 90 minutes prior to dose and 0-, 1-, 2-, 3-, 4- and 6-hours post-dose at week 2; and prior to dose at weeks 12 and 24Population: PK Analysis Set: All randomized patients who took at least 1 dose of the study drug and had sufficient setanaxib plasma concentration data to calculate at least 1 PK parameter.
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Pre-dose and Post-dose Plasma Concentrations of Setanaxib and GKT138184 Measured as the Area Under the Concentration-time Curve Over 24 Hours at Steady State (AUC0-24-ss)
Setanaxib
|
204 µg*h/mL
Geometric Coefficient of Variation 33
|
—
|
|
Pre-dose and Post-dose Plasma Concentrations of Setanaxib and GKT138184 Measured as the Area Under the Concentration-time Curve Over 24 Hours at Steady State (AUC0-24-ss)
GKT138184
|
53.9 µg*h/mL
Geometric Coefficient of Variation 58
|
—
|
SECONDARY outcome
Timeframe: within 90 minutes prior to dose and 0-, 1-, 2-, 3-, 4- and 6-hours post-dose at week 2; and prior to dose at weeks 12 and 24Population: PK Analysis Set: All randomized patients who took at least 1 dose of the study drug and had sufficient setanaxib plasma concentration data to calculate at least 1 PK parameter.
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Pre-dose and Post-dose Plasma Concentrations of Setanaxib and GKT138184 Measured as the Minimum Plasma Concentration at Steady State (Cmin-ss)
Setanaxib
|
2.27 µg/mL
Geometric Coefficient of Variation 70
|
—
|
|
Pre-dose and Post-dose Plasma Concentrations of Setanaxib and GKT138184 Measured as the Minimum Plasma Concentration at Steady State (Cmin-ss)
GKT138184
|
1.20 µg/mL
Geometric Coefficient of Variation 81
|
—
|
SECONDARY outcome
Timeframe: within 90 minutes prior to dose and 0-, 1-, 2-, 3-, 4- and 6-hours post-dose at week 2; and prior to dose at weeks 12 and 24Population: PK Analysis Set: All randomized patients who took at least 1 dose of the study drug and had sufficient setanaxib plasma concentration data to calculate at least 1 PK parameter.
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Pre-dose and Post-dose Plasma Concentrations of Setanaxib and GKT138184 Measured as the Maximum Plasma Concentration at Steady State (Cmax-ss)
Setanaxib
|
24.9 µg/mL
Geometric Coefficient of Variation 45
|
—
|
|
Pre-dose and Post-dose Plasma Concentrations of Setanaxib and GKT138184 Measured as the Maximum Plasma Concentration at Steady State (Cmax-ss)
GKT138184
|
3.72 µg/mL
Geometric Coefficient of Variation 59
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From baseline by visit up to week 28 (study visit 9, Follow-Up)Population: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Clinically significant changes in heart rate from baseline by visit. Heart rate will be measured after resting in the supine position for at least 5 minutes and will be measured 3 times with each measurement separated by at least 1 minute (the lowest value will be recorded)
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Clinically Significant Changes in Heart Rate
Clinically significant change from baseline to Week 28
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Heart Rate
Clinically significant change from baseline to Week 2
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Heart Rate
Clinically significant change from baseline to Week 6
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Heart Rate
Clinically significant change from baseline to Week 12
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Heart Rate
Clinically significant change from baseline to Week 18
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Heart Rate
Clinically significant change from baseline to Week 24 - pre-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Heart Rate
Clinically significant change from baseline to Week 24 - 3 hours post-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Heart Rate
Clinically significant change from baseline to Week 24 - 6 hours post-dose
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From baseline by visit up to week 28 (study visit 9, Follow-Up)Population: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Clinically significant changes in blood pressure from baseline by visit. Systolic and diastolic blood pressure will be measured after resting in the supine position for at least 5 minutes and will be measured 3 times with each measurement separated by at least 1 minute (the lowest value will be recorded)
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Systolic blood pressure change from baseline to Week 12
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Systolic blood pressure change from baseline to Week 2
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Systolic blood pressure change from baseline to Week 6
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Systolic blood pressure change from baseline to Week 18
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Systolic blood pressure change from baseline to Week 24 - pre-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Systolic blood pressure change from baseline to Week 24 - 3 hours post-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Systolic blood pressure change from baseline to Week 24 - 6 hours post-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Systolic blood pressure change from baseline to Week 28
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Diastolic blood pressure change from baseline to Week 2
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Diastolic blood pressure change from baseline to Week 6
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Diastolic blood pressure change from baseline to Week 12
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Diastolic blood pressure change from baseline to Week 18
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Diastolic blood pressure change from baseline to Week 24 - pre-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Diastolic blood pressure change from baseline to Week 24 - 3 hours post-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Diastolic blood pressure change from baseline to Week 24 - 6 hours post-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes Blood Pressure
Diastolic blood pressure change from baseline to Week 28
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From baseline by visit up to week 28 (study visit 9, Follow-Up)Population: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Clinically significant changes in ECGs from baseline by visit. ECGs should be collected in triplicate, 1 to 3 minutes apart, and read locally
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Clinically significant changes from baseline to Week 2
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Clinically significant changes from baseline to Week 6
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Clinically significant changes from baseline to Week 12
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Clinically significant changes from baseline to Week 18
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Clinically significant changes from baseline to Week 24 - pre-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Clinically significant changes from baseline to Week 24 - 3 hours post-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Clinically significant changes from baseline to Week 24 - 6 hours post-dose
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Clinically significant changes from baseline to Week 28
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From baseline by visit up to week 28 (study visit 9, Follow-Up)Population: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Clinically significant changes in physical examinations from baseline by visit. Physical examinations will include general appearance, skin, head, ears, eyes, nose, throat, neck, lungs, heart, abdomen, musculoskeletal, extremities, and neurological systems
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Clinically Significant Changes in Physical Examination
Clinically significant changes from baseline to Week 2
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Physical Examination
Clinically significant changes from baseline to Week 6
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Physical Examination
Clinically significant changes from baseline to Week 12
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Physical Examination
Clinically significant changes from baseline to Week 18
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Physical Examination
Clinically significant changes from baseline to Week 24
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Physical Examination
Clinically significant changes from baseline to Week 28
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From baseline by visit up to week 28 (study visit 9, Follow-Up)Population: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Clinically significant changes in hematology, serum chemistry, urinalysis, and thyroid function from baseline by visit
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Clinically Significant Changes in Hematology, Serum Chemistry, Urinalysis, and Thyroid Function
|
4 Participants
|
1 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From baseline by visit up to week 28 (study visit 9, Follow-Up)Population: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Clinically significant changes in hearing audiometric testing from baseline by visit. Pure tone audiometry (PTA) bone conduction thresholds will be conducted at frequencies 500, 1000, 2000, and 4000 Hz.
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Clinically Significant Changes in Hearing Audiometric Testing (Bone-conduction)
Clinically significant changes from baseline to Week 24
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Hearing Audiometric Testing (Bone-conduction)
Clinically significant changes from baseline to Week 28
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From baseline by visit up to week 28 (study visit 9, Follow-Up)Population: Safety Analysis Set: All randomized patients who took at least 1 dose of study drug.
Clinically significant changes in hearing audiometric testing from baseline by visit. Pure tone audiometry (PTA) air conduction thresholds will be conducted at frequencies 250, 500, 1000, 2000, 4000, and 8000 Hz.
Outcome measures
| Measure |
Setanaxib
n=13 Participants
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Placebo
n=6 Participants
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Percentage of Patients With Clinically Significant Changes in Hearing Audiometric Testing (Air-conduction)
Clinically significant changes from baseline to Week 24
|
0 Participants
|
0 Participants
|
|
Percentage of Patients With Clinically Significant Changes in Hearing Audiometric Testing (Air-conduction)
Clinically significant changes from baseline to Week 28
|
0 Participants
|
0 Participants
|
Adverse Events
Setanaxib
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
Setanaxib Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Setanaxib
n=13 participants at risk
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Setanaxib Placebo
n=6 participants at risk
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Hepatobiliary disorders
Cholecystitis acute
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
Other adverse events
| Measure |
Setanaxib
n=13 participants at risk
Patients will receive the following setanaxib doses according to age at the time of consent/assent:
* For patients aged 12 to 17 years 1200 mg/day: 2 tablets in the morning (800 mg) and 1 tablet in the evening (400 mg)
* For patients aged ≥18 years 1600 mg/day: 2 tablets in the morning (800 mg) and 2 tablets in the evening (800 mg)
Setanaxib: Setanaxib (formerly GKT137831) is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
|
Setanaxib Placebo
n=6 participants at risk
Matching placebo will be provided.
Placebo: Matching film-coated placebo tablets, containing only excipients
|
|---|---|---|
|
Nervous system disorders
Paraesthesia
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Gastrointestinal disorders
Diarrhoea
|
15.4%
2/13 • Number of events 2 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Gastrointestinal disorders
Vomiting
|
7.7%
1/13 • Number of events 2 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Gastrointestinal disorders
Dyspepsia
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.4%
2/13 • Number of events 2 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Renal and urinary disorders
Albuminuria
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Renal and urinary disorders
Proteinuria
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Reproductive system and breast disorders
Dysmenorrhea
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Reproductive system and breast disorders
Intermenstrual bleeding
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
General disorders
Fatigue
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/13 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/13 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Musculoskeletal and connective tissue disorders
Spinal Pain
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Psychiatric disorders
Insomnia
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Vascular disorders
Hypotension
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Infections and infestations
Viral infection
|
0.00%
0/13 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Investigations
Blood creatine phosphokinase increased
|
15.4%
2/13 • Number of events 3 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Investigations
Blood thyroid stimulating hormone decreased
|
0.00%
0/13 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Investigations
Blood thyroid stimulating hormone increased
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Nervous system disorders
Dizziness
|
15.4%
2/13 • Number of events 2 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Nervous system disorders
Headache
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 3 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Infections and infestations
Respiratory tract infection
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Infections and infestations
Cystitis
|
7.7%
1/13 • Number of events 2 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/13 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
16.7%
1/6 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Infections and infestations
Rhinitis
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Infections and infestations
Upper respiratory tract infection
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
|
Infections and infestations
Urinary tract infection
|
7.7%
1/13 • Number of events 1 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
0.00%
0/6 • From the time of informed consent until the end of the Follow-up Period (Study Visit 9), up to 32 weeks.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Trial Site and / or the Principal Investigator will submit material for public dissemination to the Sponsor for review at least sixty (60) days prior to submission for publication, public dissemination, or review by a publication committee. During the period for review of a proposed publication Sponsor shall be entitled to make a reasoned request to the Trial Site that publication be delayed for a period of up to six (6) months from the date of first submission to the Sponsor.
- Publication restrictions are in place
Restriction type: OTHER