Trial Outcomes & Findings for Roll-over Study to Assess Safety of Lixivaptan in Participants With ADPKD Who Completed Study PA-ADPKD-303 (NCT NCT05208866)
NCT ID: NCT05208866
Last Updated: 2023-05-12
Results Overview
Number of participants who develop serum alanine aminotransferase (ALT) levels \>3 × the upper limit of normal (ULN) which are assessed by the independent Hepatic Events Review Committee (HERC) to be at least probably related to lixivaptan and resulted in discontinuation of lixivaptan treatment. The independent HERC, after reviewing demographic, medical and medication history, safety data and other relevant data of participants who develop liver abnormalities, will determine the probable causality for liver chemistry test abnormalities of concern and the relatedness to lixivaptan using the Drug-Induced Liver Injury Network probability criteria (Fontana et al., 2009). The normal range of ALT was defined as 0-55 U/L.
TERMINATED
PHASE3
1 participants
120 days (from Screening to the end of the Maintenance Treatment Period)
2023-05-12
Participant Flow
Participant milestones
| Measure |
All Enrolled Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Lixivaptan Re-titration Period
STARTED
|
1
|
|
Lixivaptan Re-titration Period
COMPLETED
|
1
|
|
Lixivaptan Re-titration Period
NOT COMPLETED
|
0
|
|
Maintenance Treatment Period
STARTED
|
1
|
|
Maintenance Treatment Period
COMPLETED
|
0
|
|
Maintenance Treatment Period
NOT COMPLETED
|
1
|
|
Follow-up Period
STARTED
|
1
|
|
Follow-up Period
COMPLETED
|
1
|
|
Follow-up Period
NOT COMPLETED
|
0
|
Reasons for withdrawal
| Measure |
All Enrolled Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Maintenance Treatment Period
Sponsor Termination of Study
|
1
|
Baseline Characteristics
Roll-over Study to Assess Safety of Lixivaptan in Participants With ADPKD Who Completed Study PA-ADPKD-303
Baseline characteristics by cohort
Baseline data not reported
PRIMARY outcome
Timeframe: 120 days (from Screening to the end of the Maintenance Treatment Period)Number of participants who develop serum alanine aminotransferase (ALT) levels \>3 × the upper limit of normal (ULN) which are assessed by the independent Hepatic Events Review Committee (HERC) to be at least probably related to lixivaptan and resulted in discontinuation of lixivaptan treatment. The independent HERC, after reviewing demographic, medical and medication history, safety data and other relevant data of participants who develop liver abnormalities, will determine the probable causality for liver chemistry test abnormalities of concern and the relatedness to lixivaptan using the Drug-Induced Liver Injury Network probability criteria (Fontana et al., 2009). The normal range of ALT was defined as 0-55 U/L.
Outcome measures
| Measure |
All Enrolled Participants
n=1 Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Number of Participants Who Develop Serum ALT Levels >3 × ULN During the Lixivaptan Re-titration or Maintenance Treatment Periods Assessed to be Related to Lixivaptan and Resulted in Discontinuation of Lixivaptan Treatment
|
0 Participants
|
SECONDARY outcome
Timeframe: 120 days (from Screening to the end of the Maintenance Treatment Period)Number of participants who develop serum ALT levels \>5 × ULN which are assessed by the independent HERC to be at least probably related to lixivaptan and resulted in discontinuation of lixivaptan treatment. The independent HERC, after reviewing demographic, medical and medication history, safety data and other relevant data of participants who develop liver abnormalities, will determine the probable causality for liver chemistry test abnormalities of concern and the relatedness to lixivaptan using the Drug-Induced Liver Injury Network probability criteria (Fontana et al., 2009). The normal range of ALT was defined as 0-55 U/L.
Outcome measures
| Measure |
All Enrolled Participants
n=1 Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Number of Participants Who Develop Serum ALT Levels >5 x ULN During the Lixivaptan Re-titration or Maintenance Treatment Periods Assessed to be Related to Lixivaptan and Resulted in Discontinuation of Lixivaptan Treatment
|
0 Participants
|
SECONDARY outcome
Timeframe: 120 days (from Screening to the end of the Maintenance Treatment Period)Number of participants who develop serum ALT levels \>3 × ULN that were assessed by the independent HERC to be at least probably related to lixivaptan and resulted in dose reduction of lixivaptan treatment. The independent HERC, after reviewing demographic, medical and medication history, safety data and other relevant data of participants who develop liver abnormalities, will determine the probable causality for liver chemistry test abnormalities of concern and the relatedness to lixivaptan using the Drug-Induced Liver Injury Network probability criteria (Fontana et al., 2009). The normal range of ALT was defined as 0-55 U/L.
Outcome measures
| Measure |
All Enrolled Participants
n=1 Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Number of Participants Who Develop Serum ALT Values >3 × ULN During the Lixivaptan Re-titration or Maintenance Treatment Periods Assessed to be Related to Lixivaptan and Resulted in Dose Reduction of Lixivaptan Treatment
|
0 Participants
|
SECONDARY outcome
Timeframe: 140 days (from Screening to the end of the Follow-up Period)Number of participants with TEAEs during the Lixivaptan Re-titration Period, the Maintenance Treatment Period, or the Follow-up Period.
Outcome measures
| Measure |
All Enrolled Participants
n=1 Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Lixivaptan Re-titration and Maintenance Treatment Periods
|
1 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Follow-up Period
|
0 Participants
|
SECONDARY outcome
Timeframe: 140 days (from Screening to the end of the Follow-up Period)Number of participants with clinical laboratory findings (non-hepatic clinical chemistry, hematology, and urinalysis) recorded during the Lixivaptan Re-titration Period, the Maintenance Treatment Period, or the Follow-up Period, and considered to be potentially clinically important.
Outcome measures
| Measure |
All Enrolled Participants
n=1 Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Number of Participants With Potentially Clinically Important Clinical Laboratory Findings
Lixivaptan Re-titration and Maintenance Treatment Periods
|
0 Participants
|
|
Number of Participants With Potentially Clinically Important Clinical Laboratory Findings
Follow-up Period
|
0 Participants
|
SECONDARY outcome
Timeframe: 140 days (from Screening to the end of the Follow-up Period)Number of participants with vital signs findings (heart rate, diastolic and systolic blood pressure, and weight) recorded during the Lixivaptan Re-titration Period, the Maintenance Treatment Period, or the Follow-up Period, and considered to be potentially clinically important.
Outcome measures
| Measure |
All Enrolled Participants
n=1 Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Number of Participants With Potentially Clinically Important Vital Signs Findings
Lixivaptan Re-titration and Maintenance Treatment Periods
|
0 Participants
|
|
Number of Participants With Potentially Clinically Important Vital Signs Findings
Follow-up Period
|
0 Participants
|
SECONDARY outcome
Timeframe: 140 days (from Screening to the end of the Follow-up Period)Number of participants with ECG findings recorded during the Lixivaptan Re-titration Period, the Maintenance Treatment Period, or the Follow-up Period, and considered to be potentially clinically important (defined as a QT interval corrected for heart rate according to Fridericia's formula \[QTcF\] ≥ 450 msec).
Outcome measures
| Measure |
All Enrolled Participants
n=1 Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Number of Participants With Potentially Clinically Important 12-lead Electrocardiogram (ECG) Findings
Lixivaptan Re-titration and Maintenance Treatment Periods
|
0 Participants
|
|
Number of Participants With Potentially Clinically Important 12-lead Electrocardiogram (ECG) Findings
Follow-up Period
|
0 Participants
|
SECONDARY outcome
Timeframe: 140 days (from Screening to the end of the Follow-up Period)Population: The annualized change in eGRF is provided for the only participant enrolled at the time of early termination of the study.
Baseline eGFR is defined as the mean of the 3 eGFR assessments obtained at Visits 25, 26 and 27 of study PA-ADPKD-303 (if any values are missing, the remaining values will be used to determine the baseline eGFR). The endpoint eGFR is defined as the mean of 3 eGFR assessments obtained during the Follow-up Period (if any values are missing, the remaining values will be used to determine the endpoint eGFR). The change in eGFR from Baseline to Final Assessment will be provided.
Outcome measures
| Measure |
All Enrolled Participants
n=1 Participants
All participants who were enrolled in this study were included in this group.
|
|---|---|
|
Annualized Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Final Assessment
|
14.6 mL/min/1.73m^2
Standard Deviation 0.0
|
Adverse Events
Titration and Maintenance Periods
Follow-up Period
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Titration and Maintenance Periods
n=1 participants at risk
All participants who completed the Lixivaptan Re-titration Period and entered the Maintenance Period
|
Follow-up Period
n=1 participants at risk
All participants who entered the Follow-up Period, regardless of whether they had completed the Maintenance Period
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
100.0%
1/1 • Number of events 1 • Adverse events were recorded from when participants had taken their first dose of lixivaptan in the Re-titration Period until the end of the study (maximum duration: 140 days)
|
0.00%
0/1 • Adverse events were recorded from when participants had taken their first dose of lixivaptan in the Re-titration Period until the end of the study (maximum duration: 140 days)
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
100.0%
1/1 • Number of events 1 • Adverse events were recorded from when participants had taken their first dose of lixivaptan in the Re-titration Period until the end of the study (maximum duration: 140 days)
|
0.00%
0/1 • Adverse events were recorded from when participants had taken their first dose of lixivaptan in the Re-titration Period until the end of the study (maximum duration: 140 days)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60