A Phase 3 interventional study of Lixivaptan and Placebo in Autosomal Dominant Polycystic Kidney and ADPKD, sponsored by Palladio Biosciences. Terminated at 43 sites in 8 countries. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2023-02-06.
Sponsored by Palladio Biosciences · Phase 3, Interventional, and Treatment
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Among 85 studies in Polycystic Kidney, Autosomal Dominant with a site in United States — 14 open to participants now, 25 under way, 1 of them phase 3. Polycystic Kidney, Autosomal Dominant · United States.
Among the 85 studies in Polycystic Kidney, Autosomal Dominant with a site in United States, this one is the 55th largest by enrollment of the 63 interventional ones that give a count, is one of 5 that started in 2021, is one of 13 in phase 3, and was the 52nd registered.
185 studies on the registry are indexed under Polycystic Kidney, Autosomal Dominant; 42 are open to participants now.
Browse Polycystic Kidney, Autosomal Dominant studies →14 of the 85 studies in Polycystic Kidney, Autosomal Dominant with a site in United States are open now. Recruiting first, the newest first:
Vertex Pharmaceuticals Incorporated · Sites in United States
Ages 19–55 · Men only · Healthy volunteers
Kenneth Hallows · Sites in United States
Ages 18–60
Mayo Clinic · Sites in United States
Ages 18–70
Richard Fatica · Sites in United States
Ages 18+
AstraZeneca · Sites in United States, China, United Kingdom
Ages 18–65
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 30 Polycystic Kidney, Autosomal Dominant studies recruiting now, 8 (27%) set no upper age limit and 19 (63%) stop at 75 or younger; 7 take children under 18; 9 take healthy volunteers.
This study: 18 Years to 60 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
85 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 12 · Phase 2 25 · Phase 3 18 · Phase 4 2
First in 1998; this study’s year is marked. 5 started in 2021.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 12 is below the median of 51 across 138 interventional studies indexed under Polycystic Kidney, Autosomal Dominant.
Median enrollment 48 across the 63 interventional studies, 90 across the 22 observational. Of the 59 that completed or were terminated, 34 (58%) have results posted.
This tab as a report to print or save as PDF → With an account.
34 of 85 have an industry lead sponsor. The 10 most frequent:
Palladio Biosciences is the lead sponsor of 5 studies on the registry; none are open to participants now.
Palladio Biosciences has 4 studies filed under Polycystic Kidney, Autosomal Dominant — fewer than ten, so there is no comparison to draw.
16 of the studies in Polycystic Kidney, Autosomal Dominant with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05193981Mayo ClinicA Study to Evaluate Homocysteine Metabolism and Endothelial Function in ADPKD | 2026-12 |
| NCT05288998Mayo ClinicIntrarenal Microvasculature in ADPKD | 2026-12 |
| NCT06786260Loma Linda UniversityDi-PKD: A Pilot Trial of Dietary Intervention in Patients With Autosomal Dominant Polycystic Kidney Disease | 2026-12 |
| NCT04344769Mayo ClinicCharacterization of the Nrf2 Response in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) | 2026-12 |
| NCT04630613Mayo ClinicNOX4 and Related Biomarkers in ADPKD | 2026-12 |
Add all 16 to your calendar (.ics) → With an account.
Among the 85 studies in Polycystic Kidney, Autosomal Dominant with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This is a Phase 3 trial consisting of a 2-arm, double-blind, placebo-controlled, randomized phase (Part 1) followed by a single-arm open-label phase (Part 2) to demonstrate the efficacy and safety of lixivaptan in participants with autosomal dominant polycystic kidney disease (ADPKD). Part 1 of the trial is designed to demonstrate the efficacy of lixivaptan in slowing the decline in kidney function as measured by the difference in estimated glomerular filtration rate (eGFR) between the lixivaptan-treated and placebo-treated participants. Part 2 of the study is designed to provide confirmation of the durability of this effect. Additionally, both parts of the study will contribute to understanding the safety of lixivaptan, particularly any effects on liver chemistry tests.
Part 1: Approximately 2250 participants with ADPKD will be screened in order to qualify the 1350 individuals who will proceed through the Placebo Run-in Period, the Lixivaptan Titration Period, and then be randomized to receive lixivaptan or placebo in the Double-blind, Randomized Treatment Period. Throughout Part 1 the study drug will look identical regardless of whether it is placebo or lixivaptan.
After meeting entry criteria during screening, participants will enter a 1-week single-blind, Placebo Run-in Period to obtain select baseline measurements. This will be followed by a 5- to 6-week single-blind dose titration period during which lixivaptan administered twice daily (BID) will be titrated to the highest tolerated dose (the Lixivaptan Titration Period). The starting dose (Level 1) will be 50 mg BID) and this will be increased weekly through Levels 2 (100 mg BID), 3 (150mg BID), and 4 (200 mg BID). The minimum dose to enter the next period, the Double-blind, Randomized Treatment Period, is Level 2 (100 mg BID). Those participants successfully titrated and tolerating the drug will then be randomized (each participant has a 2/3 chance of receiving lixivaptan and a 1/3 chance of receiving placebo) to either continue receiving lixivaptan or switch immediately to matching placebo in a double-blind manner. All dosing throughout the study will be 4 capsules BID. Depending on the study period, treatment arm to which the participant is randomized, and current dose level, the set of 4 capsules may be all placebo, all active or a combination of active and placebo. Double-blind treatment will continue for 52 weeks after which study drug will be held, and final assessments obtained off-treatment over 3 visits starting on the 8th day after the last dose of double-blind study drug through the 28th day after the last dose of study drug.
For Part 1 summaries after randomization, the analysis results will be presented by 2 treatment groups: lixivaptan and placebo.
Part 2: All participants entering Part 1, who have not discontinued due to an adverse event or withdrawn consent, will continue into Part 2 of the study, and will proceed through the Lixivaptan Re-titration Period and a Maintenance Treatment Period.
The Lixivaptan Re-titration Period will be a 2- to 4-week period during which dosing with lixivaptan will start at Level 1 (50 mg BID) for all participants and will be increased weekly until the dose level of lixivaptan, or the inferred lixivaptan dose level for participants randomized to placebo treatment, taken at the end of the Double-blind, Randomized Treatment Period in Part 1 is achieved. Lixivaptan treatment will continue for 52 weeks during the Maintenance Treatment Period after which study drug will be held, and final assessments obtained off-treatment over 3 visits starting on the 8th day after the last dose of lixivaptan through the 28th day after the last dose of lixivaptan.
Since all participants in Part 2 are treated with lixivaptan, the summaries for Part 2 will be presented in the following treatment cohorts and overall (treatment cohorts combined):
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Lixivaptan capsules, 100-200 mg twice a day (BID)
Drug: Lixivaptan
Matching placebo capsules BID
Drug: Placebo
Oral vasopressin V2 receptor antagonist
Matching placebo
Annualized Change in Estimated Glomerular Filtration Rate (eGFR) - Part 1
The annualized change in eGFR will be calculated from the Chronic Kidney Disease Epidemiology Collaboration eGFR creatinine equation refit without the race variable (CKD-EPIcr_R) equation for serum creatinine from baseline (mean of 3 eGFR determinations obtained during Screening and Placebo Run-in Periods \[Visits 1a/1b (if required), Visit 2, and Visit 3\]) to final assessment (mean of 3 eGFR determinations obtained during Follow-up Period I or, for participants who discontinue treatment prior to Visit 22, during the Follow-up Period 8 to 28 days following study drug treatment discontinuation).
Time frame: Baseline to the end of Follow-up Period I (up to 71 weeks)
Annualized Change in Estimated Glomerular Filtration Rate (eGFR) - Part 2
The annualized change in eGFR will be calculated from the CKD-EPIcr_R equation for serum creatinine from baseline (mean of 3 eGFR determinations obtained during obtained during Follow-up Period I) to final assessment (mean of 3 eGFR determinations obtained during Follow-up Period II or, for participants who discontinue treatment prior to Visit 43, during the Follow-up Period 8 to 28 days following study drug treatment discontinuation).
Time frame: Baseline to the end of Follow-up Period II (up to 64 weeks)
Number of Participants With Serum Alanine Aminotransferase (ALT) Levels >3 × the Upper Limit of Normal (ULN) - Part 1
Number of participants randomized to lixivaptan with serum ALT levels \>3 × the ULN compared to those randomized to placebo. The normal range of ALT was defined as 0-33 or 0-44 U/L, depending on the lab used.
Time frame: From the end of the Single-blind Lixivaptan Titration Period to the end of Follow-up Period I (maximum of 102 days)
Number of Participants With Serum ALT Levels >3 × the ULN - Part 2
Number of participants randomized to lixivaptan with serum ALT levels \>3 × ULN compared to those randomized to placebo. The normal range of ALT was defined as 0-33 or 0-44 U/L, depending on the lab used.
Time frame: Duration of the Lixivaptan Re-titration Period and the Maintenance Treatment Period (56 weeks) and Follow-up Period II (4 weeks)
eGFR Slope - Part 1
Annualized rate of change (slope) in eGFR for serum creatinine, based on all eGFR determinations from baseline to all visits with on-treatment eGFR values during the Double-blind, Randomized Treatment Period. The baseline value will be calculated as the mean of 3 eGFR determinations obtained during Screening and Placebo Run-in Periods (Visits 1a/1b \[if required\], Visit 2, and Visit 3).
Time frame: Baseline to the end of Double-blind Randomized Treatment Period (up to 67 weeks), with changes from baseline calculated every 4 weeks during the Double-blind Randomized Treatment Period
eGFR Slope - Part 2
Annualized rate of change (slope) in eGFR for serum creatinine, based on all eGFR determinations from baseline to all visits with on-treatment eGFR values during the Maintenance Treatment Period. The baseline value will be calculated as the mean of 3 eGFR determinations obtained during obtained during Follow-up Period I.
Time frame: Baseline to the end of the Maintenance Treatment Period (up to 60 weeks), with changes from baseline calculated every 4 weeks during the Maintenance Treatment Period
Height-adjusted Total Kidney Volume (htTKV) - Part 1
Annualized percent change in htTKV, determined by magnetic resonance imaging (MRI), from baseline (obtained at Screening \[Visit 1a\]) to the end of Follow-up Period I.
Time frame: Baseline to the end of Follow-up Period I (up to 71 weeks)
Height-adjusted Total Kidney Volume (htTKV) - Part 2
Annualized percent change in htTKV, determined by MRI from baseline (obtained at Visit 25/Week 56) to the end of Follow-up Period II.
Time frame: Baseline to the end of Follow-up Period II (up to 60 weeks)
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Lixivaptan Titration Period in Part 1
Number of participants with TEAEs during the Lixivaptan Titration Period.
Time frame: Up to 6 weeks
Number of Participants With TEAEs After Randomization in Part 1
Number of participants with TEAEs by treatment group after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I)
Time frame: From Randomization to study completion (up to 102 days)
Number of Participants With TEAEs in Part 2
Number of participants with TEAEs by treatment cohort during Part 2 (Lixivaptan Re-titration Period, Maintenance Treatment Period, and Follow-up Period II).
Time frame: Up to 60 weeks
Number of Participants With Potentially Clinically Important Clinical Laboratory Findings During the Lixivaptan Titration Period in Part 1
Number of participants with clinical laboratory findings (clinical chemistry, hematology and urinalysis) recorded during the Lixivaptan Titration Period and considered to be potentially clinically important.
Time frame: Up to 6 weeks
Number of Participants With Potentially Clinically Important Clinical Laboratory Findings After Randomization in Part 1
Number of participants with clinical laboratory findings (clinical chemistry, hematology and urinalysis) recorded after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I) and considered to be potentially clinically important, by treatment group.
Time frame: From Randomization to last clinical laboratory evaluation (up to 102 days)
Number of Participants With Potentially Clinically Important Clinical Laboratory Findings in Part 2
Number of participants with clinical laboratory findings (clinical chemistry, hematology and urinalysis) recorded during Part 2 (Lixivaptan Re-titration Period, Maintenance Treatment Period, and Follow-up Period II) and considered to be potentially clinically important, by treatment cohort.
Time frame: Up to 60 weeks
Number of Participants With Potentially Clinically Important Vital Signs Findings During the Lixivaptan Titration Period in Part 1
Number of participants with vital signs findings (heart rate, diastolic and systolic blood pressure, weight) recorded during the Lixivaptan Titration Period and considered to be potentially clinically important.
Time frame: Up to 6 weeks
Number of Participants With Potentially Clinically Important Vital Signs Findings After Randomization in Part 1
Number of participants with vital signs findings (heart rate, diastolic and systolic blood pressure, weight) recorded after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I) and considered to be potentially clinically important, by treatment group.
Time frame: From Randomization to last measurement of vital signs (up to 102 days)
Number of Participants With Potentially Clinically Important Vital Signs Findings in Part 2
Number of participants with vital signs findings (heart rate, diastolic and systolic blood pressure, weight) recorded during Part 2 (Lixivaptan Re-titration Period, Maintenance Treatment Period, and Follow-up Period II) and considered to be potentially clinically important, by treatment cohort.
Time frame: Up to 60 weeks
Number of Participants With Potentially Clinically Significant 12-lead Electrocardiogram (ECG) Findings During the Lixivaptan Titration Period in Part 1
Number of participants with ECG findings recorded during the Lixivaptan Titration 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).
Time frame: Up to 6 weeks
Number of Participants With Potentially Clinically Significant 12-lead ECG Findings After Randomization in Part 1
Number of participants with ECG findings recorded after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I) and considered to be potentially clinically important (defined as a QTcF ≥ 450 msec), by treatment group.
Time frame: From Randomization to last ECG (up to 102 days)
Number of Participants With Potentially Clinically Significant 12-lead ECG Findings in Part 2
Number of participants with ECG findings recorded during Part 2 (Lixivaptan Re-titration Period, Maintenance Treatment Period, and Follow-up Period II) and considered to be potentially clinically important (defined as a QTcF ≥ 450 msec), by treatment cohort.
Time frame: Up to 60 weeks
| Milestone | All Subjects Pre-randomization | Lixivaptan | Placebo |
|---|---|---|---|
| Started | 12 | 0 | 0 |
| Treated in placebo run-in | 12 | 0 | 0 |
| Treated in lixivaptan titration period | 8 | 0 | 0 |
| Completed | 5 | 0 | 0 |
| Not completed | 7 | 0 | 0 |
| Milestone | All Subjects Pre-randomization | Lixivaptan | Placebo |
|---|---|---|---|
| Started | 0 | 4 | 1 |
| Randomized | 0 | 4 | 1 |
| Completed | 0 | 4 | 1 |
| Not completed | 0 | 0 | 0 |
| Milestone | All Subjects Pre-randomization | Lixivaptan | Placebo |
|---|---|---|---|
| Started | 0 | 3 | 1 |
| Completed | 0 | 3 | 1 |
| Not completed | 0 | 0 | 0 |
| Milestone | All Subjects Pre-randomization | Lixivaptan | Placebo |
|---|---|---|---|
| Started | 0 | 0 | 0 |
| Completed | 0 | 0 | 0 |
| Not completed | 0 | 0 | 0 |
| Milestone | All Subjects Pre-randomization | Lixivaptan | Placebo |
|---|---|---|---|
| Started | 0 | 0 | 0 |
| Completed | 0 | 0 | 0 |
| Not completed | 0 | 0 | 0 |
| Milestone | All Subjects Pre-randomization | Lixivaptan | Placebo |
|---|---|---|---|
| Started | 0 | 0 | 0 |
| Completed | 0 | 0 | 0 |
| Not completed | 0 | 0 | 0 |
The annualized change in eGFR will be calculated from the Chronic Kidney Disease Epidemiology Collaboration eGFR creatinine equation refit without the race variable (CKD-EPIcr_R) equation for serum creatinine from baseline (mean of 3 eGFR determinations obtained during Screening and Placebo Run-in Periods \[Visits 1a/1b (if required), Visit 2, and Visit 3\]) to final assessment (mean of 3 eGFR determinations obtained during Follow-up Period I or, for participants who discontinue treatment prior to Visit 22, during the Follow-up Period 8 to 28 days following study drug treatment discontinuation).
No measurements were reported for this outcome.
The annualized change in eGFR will be calculated from the CKD-EPIcr_R equation for serum creatinine from baseline (mean of 3 eGFR determinations obtained during obtained during Follow-up Period I) to final assessment (mean of 3 eGFR determinations obtained during Follow-up Period II or, for participants who discontinue treatment prior to Visit 43, during the Follow-up Period 8 to 28 days following study drug treatment discontinuation).
No measurements were reported for this outcome.
Number of participants randomized to lixivaptan with serum ALT levels \>3 × the ULN compared to those randomized to placebo. The normal range of ALT was defined as 0-33 or 0-44 U/L, depending on the lab used.
| Participants | Lixivaptan | Placebo |
|---|---|---|
| Number of Participants With Serum Alanine Aminotransferase (ALT) Levels >3 × the Upper Limit of Normal (ULN) - Part 1 | 0 | 1 |
Number of participants randomized to lixivaptan with serum ALT levels \>3 × ULN compared to those randomized to placebo. The normal range of ALT was defined as 0-33 or 0-44 U/L, depending on the lab used.
No measurements were reported for this outcome.
Annualized rate of change (slope) in eGFR for serum creatinine, based on all eGFR determinations from baseline to all visits with on-treatment eGFR values during the Double-blind, Randomized Treatment Period. The baseline value will be calculated as the mean of 3 eGFR determinations obtained during Screening and Placebo Run-in Periods (Visits 1a/1b \[if required\], Visit 2, and Visit 3).
No measurements were reported for this outcome.
Annualized rate of change (slope) in eGFR for serum creatinine, based on all eGFR determinations from baseline to all visits with on-treatment eGFR values during the Maintenance Treatment Period. The baseline value will be calculated as the mean of 3 eGFR determinations obtained during obtained during Follow-up Period I.
No measurements were reported for this outcome.
Annualized percent change in htTKV, determined by magnetic resonance imaging (MRI), from baseline (obtained at Screening \[Visit 1a\]) to the end of Follow-up Period I.
No measurements were reported for this outcome.
Annualized percent change in htTKV, determined by MRI from baseline (obtained at Visit 25/Week 56) to the end of Follow-up Period II.
No measurements were reported for this outcome.
Number of participants with TEAEs during the Lixivaptan Titration Period.
| Participants | Lixivaptan Titration Period Participants |
|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Lixivaptan Titration Period in Part 1 | 3 |
Number of participants with TEAEs by treatment group after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I)
| Participants | Lixivaptan | Placebo |
|---|---|---|
| Number of Participants With TEAEs After Randomization in Part 1 | 1 | 1 |
Number of participants with TEAEs by treatment cohort during Part 2 (Lixivaptan Re-titration Period, Maintenance Treatment Period, and Follow-up Period II).
No measurements were reported for this outcome.
Number of participants with clinical laboratory findings (clinical chemistry, hematology and urinalysis) recorded during the Lixivaptan Titration Period and considered to be potentially clinically important.
| Participants | Lixivaptan Titration Period Participants |
|---|---|
| Number of Participants With Potentially Clinically Important Clinical Laboratory Findings During the Lixivaptan Titration Period in Part 1 | 0 |
Number of participants with clinical laboratory findings (clinical chemistry, hematology and urinalysis) recorded after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I) and considered to be potentially clinically important, by treatment group.
| Participants | Lixivaptan | Placebo |
|---|---|---|
| Number of Participants With Potentially Clinically Important Clinical Laboratory Findings After Randomization in Part 1 | 0 | 0 |
Number of participants with clinical laboratory findings (clinical chemistry, hematology and urinalysis) recorded during Part 2 (Lixivaptan Re-titration Period, Maintenance Treatment Period, and Follow-up Period II) and considered to be potentially clinically important, by treatment cohort.
No measurements were reported for this outcome.
Number of participants with vital signs findings (heart rate, diastolic and systolic blood pressure, weight) recorded during the Lixivaptan Titration Period and considered to be potentially clinically important.
| Participants | Lixivaptan Titration Period Participants |
|---|---|
| Number of Participants With Potentially Clinically Important Vital Signs Findings During the Lixivaptan Titration Period in Part 1 | 0 |
Number of participants with vital signs findings (heart rate, diastolic and systolic blood pressure, weight) recorded after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I) and considered to be potentially clinically important, by treatment group.
| Participants | Lixivaptan | Placebo |
|---|---|---|
| Number of Participants With Potentially Clinically Important Vital Signs Findings After Randomization in Part 1 | 0 | 0 |
Number of participants with vital signs findings (heart rate, diastolic and systolic blood pressure, weight) recorded during Part 2 (Lixivaptan Re-titration Period, Maintenance Treatment Period, and Follow-up Period II) and considered to be potentially clinically important, by treatment cohort.
No measurements were reported for this outcome.
Number of participants with ECG findings recorded during the Lixivaptan Titration 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).
| Participants | Lixivaptan Titration Period Participants |
|---|---|
| Number of Participants With Potentially Clinically Significant 12-lead Electrocardiogram (ECG) Findings During the Lixivaptan Titration Period in Part 1 | 0 |
Number of participants with ECG findings recorded after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I) and considered to be potentially clinically important (defined as a QTcF ≥ 450 msec), by treatment group.
| Participants | Lixivaptan | Placebo |
|---|---|---|
| Number of Participants With Potentially Clinically Significant 12-lead ECG Findings After Randomization in Part 1 | 0 | 0 |
Number of participants with ECG findings recorded during Part 2 (Lixivaptan Re-titration Period, Maintenance Treatment Period, and Follow-up Period II) and considered to be potentially clinically important (defined as a QTcF ≥ 450 msec), by treatment cohort.
No measurements were reported for this outcome.
Collected over Adverse events were recorded from when participants had taken their first dose of lixivaptan in the Lixivaptan Titration Period until the end of the study (maximum duration: 123 days). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Lixivaptan Titration Period | 0/8 (0%) | 0/8 (0%) | 3/8 (37.5%) |
| Lixivaptan (Randomized Treatment Period) | 0/4 (0%) | 0/4 (0%) | 1/4 (25%) |
| Placebo (Randomized Treatment Period) | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Event | Lixivaptan Titration Period | Lixivaptan (Randomized Treatment Period) | Placebo (Randomized Treatment Period) |
|---|---|---|---|
| Transaminases increasedInvestigations | 0/8 | 0/4 | 1/1 |
| HypertensionVascular disorders | 2/8 | 0/4 | 0/1 |
| Dry eyeEye disorders | 0/8 | 1/4 | 0/1 |
| Acute kidney injuryRenal and urinary disorders | 0/8 | 1/4 | 0/1 |
| HypotensionVascular disorders | 0/8 | 1/4 | 0/1 |
| Abdominal distensionGastrointestinal disorders | 1/8 | 0/4 | 0/1 |
| DiarrhoeaGastrointestinal disorders | 1/8 | 0/4 | 0/1 |
| NasopharyngitisInfections and infestations | 1/8 | 0/4 | 0/1 |
| Influenza A virus test positiveInvestigations | 1/8 | 0/4 | 0/1 |
| PolydipsiaMetabolism and nutrition disorders | 1/8 | 0/4 | 0/1 |
| Age, Continuous(Years) | All Enrolled Participants |
|---|---|
| Mean | 44.9 ± 8.40 |
| Sex: Female, Male(Participants) | All Enrolled Participants |
|---|---|
| Female | 5 |
| Male | 7 |
| Ethnicity (NIH/OMB)(Participants) | All Enrolled Participants |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 11 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | All Enrolled Participants |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 2 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 9 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | All Enrolled Participants |
|---|---|
| United States | 10 |
| Bulgaria | 2 |
| Body Mass Index(kg/m^2) | All Enrolled Participants |
|---|---|
| Mean | 28.47 ± 6.795 |
| Family History of Autosomal Dominant Polycystic Kidney Disease(Participants) | All Enrolled Participants |
|---|---|
| Yes | 10 |
| No | 2 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Oct 2021 to primary completion in Aug 2022, 9 months.
Results were first posted in Feb 2023, 6 months after primary completion.
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Studies of the drug tested here: Lixivaptan
Polycystic Kidney, Autosomal Dominant →
Palladio Biosciences