Roll-over Study to Assess Safety of Lixivaptan in Participants With ADPKD Who Completed Study PA-ADPKD-303

Palladio Biosciences

Status and phase

Terminated

Phase 3

Conditions

Polycystic Kidney Disease, Adult

Treatments

Drug: Lixivaptan

Study type

Interventional

Funder types

Industry

Identifiers

NCT05208866

PA-ADPKD-304

Details and patient eligibility

About

This is a Phase 3, open-label, roll-over study to demonstrate the continued hepatic and non-hepatic safety and renal efficacy of lixivaptan in participants with ADPKD who previously experienced abnormal liver chemistry test results while treated with tolvaptan, were permanently discontinued from the drug for that reason, and subsequently completed study PA-ADPKD-303, the open-label lead-in study with lixivaptan.

Full description

This is a Phase 3, open-label, roll-over study to demonstrate the continued hepatic and non-hepatic safety and renal efficacy of lixivaptan in participants with ADPKD who previously experienced abnormal liver chemistry test results while treated with tolvaptan that resulted in permanent discontinuation of tolvaptan for that reason, and subsequently completed study PA-ADPKD-303, the open-label lead-in study with lixivaptan.

Assessments completed during the final 4 visits of PA-ADPKD-303, the lead-in study, will serve as the screening and baseline assessments for this roll-over study. Evaluation of eligibility will be completed at Visit 1 of this study, following signing of informed consent. Participants satisfying all study entry criteria at Visit 1 will be considered enrolled following completion of all Visit 1 study procedures and will be dispensed lixivaptan treatment to start the Lixivaptan Re-titration Period (1 to 2 weeks). During the Lixivaptan Re-titration Period, participants will have their dose of lixivaptan re-established based on the dose they were receiving at the completion of the lead-in study. Participants will continue on lixivaptan treatment for up to 104 weeks during the Maintenance Treatment Period and will be assessed at a study visit every 12 weeks. In between the quarterly study visits, participants will be required to have blood drawn for liver chemistry determinations every 4 weeks. At the end of 104 weeks, lixivaptan treatment will be stopped, and participants will enter a 4-week Follow-up Period during which final assessments of safety and efficacy will be obtained over 3 visits during a 28-day period.

Enrollment

1 patient

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Male or female participants with ADPKD who completed study PA-ADPKD-303
Continued control of hypertension without the use of a diuretic
Continued adherence to prohibitions on concomitant medications stated in the study PA-ADPKD-303 protocol
Willing to practice acceptable methods of birth control (both males who have partners of child-bearing potential and females of childbearing potential).
Able to provide informed consent.

Exclusion criteria

Any contraindication to continued treatment with lixivaptan
Clinically significant incontinence, overactive bladder, or urinary retention (e.g., benign prostatic hyperplasia)
New York Heart Association Functional Class 3 or 4 heart failure or other significant cardiac or electrocardiogram (ECG) findings that could pose a safety risk to the participant
Hypovolemia on physical examination at Screening
The following laboratory results based on serum drawn at Visit 24 of PA-ADPKD-303:
- Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values >1.5 × ULN
- Total bilirubin values >1.5 × ULN
eGFR <20 mL/min/1.73 m^2 based on laboratory results from Visit 26 of PA-ADPKD-303
A finding at Screening that precludes safe participation in the study or participants who are likely to be non-compliant with study procedures in the opinion of the Investigator or medical monitor