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Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04064346
Recruitment Status : Not yet recruiting
First Posted : August 21, 2019
Last Update Posted : May 8, 2020
Sponsor:
Information provided by (Responsible Party):
Palladio Biosciences

Brief Summary:
This is a Phase 3, double-blind, placebo-controlled, randomized trial to demonstrate the efficacy and safety of lixivaptan in subjects with ADPKD. Approximately 2000 subjects with ADPKD meeting the entry criteria will be screened in order to randomize 1200 subjects to lixivaptan or placebo in a 2:1 ratio in this study. After meeting entry criteria during a 2-4 week screening period (that may extend up to 8 weeks), subjects will enter a 1-week single-blind placebo run-in period to obtain certain baseline measurements followed by a 3-6 week single-blind dose titration period during which lixivaptan administered twice daily will be titrated to a dose that is tolerated and results in a reduced trough urine osmolality (or the maximum dose level). The minimum dose to enter the Double-blind, Randomized Treatment Period is 100 mg twice a day (BID). Those subjects successfully titrated and tolerating the drug will then be randomized (2:1) to either continue lixivaptan or switch immediately to matching placebo in a double-blind manner. Randomization will be stratified for Chronic Kidney Disease (CKD) Stage, hypertension status, age, and region. Double-blind treatment will continue for 52 weeks after which study drug will be held, and final assessments obtained during 3 visits starting on the 8th day after the last dose of double-blind study drug and lasting up to the 21st day.

Condition or disease Intervention/treatment Phase
Polycystic Kidney Disease, Adult Drug: Lixivaptan Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 1200 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Double-blind, randomized, placebo-controlled
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Sponsor
Primary Purpose: Treatment
Official Title: A 52-Week, Phase 3, Double-blind, Placebo-controlled, Randomized Study of the Efficacy and Safety of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Estimated Study Start Date : April 2021
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : October 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Diseases

Arm Intervention/treatment
Experimental: Lixivaptan
Lixivaptan capsules, 100-200 mg twice a day (BID)
Drug: Lixivaptan
Oral vasopressin 2 receptor antagonist

Placebo Comparator: Placebo
Matching placebo capsules BID
Drug: Placebo
Matching placebo




Primary Outcome Measures :
  1. Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: 52 weeks ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of ADPKD
  • eGFR ≥30 mL/min/1.73 m2 and ≤90 mL/min/1.73 m2
  • Body mass index (BMI) between 18 and 35 kg/m2
  • Minimum overnight, fasting spot urine osmolality ≥300 milliosmole (mOsm)/kg

Exclusion Criteria:

  • Known sensitivity or idiosyncratic reaction to any compound present in lixivaptan and related compounds.
  • Hypovolemia or inability to perceive thirst
  • Unable to swallow the study drug
  • Abnormal serum sodium concentration at Screening
  • Subjects who have taken any investigational drug or used an investigational device within 30 days, or 5 half-lives, whichever is longer, prior to Screening
  • Subjects who are taking, have taken within the past 2 weeks, or are expected to be taking, strong or moderate CYP3A4 or CYP2C8 inhibitors or inducers including regular use of grapefruit juice or Seville oranges
  • Prior use of tolvaptan or lixivaptan within the past 3 months.
  • Prior use of conivaptan, somatostatin analogs (e.g. lanreotide, pasireotide, octreotide, etc.), metformin, nicotinamide, bardoxolone, venglustat, demeclocycline, or mammalian target of rapamycin (mTOR) kinase inhibitors (e.g. everolimus, sirolimus, etc.) to treat ADPKD within the past 6 months
  • Requirement for chronic diuretic use
  • Advanced diabetes (e.g., glycosylated hemoglobin [HgbA1c] >7.5%, and/or glycosuria by dipstick, significant proteinuria [>300 mcg albumin/mg creatinine]), other significant renal disease, renal cancer, transplanted kidney, single kidney, recent kidney surgery within the past 6 months (including cyst drainage or fenestration) or acute kidney injury within past 6 months
  • 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 subject.
  • History of testing positive for hepatitis B surface antigen (HBsAg), hepatitis C (HCV), or human immunodeficiency virus (HIV).
  • History of clinically significant drug or alcohol abuse in the past 2 years.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04064346


Contacts
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Contact: Elaine Richardson 833-753-5494 ClinicalTrials@palladiobio.com
Contact: Neil Shusterman, MD ClinicalTrials@palladiobio.com

Sponsors and Collaborators
Palladio Biosciences
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Responsible Party: Palladio Biosciences
ClinicalTrials.gov Identifier: NCT04064346    
Other Study ID Numbers: PA-ADPKD-301
First Posted: August 21, 2019    Key Record Dates
Last Update Posted: May 8, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Kidney Diseases
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Urologic Diseases
Kidney Diseases, Cystic
Abnormalities, Multiple
Congenital Abnormalities
Ciliopathies
Genetic Diseases, Inborn