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Lixivaptan in a Single Subject With Intractable Pain Due to Polycystic Kidney Disease

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03717181
Recruitment Status : Enrolling by invitation
First Posted : October 24, 2018
Last Update Posted : January 30, 2020
Sponsor:
Information provided by (Responsible Party):
Palladio Biosciences

Brief Summary:
Abdominal pain, sometimes severe, is a known symptom of polycystic kidney disease (PKD) related to the cystic expansion. This study will investigate whether the vasopressin V2 receptor antagonist lixivaptan can alleviate intractable pain in a pediatric PKD patient who cannot tolerate the related vasopressin V2 receptor antagonist tolvaptan.

Condition or disease Intervention/treatment Phase
Polycystic Kidney Drug: Lixivaptan Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Expanded Access Use of Lixivaptan in a Single Subject With Intractable Pain Due to Polycystic Kidney Disease
Actual Study Start Date : May 22, 2019
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : January 31, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Diseases

Arm Intervention/treatment
Experimental: Lixivaptan Drug: Lixivaptan
Daily dose of lixivaptan optimized using a predetermined titration protocol.




Primary Outcome Measures :
  1. Pain intensity [ Time Frame: 6 months ]
    Assessment of abdominal pain intensity on a standard numerical rating scale of pain captured as a whole number between 0 and 10 where 0 is "no pain" and 10 is "worst pain imaginable". Improvement in pain is represented as a lower pain intensity score. There are no subscales.



Information from the National Library of Medicine

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Ages Eligible for Study:   15 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Capable of understanding the written informed consent/assent.
  2. Provides signed and witnessed written informed consent/assent, and agrees to comply with protocol requirements and study-related procedures.
  3. At least 15 years of age at the time of Screening.
  4. Subject has been diagnosed with ADPKD by modified Ravine criteria:

    • If the subject has a family history of ADPKD, a minimum of 3 cysts per kidney by sonography or 5 cysts by computerized tomography or MRI; or
    • If the subject does not have a family history of ADPKD, a minimum of 10 cysts per kidney by any radiologic method and exclusion of other cystic kidney diseases.
  5. Considered by the Investigator to be in good health relative to underlying CKD status and clinically stable with respect to underlying CKD, based on medical evaluation that includes medical and surgical history, as well as a complete physical examination including vital signs, ECG, and laboratory test results.

Exclusion Criteria:

  1. Has a known sensitivity or idiosyncratic reaction to any compound present in lixivaptan or any compound listed as being present in the formulation.
  2. Is pregnant or breast feeding.
  3. Has taken any investigational drug or used an investigational device within 30 days or 5 half-lives, whichever is longer, prior to Screening.
  4. Has taken tolvaptan, conivaptan, somatostatin analogs (e.g. lanreotide, pasireotide, octreotide, etc.), mTOR kinase inhibitors (e.g. everolimus, sirolimus, etc.), or oral or intravenous antibiotics within 30 days or 5 half-lives, whichever is longer, prior to Day 1.
  5. Has a history of testing positive for hepatitis B surface antigen, hepatitis C, or human immunodeficiency virus (HIV).
  6. Consumes grapefruit or Seville oranges (or their juices, or foods containing their extract) from 7 days prior to the first dose of study medication and until after the final dose.
  7. Has clinically significant liver disease, or clinically significant liver function abnormalities or serology other than that expected for ADPKD with cystic liver disease at baseline.
  8. Has any clinically significant laboratory abnormalities or concomitant conditions other than ADPKD (including treatment for such conditions) that, in the opinion of the Investigator, could either interfere with the study drug or pose an unacceptable risk to the subject.

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): NCT03717181


Locations
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United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Palladio Biosciences

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Responsible Party: Palladio Biosciences
ClinicalTrials.gov Identifier: NCT03717181    
Other Study ID Numbers: PA-103
First Posted: October 24, 2018    Key Record Dates
Last Update Posted: January 30, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Pain, Intractable
Urologic Diseases
Kidney Diseases, Cystic
Abnormalities, Multiple
Congenital Abnormalities
Ciliopathies
Genetic Diseases, Inborn
Pain
Neurologic Manifestations
Signs and Symptoms