Lixivaptan in a Single Subject With Intractable Pain Due to Polycystic Kidney Disease
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ClinicalTrials.gov Identifier: NCT03717181 |
Recruitment Status :
Active, not recruiting
First Posted : October 24, 2018
Last Update Posted : January 29, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Polycystic Kidney | Drug: Lixivaptan | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 1 participants |
Allocation: | N/A |
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 |
Actual Primary Completion Date : | November 4, 2020 |
Estimated Study Completion Date : | February 28, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: Lixivaptan
Lixivaptan capsule, up to 200 mg PO BID
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Drug: Lixivaptan
Daily dose of lixivaptan optimized using a predetermined titration protocol. |
- 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.

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Ages Eligible for Study: | 15 Years to 60 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Capable of understanding the written informed consent/assent.
- Provides signed and witnessed written informed consent/assent, and agrees to comply with protocol requirements and study-related procedures.
- At least 15 years of age at the time of Screening.
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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.
- 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:
- Has a known sensitivity or idiosyncratic reaction to any compound present in lixivaptan or any compound listed as being present in the formulation.
- Is pregnant or breast feeding.
- Has taken any investigational drug or used an investigational device within 30 days or 5 half-lives, whichever is longer, prior to Screening.
- 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.
- Has a history of testing positive for hepatitis B surface antigen, hepatitis C, or human immunodeficiency virus (HIV).
- 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.
- 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.
- 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.

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
United States, Minnesota | |
Mayo Clinic | |
Rochester, Minnesota, United States, 55905 |
Principal Investigator: | Marie C Hogan, MD, PhD | Mayo Clinic |
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 29, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Kidney Diseases Polycystic Kidney Diseases Pain, Intractable Urologic Diseases Kidney Diseases, Cystic Genetic Diseases, Inborn |
Pain Abnormalities, Multiple Congenital Abnormalities Ciliopathies Neurologic Manifestations |