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

NCT03717181 | Unknown

• 1 other identifier: PA-103
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Polycystic Kidney

Interventions

Lixivaptan DRUG

Daily dose of lixivaptan optimized using a predetermined titration protocol.

Eligibility Criteria

• Age: 15 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• 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
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Palladio Biosciences: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Polycystic Kidney Diseases

Interventions

lixivaptan

Condition Hierarchy (Ancestors)

Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Study Officials

• Marie C Hogan, MD, PhD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 22, 2018
• First Posted: October 24, 2018
• Last Updated: October 14, 2021

Record last verified: 2021-10

Locations

US (1)