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Kidney Week

Abstract: SA-PO516

ELiSA, a Phase 2 Clinical Study with the Novel Vasopressin V2 Receptor Antagonist Lixivaptan in Patients with Autosomal Dominant Polycystic Kidney Disease

Session Information

  • ADPKD: Clinical Studies
    October 27, 2018 | Location: Exhibit Hall, San Diego Convention Center
    Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidney

  • 1001 Genetic Diseases of the Kidney: Cystic

Authors

  • Pellegrini, Lorenzo, Palladio Biosciences, Newtown, Pennsylvania, United States
  • Hogan, Linda C., Palladio Biosciences, Newtown, Pennsylvania, United States
  • Subach, Ruth Ann, DS InPharmatics, LLC, Harleysville, Pennsylvania, United States
Background

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is one of the most prevalent inherited genetic diseases of the kidney in humans. Recent advances established vasopressin V2 receptor inhibition as a clinically validated mechanism of action for the treatment of ADPKD; however, safe and effective disease-modifying therapies for ADPKD are still lacking. Here we describe a first-in-patients clinical study with lixivaptan, a potent, selective vasopressin V2 antagonist, in patients with ADPKD.

Methods

ELiSA (Evaluation of Lixivaptan in Subjects with ADPKD) is a phase 2, open-label, multicenter clinical study of lixivaptan in patients with ADPKD to be conducted at up to 15 clinical sites in the United States. Eligible patients will include adults aged 18–60 years with relatively preserved kidney function (chronic kidney disease CKD1 and CKD2; n = 16) or moderately impaired renal function (CKD3; n = 16). Two doses of lixivaptan, administered for 7 days, will be tested in eight patients within each patient group.

Results

The primary objectives of the ELiSA study are to characterize the safety, tolerability and pharmacokinetic profile of lixivaptan and its major metabolites following multiple doses in ADPKD patients with various degrees of kidney function impairment. The secondary objectives are to characterize the effect of lixivaptan on pharmacodynamic markers that have been shown to correlate with the clinical activity of vasopressin antagonists in the treatment of ADPKD, including urine osmolality, urine output, serum creatinine, plasma copeptin and total kidney volume.

Conclusion

The ELiSA clinical study is an important milestone toward the potential approval of lixivaptan as a safe and effective therapy for the treatment of ADPKD in a broad patient population. Study results will inform the design and execution of an upcoming pivotal registration study with lixivaptan for the treatment of ADPKD.

Funding

  • Commercial Support