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Clinical Trials: Study Design

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Key clinical studies for SAMSCA

The following are study designs for SALT-1, SALT-2 and SALTWATER, the key clinical trials for SAMSCA. SALT‑1 and SALT‑2 are short‑term, double‑blind studies, whereas SALTWATER is a long-term, open-label study.1,2

Pivotal trials: SALT-1 and SALT-2

  • SALT stands for Study of Ascending Levels of Tolvaptan in Hyponatremia
  • Includes patients with euvolemic or hypervolemic hyponatremia (serum sodium <135 mEq/L) due to a variety of causes, including heart failure, liver cirrhosis, and SIADH

Salt-1 Salt-2 study design

Patients were evaluated at baseline, 8 hours after the first administration of study drug, and on Days 2, 3, 4, 11, 18, 25, 30, and 37. Study drugs were withheld after Day 30, and the effect of discontinuation of the study drug was assessed on Day 37.

*Daily titration based on serum sodium levels.

Objective

  • To investigate whether tolvaptan, an oral vasopressin V2-receptor antagonist that promotes aquaresis—excretion of electrolyte-free water—might be of benefit in hyponatremia

Methods

  • SALT-1 and SALT-2: Two multicenter, randomized, double-blind, placebo-controlled trials (N=448)
    – Identical study designs to assess reproducibility1
  • Patients (aged ≥18 years) had hypervolemic or euvolemic hyponatremia (serum sodium <135 mEq/L) due to causes including heart failure (HF), cirrhosis, or SIADH1
  • Patients were stratified by hyponatremia status (serum sodium 130-134 mEq/L for mild or <130 mEq/L for severe) and presence or absence of heart failure

Primary endpoint

  • The change in the average daily area under the curve (AUC) for the serum sodium concentration from baseline to Day 4 and from baseline to Day 301

SALTWATER

  • SALTWATER stands for the Safety and sodium Assessment of Long-term Tolvaptan With hyponatremia: A year-long, open-label Trial to gain Experience under Real-world conditions
  • A 4-year sequential, open-label extension of the randomized, placebo-controlled, double-blind trials – SALT-1 and SALT-22
SALTWATER study design

Objective

  • To determine whether SAMSCA provides sustained, long-term improvement in serum sodium concentration

Methods

  • Patients from SALT-1 and SALT-2 were all previously on SAMSCA or placebo
  • The median length of time between completion of SALT-1 and SALT-2 and enrollment in SALTWATER was 30 days per patient2
  • Patients were followed for up to 4 years2

References:

  1. Schrier RW, Gross P, Gheorghiade M, et al; SALT Investigators. Tolvaptan, a selective oral vasopressin V2-receptor antagonist, for hyponatremia. N Engl J Med. 2006;355(20):2099-2112.
  2. Berl T, Quittnat-Pelletier F, Verbalis JG, et al; SALTWATER Investigators. Oral tolvaptan is safe and effective in chronic hyponatremia.
    J Am Soc Nephrol. 2010;21(4):705-712.