Clinical Trials

We partner with researchers from around the world to conduct clinical trials studying our investigational therapies.

In late 2016, we announced positive results from the Phase 2 DUET study of sparsentan in which the overall sparsentan treatment group achieved statistical significance in the study's primary efficacy endpoint, reduction of proteinuria.1 Currently, the study is in its open-label phase, and the Company is working with the FDA to find the most expeditious path forward for sparsentan. This investigational therapy could be the first FDA-approved pharmacologic treatment option for patients with focal segmental glomerulosclerosis (FSGS), a rare kidney disorder that often leads to end-stage renal disease.2  

We are working to initiate the Phase 3 FORT study of RE-024, our investigational therapy for pantothenate kinase-associated neurodegeneration (PKAN), and expect to begin dosing patients in the coming months. The study will be conducted according to a Special Protocol Assessment (SPA) agreement with the FDA. As part of this process, Retrophin and the agency have agreed that the design of this pivotal trial is adequate to support a New Drug Application. RE-024 could be the first approved replacement therapy targeting the underlying cause of this rare and life-threatening neurodegenerative condition, as current therapeutic strategies are limited to symptom management.3 In 2015, we completed a Phase 1 single ascending dose study of RE-024 in 40 healthy adult volunteers. The double-blind, placebo-controlled study evaluated five oral dose levels of the compound up to 1,800 mg.

To learn more about our clinical trials, including enrollment and study centers, contact Retrophin at medinfo@retrophin.com or visit the National Institutes of Health clinical trial registry.

 

  1. Trachtman H, Nelson P, Radko K on behalf of the DUET Investigators. Efficacy and Safety of Sparsentan, a Dual Angiotensin II and Endothelin Type A Receptor Antagonist, in Patients with Focal Segmental Glomerulosclerosis: A Phase 2 Trial (DUET).  Presented at the American Society of Nephrology Kidney Week Meeting; November 15-20 2016, Chicago, IL.
  2. Middleton, et al. Nephrology Rounds 2007; 5(4).
  3. Gregory, et al. GeneReviews. 2002.