Clinical Trials

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

Fosmetpantotenate

In December 2018, Retrophin completed patient enrollment in the pivotal Phase 3 FOsmetpantotenate Replacement Therapy (FORT) Study (PKANFORTStudy.com), which is an international, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of fosmetpantotenate in approximately 82 patients with PKAN aged six to 65.

The PKAN-ADL is a novel, PKAN-specific, patient-reported outcome scale measuring motor abilities to function in daily living for patients with PKAN. The scale is an adaptation of Part II of the comprehensive and widely-referenced Unified Parkinson's Disease Rating Scale (UPDRS). For the purposes of this trial, the UPDRS was adapted to be optimally relevant to PKAN through a systematic revision involving experts, patient advocacy leaders and regulatory interaction.

The FORT Study (PKANFORTStudy.com) is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA). As part of this process, Retrophin and the Agency have agreed that the design of this pivotal trial is will be adequate to support a New Drug Application (NDA) if the data from the trial are positive. Fosmetpantotenate was also granted fast track status in the U.S., which is designed to facilitate the development and expedite the regulatory review of medicines intended to treat serious conditions with unmet medical needs, in order to reach patients earlier.

The FDA and European Commission both granted orphan drug designation to fosmetpantotenate for the treatment of PKAN. In the U.S., this status provides Retrophin with development and regulatory incentives, as well as seven years of marketing exclusivity, if fosmetpantotenate is approved for PKAN. Similarly, fosmetpantotenate is eligible for a single marketing authorization that is valid in all EU countries and 10 years of market exclusivity in the region, upon approval for PKAN. Fosmetpantotenate was also granted fast track status in the U.S., which is designed to facilitate the development and expedite the regulatory review of medicines intended to treat serious conditions with unmet medical needs, in order to reach patients earlier.

For additional information about the FORT Study (PKANFORTStudy.com) and to learn more about eligibility, patients can visit pkanfortstudy.com and ClinicalTrials.gov.

Sparsentan

In the fourth quarter of 2018, we initiated the PROTECT Study (http://IgANPROTECT.com) a global, pivotal Phase 3 clinical trial evaluating the long-term nephroprotective potential of sparsentan for the treatment of IgA nephropathy (IgAN). The PROTECT Study, which is expected to enroll approximately 280 patients with IgAN, is designed to include a primary efficacy endpoint evaluating changes in proteinuria to serve as the basis for an NDA filing for Subpart H accelerated approval of sparsentan in the U.S., as well as an application for Conditional Marketing Authorization (CMA) in Europe. Secondary efficacy endpoints are expected to evaluate changes in estimated glomerular filtration rate, or eGFR, which is widely regarded as the best overall measure of kidney function.

In the second quarter of 2018, we initiated DUPLEX (FSGSDUPLEX.com), a pivotal Phase 3 clinical trial of sparsentan in focal segmental glomerulosclerosis (FSGS), which is designed to include an interim analysis of proteinuria to serve as the basis for an NDA filing for Subpart H accelerated approval of sparsentan. The confirmatory endpoint of the study is expected to compare changes from baseline in estimated glomerular filtration rate or, eGFR, which is widely regarded as the best overall measure of kidney function. Sparsentan could be the first FDA-approved pharmacologic treatment option for patients with this rare kidney disorder that often leads to end-stage renal disease.2

In the Phase 2 DUET study, which is currently in its open-label phase, the overall sparsentan treatment group experienced statistically significant reductions in proteinuria, and results showed sparsentan was generally safe and well tolerated in the study.3

To learn more about our clinical trials, including enrollment and study centers, contact Retrophin at medinfo@retrophin.com or visit Clinical Trials.gov.

  1. Gregory, et al. GeneReviews. 2002.
  2. Middleton, et al. Nephrology Rounds 2007; 5(4).
  3. 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.