US-based La Jolla Pharmaceutical has initiated a Phase II extension trial of GCS-100 for the treatment of patients with chronic kidney disease (CKD).
The extension trial (GCS-100-CS-4003) was started at the request of the company's lead investigators, to assess long-term safety and efficacy of GCS-100 in patients who completed the initial 12-week Phase II trial (GCS-100-CS-4002).
The Phase II extension trial will enrol CKD patients who completed the 12-week Phase II trial, including a maximum of 115 patients at five sites.
In the trial, patients will be randomly assigned 1:1 to treatment with 1.5mg/m2 GCS-100 or 30mg/m2 GCS-100.
The company said that all doses of trial drug will be given intravenously once weekly for up to eight consecutive weeks with a four week follow-up period and then at least once a month for a total of one year.
Primary objective of the Phase II extension trial is to determine the safety and tolerability of extended dosing with GCS-100, while the secondary objective will be to determine the efficacy as measured by eGFR of GCS-100 administered for eight weeks relative to the effect of eight weeks of treatment for the same patient on the GCS-100-CS-4002 trial including those who were on placebo.
The trial will also compare crossover data for individual patients from the initial Phase II trial, as well as the extension trial.
A total of 121 patients with at least 18 years of age who have Stage 3b or 4 CKD were enrolled in the initial 12 week Phase II trial.
In the initial Phase II trial, patients were randomly assigned 1:1:1 to treatment with placebo, 1.5mg/m2 GCS-100 or 30mg/m2 GCS-100.
The company said that randomisation was stratified into two groups according to baseline renal function as defined by eGFR values of 15-29 and 30-44 mL/min/1.73m2 respectively.
Initial data from the trial will likely be available in March 2014.
GCS-100 is a complex polysaccharide with the unique ability to bind to and block the effects of galectin-3, a soluble protein, which has been implicated in a number of human diseases that include cancer and chronic organ failure.
Image: GCS-100, a complex polysaccharide, has the ability to bind to and block the effects of galectin-3. Photo: courtesy of freedigitalphotos.net.