PHILADELPHIA—An investigational drug that inhibits galectin-3, a protein implicated in interstitial and glomerular fibrosis resulting in progressive loss of kidney function, significantly improved renal function in patients with chronic kidney disease (CKD), according to findings presented at the 2014 Kidney Week meeting.
In a phase 2 randomized study, researchers led by Pablo E. Pergola, MD, PhD, of Renal Associates PA, in San Antonio, Tex., compared the drug, called GCS-100, with placebo in 121 CKD patients, of whom 117 completed the trial. Subjects had a mean age of 64 years (range 29-85 years).
CKD was due to diabetes in 79 patients, hypertension in 29 patients, and other causes in 9 patients. Dr. Pergola’s group randomly assigned patients to receive GCS-100 at a dosage of either 1.5 or 3.0 mg/m2 or placebo (saline) for 8 weeks, followed by a 5-week observation period.
At a dose of 1.5 mg/m2, GCS-100 resulted in a significant 1.26 mL/min/1.73 m2 improvement in estimated glomerular filtration rate (eGFR) compared with placebo after 8 weeks of dosing. Patients with a diabetes etiology experienced a significant 2.33 mL/min/1.73 m2 improvement versus placebo.
Dr. Pergola and his colleagues confirmed the efficacy of CGS-100 at a dosage of 1.5 mg/m2 in a randomized extension study in patients who originally had been assigned to placebo.
The researchers noted that GCS-100 was safe and well tolerated, with no serious adverse events or early terminations observed in the 1.5 mg/m2 group.