Bedankt opstapelen...
Ben dit tegengekomen van de "concurrent" galectin pharma mbt hun fase II protocol. Ik denk dat het relevant is vermits de FDA inderdaad neigt naar cirrhose reversal bewijs:
The program includes two clinical studies. The first is a multicenter, randomized, placebo-controlled, double-blind, parallel-group Phase 2 trial to evaluate the safety and efficacy of GR-MD-02 for the treatment of liver fibrosis and resultant portal hypertension in patients with NASH cirrhosis (the NASH-CX trial). This trial is expected to commence in the second quarter of 2015 with data readout expected in the fourth quarter of 2017. In addition, the Company will conduct a smaller trial of shorter duration in NASH patients with advanced fibrosis (the NASH-FX trial).
The NASH-CX trial was designed with guidance from the U.S. Food and Drug Administration (FDA) received during an End-of-Phase 1 meeting. The NASH-CX trial will include 45 sites (and up to 60 sites, if necessary) in the U.S. and Canada and will enroll a total of 156 patients. It will be comprised of three parallel treatment arms of 52 patients each, with one arm receiving 8 mg/kg of GR-MD-02, the expected therapeutic dose, one arm receiving 2 mg/kg of GR-MD-02 and a third arm receiving placebo. Patients will receive a total of 26 infusions every other week for one year and will be evaluated to determine the change in the hepatic venous pressure gradient (HVPG) as compared with placebo.
The FDA has indicated that HVPG may serve as a surrogate primary endpoint for NASH cirrhosis. HVPG will be correlated with secondary endpoints of fibrosis on liver biopsy as well as with measurement of liver stiffness (FibroScan®) and assessment of liver metabolism (13C-methacetin breath test, Exalenz), which are non-invasive measures of the liver that may be used in future studies.