Date: 2014-09-02
Type of information: Initiation of the trial
phase: 1b
Announcement: initiation of the trial
Company: Catalyst Pharmaceutical Partners (USA - FL)
Product: CPP-115
Action
mechanism: CPP-115 works by the inhibition of GABA aminotransferase, which leads to increased brain GABA levels that reduce epileptogenesis. Non-clinical data of CPP-115 indicate that there may be a significant reduction, and possibly elimination, of visual field defects (VFDs) from the use of CPP-115 compared to vigabatrin, Sabril ®, which works by a similar mode of action. Based on animal testing to date, CPP-115 has been shown to be at least 200 times more potent than vigabatrin in both in-vitro and animal model studies. The increased potency could enable the development of dosage forms potentially administrable by other routes of administration compared with the marketed oral, immediate release formulations of vigabatrin. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted E.U. orphan medicinal product designation for the treatment of West Syndrome by the European Commission.
Disease:
Therapeutic area: Rare diseases
Country:
Trial details:
Latest
news: * On September 2, 2014, Catalyst Pharmaceutical Partners announced the initiation of its second Phase 1 safety and tolerance study for CPP-115, a novel GABA aminotransferase (GABA-AT) inhibitor. The Phase 1(b) trial is designed in 2 parts to evaluate the safety and tolerability of single and multiple doses of CPP-115, including CNS side effects, and respiratory and cardiovascular safety. In addition: Part 1 of the trial is an open label, single descending dose study in up to 3 cohorts of 2 normal healthy, male volunteers, intended to verify that the drug crosses the blood-brain barrier at levels sufficient to significantly raise brain GABA levels as measured by Magnetic Resonance Spectroscopy (MRS). Catalyst believes increases in brain GABA, an inhibitory neurotransmitter, to be a biomarker for the potential efficacy of CPP-115 as a treatment for Tourette Syndrome, Post-Traumatic Stress Disorder (PTSD) and infantile spasms. If the first dose shows a significant increase, lower single doses will be evaluated to determine a minimal effective dose to be able to set the doses to be evaluated in the multiple dose part of the trial. Part 2 of the study is designed as a randomized, double-blind, multiple ascending dose study in 4 cohorts of eight normal healthy, male volunteers. In addition to studying basic human safety and tolerability, the effect of multiple doses of CPP-115 on brain GABA levels will also be followed by MRS.
