5 results
The primary objective of this study is to assess the objective response rate (ORR, the percentage of treated patients in whom the tumor significantly reduces in size or becomes non-detectable) of SAR240550 administered as a 60min intravenous…
primary • To determinne the safety and tolerability of sorafenib when administered in combination with gemcitabine and carboplatin.• To determine the maximal tolerated dose (MTD), dose limiting toxicity (DLT) and optimal treatment schedule of…
Primary:* To determine the efficacy profile of PA101 delivered via a high efficiency nebulizer (eFlow®,PARI) in comparison with placebo following 6 weeks of treatment in patients with indolentsystemic mastocytosis (ISM) who are symptomatic despite…
The objectives of the study are as follows:Primary:• Part 1: To assess the pharmacokinetics and relative bioavailability of three different formulations of PA101 (4% cromolyn sodium with and without mannitol, and 6% cromolyn sodium without mannitol…
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.