6 results
To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)
Primary objectiveTo evaluate the long-term safety of 10-, 10-, 20-mg/kg ataluren in patients with nonsense mutation cystic fibrosis (nmCF), who previously participated in pivotal study PTC124-GD-021-CF, as determined by adverse events and laboratory…
The objective is to compare the efficacy and safety of intravenous high-dose penicillin G and hydrocortisone versus placebo in combination with riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).
The main objective of the study is to characterize, quantify, and model hemodynamic changes of different vessel groups in the human brain in response to external stimulation.
This study will evaluate the safety, tolerability, and pharmacokinetics of idasanutlin as a single agent and the safety, tolerability, pharmacokinetics, and preliminary efficacy of idasanutlin in combination with either chemotherapy or venetoclax in…
To determine the immune-related progression free survival (irPFS) rate at 21 weeks and objective response rate (irORR) at 30 weeks in patients with pMMR CRC, dMMR mCRC, dMMR EC , dMMR SBC, dMMR SC treated with pembrolizumab combined with ataluren…