8 results
The primary objective of the study is to determine concentration of circulation cytokines na administration of LPS in the presence of iron sucrose (Venofer®), Deferasirox (Exjade®) of placebo.
Use of iron chelation therapy has demonstrated benefits in terms of morbidity and mortality for chronically-transfused thalassemia patients with iron overload. Recent retrospective data (Leitch 2007, Rose 2010, Sanz 2008) suggest that overall…
To study the safety and efficacy of deferasirox as treatment of oxidative stress in adult subjects with sickle cell disease.
Primary objective:To assess the efficacy of PXT3003 compared to Placebo on the disability measured by the ONLS score in CMT1A patients treated for 15 months.Secondary objectives:- To assess the efficacy of PXT3003 compared to Placebo on clinical and…
Primary Objective:To evaluate long-term safety and tolerability of REN001 in subjects with PMM.Secondary Objective:To evaluate subjects with mtDNA-PMM who are receiving long-term treatment with REN001 in terms of PMM associated symptoms, exercise…
Main Objective:To evaluate the efficacy of REN001 in subjects with PMM treated for 24 weeks, assessed by the effect on exercise endurance.Secondary Objective:To evaluate the efficacy of REN001 in subjects with PMM treated for 24 weeks, assessed by…
Primary:To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of (RS)-baclofen, naltrexone hydrochloride, and D-sorbitol) compared to placebo in subjects with CMT1A.Secondary: To evaluate the safety and tolerability of PXT3003…
The primary objectives:Period 1:The primary objective is to assess the long-term (up to 2 years) safety and tolerability of two doses of PXT3003.Period 2:For patients continuing after V9, the main objective will be to offer patients the opportunity…