6 results
- To gather long-term data on the safety and tolerability of idebenone inFriedreich*s Ataxia- To explore the effect of idebenone after longer term administration onneurological symptoms and signs as assessed by the InternationalCooperative Ataxia…
The primary objective of this study is to evaluate the long-term safety and tolerability of OV101 in individuals with AS assessed by the incidence and severity of AEs and SAEs in subjects who are at least 2 years old.The secondary objectives of this…
Primary:* To assess the efficacy of idebenone compared to placebo, in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in Forced Vital Capacity percent predicted (FVC %p) using…
All efficacy study objectives will be assessed in the pediatric AS study population of subjects who are 4 to 12 years old. All safety study objectives will be assessed in the pediatric AS study population of subjects who are 2 to 12 years old.…
Part 1In Part 1 of the study, no study compound will be administered, but a bone marrow aspiration and a blood sample will be taken to evaluate an assay that will be used for Part 2 and Part 3 of the study. Because no nirogacestat is given, we can…
DOSE EXPLORATION:Primary:To determine the safety and tolerability of GSK2857916 in combination with other anti-cancer treatments (in each sub-study), and to establish the recommended Phase 2 dose for each sub-study combination treatment.Secondary:…