4 results
A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase deficiency
The primary objective of this study is to evaluate the safety of sebelipase alfa in a more broad population of subjects with LAL Deficiency than have been previously studied.The secondary objectives of this study are (1) to evaluate effects of…
Primary ObjectivesParts 2To evaluate the safety and tolerability of INCB001158 for patients with advanced/metastatic and/or treatment-refractory solid tumorsParts 3To evaluate the safety and tolerability of INCB001158 in combination with…
To assesses the efficacy of Tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1.
The primary objective is to evaluate the reproducibility of the Corneometer® as a measurement instrument to quantify hydration of the vaginal wall, evaluated by intra- and interobserver variability. The secondary objective is to measure the response…