3 results
Primary Objective: To determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary Objectives: Safety, formula acceptability and…
The primary objective of this study is to evaluate feasibility of performing MR-HIFU treatment of uterine fibroids in a Philips Sonalleve MR-HIFU system equipped with a Direct Skin Cooling device. Secondary objective is evaluation of safety.
CORE PERIODPrimary:* Evaluate the safety and tolerability of up to 24 weeks of AG-348 administration in patients with pyruvate kinase deficiency (PK deficiency).Secondary:* Evaluate the pharmacokinetics (PK) of AG-348 and the metabolite AGI-8702.*…