5 results
The objective of this pivotal study is to assess the PK and PD as well as safety and tolerability of RP103 compared to Cystagon® in patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support the registration…
The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
Primary To test the hypothesis that at least 1 dose of LY2951742 (120 mg or 240 mg/month) is superior to placebo in the prevention of migraine headache in patients with chronic migraineKey Secondary Objectives If LY2951742 (120 or 240 mg/month) is…
To describe and investigate safety and tolerability of the intradermal delivery of two fractional doses of 3/3 µg Comirnaty Original/Omicron BA.4-5 vaccin in patients with Fibrodysplasia Ossificans Progressiva. Additionally to measure the…