4 results
The primary study objective is to assess the proportion of patients who remain within the IGF-I age adjusted normal limits with pasireotide LAR (60 mg) monotherapy, after 24 weeks of treatment.Secondary study objectives are assessment of the…
Primary Study Objective:To assess the efficacy and safety of the co-administration of low-dose pegvisomant (40 mg, administered via subcutaneous injection given once a week) and long-acting somatostatin analogs (administered once monthly) on the…
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
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…