2 results
Approved WMOPending
The primary objective of this pilot study is to evaluate the effects of 6 months of recombinant human IGF-I (rhIGF-I) treatment on the body composition.The secondary objective is to assess the effects of the treatment on the liver function,…
Approved WMORecruiting
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…