4 results
1. Overall survival (primary efficacy endpoint)2a. Efficacy: progression-free survival (PFS), overall response rate (ORR = CR + PR), and a clincal benefit rate (CBR) will be evaluated as CR + PR + SD for at least 3 months.2b. Safety: adverse events…
Progesterone for Breast Development in Trans Women; Assessment of effects and safety -a pilot trial-
To explore the effects on breast development of addition of progesterone to the treatment with estradiol in trans women after vaginoplasty or orchiectomy. Secondary objectives include safety and patient satisfaction, mood, and sleep.
The primary objectives are: To evaluate the CNS activity of DNL310 vs idursulfase as measured by the cerebrospinal fluid (CSF) concentration of heparan sulfate (HS) in participants with the neuronopathic form of mucopolysaccharidosis type II (nMPS…
The primary objective of the study is the following:• To characterize the safety and tolerability of DNL310 in pediatric subjects with MPS II (all cohorts)The secondary objectives of the study are as follows:• To characterize the CNS effects of…