Results 321 to 330 of about 14,301,803 (394)
Abstract Bexicaserin (LP352) is a selective 5‐hydroxytryptamine 2C (5‐HT2C) superagonist in development for the treatment of seizures in developmental and epileptic encephalopathies (DEEs). This double‐blind, placebo‐controlled, single ascending dose (SAD) Phase 1 study aimed to assess the safety, tolerability, and pharmacokinetic (PK) and ...
Rosa Chan+6 more
wiley +1 more source
Abstract Bexicaserin (LP352) is a selective superagonist of the 5‐hydroxytryptamine 2C (5‐HT2C) receptor currently in development for the treatment of seizures that arise from developmental and epileptic encephalopathies. This phase 1, double‐blind, placebo‐controlled multiple ascending dose (MAD) study assessed the safety, tolerability, and ...
Jonathan Williams+7 more
wiley +1 more source
Supplementing Single‐Arm Trials with External Control Arms—Evaluation of German Real‐World Data
As single‐arm trials (SATs) are increasingly used in pharmaceutical research, the validity of such study designs needs to be critically assessed. We characterize the feasibility of supplementing SATs with real‐world data (RWD)‐derived external control arms by determining the proportion of SATs on breast cancer and amyotrophic lateral sclerosis (ALS ...
Martin Russek+2 more
wiley +1 more source
Optimal dosing of oncological drugs is historically determined based on the “higher is better” paradigm. However, a paradigm shift in optimal dose selection has occurred in the development of new modalities, including molecularly targeted drugs, antibody drugs, and immunotherapies.
Hiroe Kitagaki+3 more
wiley +1 more source
The potential of real‐world data (RWD), particularly from patient registries, has been increasingly recognized over the last decade by academia, regulators, and health technology assessment (HTA) bodies for its role in assessing a product's effectiveness and supporting regulatory submissions.
Sonia Zebachi+13 more
wiley +1 more source