This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF ...
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
The specific aim of this study is to evaluate the performance of three substance use screening instruments (S2BI, BSTAD, and TAPS Tool) against a criterion standard of Diagnostic Statistical Manual - ...
Despite major technological advances, management of type one diabetes mellitus (T1D) remains suboptimal, putting millions of people at risk for immediate and long-term complications. After meals, a ...
The goal of this study is to determine if preoperative temperament is associated with postoperative pain, emergence agitation, emotional and behavioral changes, and overall parent satisfaction. This ...
This research clinical trial is studying the creation and administration of GVAX, an irradiated GM-CSF secreting, autologous neuroblastoma cell vaccine (GVAX) in combination with nivolumab and ...
The overarching goal of the study is to improve the surgical treatment outcomes of FAI, which is affecting an increasing number of military personnel and young active individuals in the general ...
Investigators from eight tertiary care, level 1 pediatric trauma centers have developed a protocol for the establishment of a formal, prospective multi-center adolescent clavicle registry, with ...
The purpose of this study evaluate the relationship between inflammation and epilepsy in neonates with seizures after birth.
The purpose of this 5-year prospective research study is to characterize the association between concussions, sub-concussive exposure, and long-term neurologic health outcomes in former NFL players. ...