Clinical Research Directory

A Phase II Study of Basmisanil in Children and Adolescents With Dup15q Syndrome

The primary goal of this Phase 2 clinical trial is to determine the effects of a selective negative allosteric modulator (NAM) of GABAA α5 receptor, Basmisanil (NTX-1511), on brain wave (EEG) characteristics in children and adolescents with Dup15q syndrome. The main questions this trial aims to answer are: * How does NTX-1511 affect brain waves (as measured by EEG) in subjects with Dup15q syndrome? * Does NTX-1511 impact other developmental features of subjects with Dup15q syndrome? Researchers will compare the effects of NTX-1511 with matching placebo (look-alike granules that contain no drug). Participants will: * Take NTX-1511 or matching placebo 3 times every day for 12 weeks * Visit the clinic 7 times over the course of 23 weeks

Parent and Infant Inter(X)Action Intervention (PIXI)

The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or intellectual and developmental disabilities. The proposed project will capitalize and expand upon existing empirically based interventions designed to improve outcomes for infants with suspected developmental delays. Participants will be infants with a confirmed diagnosis of a neurogenetic disorder (e.g., fragile X, Angelman, Prader-Willi, Dup15q, Phelan-McDermid, Rhett, Smith Magenis, Williams, Turner, Kleinfelter, Down syndromes, Duchenne muscular dystrophy) within the first year of life and their parents/caregivers. The intervention, called the Parent and Infant Inter(X)action Intervention (PIXI) is a comprehensive program inclusive of parent education about early infant development and the neurogenetic disorder for which they were diagnosed, direct parent coaching around parent-child interaction, and family/parent well-being support. The protocol includes repeated comprehensive assessments of family and child functioning, along with an examination of feasibility and acceptability of the program.