Evaluating Methylphenidate Treatment for Attention and ADHD Symptoms in Children and Adolescents with KBG Syndrome

What this Trial Is About

Researchers are evaluating the effects of methylphenidate on attention deficit and ADHD-related symptoms in children and adolescents with KBG syndrome, a neurodevelopmental disorder marked by developmental delays, intellectual disability, distinctive facial features, and behavioral issues. This trial aims to determine how methylphenidate impacts ADHD-like symptoms in this population, addressing an unmet need for evidence-based treatments. The study also explores the effectiveness in patients who show ADHD symptoms but may not meet full diagnostic criteria. Participants will receive multiple treatment blocks alternating between methylphenidate capsules and placebo capsules in a randomized, crossover design. The study uses a quadruple masking approach to compare the effects of the active drug versus placebo. Treatment and placebo periods occur over several weeks, with repeated assessments during weeks 1, 3, 5, 7, 9, and 11. During the trial, participants and their caregivers will complete various questionnaires and rating scales daily and at specific weeks to monitor ADHD symptoms, emotional problems, adverse effects, and family functioning. Assessments include the Strengths and Difficulties Questionnaire, Emotion Dysregulation Inventory, Goal Attainment Scoring, Personal Questionnaire, and the Autism Diagnostic Observation Scale. The study monitors safety and treatment effects over approximately 11 weeks, with data collected to evaluate daily changes and overall outcomes.

Methylphenidate in KBG Syndrome: N-of-1 Series