What this Trial Is About

Researchers are evaluating the effect of methylphenidate on attention deficit and ADHD-related symptoms in children and adolescents aged 6 to 20 years with KBG syndrome, a neurodevelopmental disorder marked by developmental delay, intellectual disability, and behavioral challenges such as ADHD and autism spectrum disorder. The study addresses an unmet need for evidence-based treatments for behavioral problems in KBG syndrome, aiming to understand methylphenidate's effectiveness and safety using an N-of-1 series trial design. Participants will receive multiple treatment blocks alternating between methylphenidate hydrochloride capsules and placebo capsules containing microcrystalline cellulose. The study involves comparing these treatments to assess their impact on ADHD symptoms in this specific population, including those with a formal or probable ADHD diagnosis. During the study, participants will complete the Strengths and Difficulties Questionnaire focusing on the ADHD subscale at baseline and multiple times over 11 weeks (weeks 1, 3, 5, 7, 9, and 11). Caregivers will provide proxy reports, and researchers will monitor behavioral responses to treatment, collecting data to evaluate methylphenidate's impact on attention and related symptoms in children and adolescents with KBG syndrome.

Methylphenidate in KBG Syndrome: N-of-1 Series