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RECRUITINGPhase 4INTERVENTIONAL

Methylphenidate in KBG Syndrome: N-of-1 Series

Effectiveness of Methylphenidate in Children and Adolescents With KBG Syndrome: An N-of-1 Series

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

The goal of this clinical trial\] is to learn about the effect of methylphenidate in children and adolescents with KBG syndrome. The main question it aims to answer is: • What is the effectiveness of methylphenidate on attention deficit and ADHD-related symptoms in children and adolescents with KBG syndrome? Participants will receive multiple blocks of treatment with methylphenidate and placebo and fill out various questionnaires.

Who May Be Eligible (Plain English)

Who May Qualify: - Age 6-20 years - Molecularly confirmed diagnosis of KBG syndrome (pathogenic ANKRD11 variant or a chromosome 16q24 deletion including ANKRD11) - Attention deficit or ADHD-related symptoms or a formal ADHD diagnosis, with a significant impact on daily life\* - Presence of a subject's caregiver or supervisor for proxy-reports Who Should NOT Join This Trial: - Family history of acute cardiac death that warrants further cardiac investigation - Cardiovascular disease in medical history (severe hypertension, heart failure, arterial occlusive disease, potentially life-threatening arrythmias, angina pectoris, hemodynamically significant congenital heart defect, cardiomyopathy, myocardial infarction and channelopathy) - Current or previous presence of hyperthyroidism, glaucoma or pheochromocytoma - Use of (psychotropic/stimulant) drugs which interact with MPH - Schizophrenic or psychotic disorder in medical history - Unstable epilepsy (not controlled with medication) - History of frequent drug and/or alcohol abuse - Excessive alcohol/drug use and/or intoxication with one or both during the study - Pregnant or lactating women - Inability to understand or speak Dutch Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * Age 6-20 years * Molecularly confirmed diagnosis of KBG syndrome (pathogenic ANKRD11 variant or a chromosome 16q24 deletion including ANKRD11) * Attention deficit or ADHD-related symptoms or a formal ADHD diagnosis, with a significant impact on daily life\* * Presence of a subject's caregiver or supervisor for proxy-reports Exclusion Criteria: * Family history of acute cardiac death that warrants further cardiac investigation * Cardiovascular disease in medical history (severe hypertension, heart failure, arterial occlusive disease, potentially life-threatening arrythmias, angina pectoris, hemodynamically significant congenital heart defect, cardiomyopathy, myocardial infarction and channelopathy) * Current or previous presence of hyperthyroidism, glaucoma or pheochromocytoma * Use of (psychotropic/stimulant) drugs which interact with MPH * Schizophrenic or psychotic disorder in medical history * Unstable epilepsy (not controlled with medication) * History of frequent drug and/or alcohol abuse * Excessive alcohol/drug use and/or intoxication with one or both during the study * Pregnant or lactating women * Inability to understand or speak Dutch

Treatments Being Tested

DRUG

Methylphenidate Hydrochloride

Methylphenidate hydrochloride in capsules

DRUG

Placebo

Microcrystalline cellulose in capsules

Locations (1)

Radboud University Medical Center
Nijmegen, Gelderland, Netherlands