Methylphenidate in KBG Syndrome: N-of-1 Series
Recruiting · NCT06465641 · Interventional (participants receive a specific treatment) · Lead sponsor: Radboud University Medical Center
View the official record on ClinicalTrials.gov →Interventions studied
Methylphenidate HydrochloridePlacebo
What this trial is about
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 can take part
Age range
6 Years to 20 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 4
Study type
Interventional (participants receive a specific treatment)
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
Where it is running
1 location listed.
- Radboud University Medical Center - Nijmegen, Gelderland, Netherlands