Methylphenidate in KBG Syndrome: N-of-1 Series
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age6-20
SponsorRadboud University Medical Center
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.
Eligibility criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Methylphenidate Hydrochloride
- Placebo
Treatment groups
15 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov