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
This trial does not accept healthy volunteersQualifiers
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
Design model
Crossover
Treatments tested in this trial
Methylphenidate Hydrochloride
DrugMethylphenidate hydrochloride in capsules
Placebo
DrugMicrocrystalline cellulose in capsules
Treatment groups
15 Participants
are divided into 2 treatment groupsGroup A: MethylphenidateActive comparator 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Strenghts and difficulties questionnaire, ADHD subscale
Minimum score 1, maximum score 10 (higher score is worse outcome).
Time frame
Baseline, and daily in week 1,3,5,7,9,11Secondary outcomes
1
Strenghts and difficulties questionnaire, emotional problems subscale
Minimum score 1, maximum score 10 (higher score is worse outcome).
Time frame
Baseline, and daily in week 1,3,5,7,9,112
Dutch shortened version of the Emotion Dysregulation Inventory (EDI) reactivity index
Minimum score 0, maximum score 28 (higher score is worse outcome)
Time frame
Baseline, and daily in week 1,3,5,7,9,113
Goal Attainment Scoring (GAS)
Personal goals, no minimum or maximum score
Time frame
Baseline, and at the end of week 1,3,5,7,9,114
Personal Questionnaire (PQ)
Personal goals, no minimum or maximum score
Time frame
Baseline, and at the end of week 1,3,5,7,9,11Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov