Actively Recruiting
Effectiveness of Methylphenidate in Children and Adolescents With KBG Syndrome: An N-of-1 Series
Led by Radboud University Medical Center · Updated on 2024-12-27
15
Participants Needed
1
Research Sites
39 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effect of methylphenidate on attention deficit and ADHD-related symptoms in children and adolescents with KBG syndrome, a neurodevelopmental disorder marked by developmental delay, intellectual disability, and various behavioral problems including ADHD. This study aims to provide evidence-based data on methylphenidate's effectiveness and dosing in this specific group, including those with probable ADHD who may not meet formal diagnostic criteria. Participants receive multiple treatment blocks alternating between methylphenidate capsules and placebo capsules in a randomized, quadruple-blind design. The study uses an N-of-1 series approach, where each participant experiences both treatments over time. This design helps assess individual responses to the medication compared to placebo. During the study, participants and their caregivers complete various questionnaires and assessments at baseline and multiple timepoints, including the Strengths and Difficulties Questionnaire focusing on ADHD symptoms, emotional problems, and other behavioral measures. Adverse effects are monitored daily during treatment weeks. The study also includes evaluations like the Autism Diagnostic Observation Scale and family functioning assessments. Participation spans multiple weeks with frequent monitoring to capture detailed symptom changes and safety data.
CONDITIONS
Brief Title
Methylphenidate in KBG Syndrome: N-of-1 Series
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 6 to 20 years
- Molecularly confirmed diagnosis of KBG syndrome (pathogenic ANKRD11 variant or chromosome 16q24 deletion including ANKRD11)
- Attention deficit or ADHD-related symptoms or a formal ADHD diagnosis with significant impact on daily life
- Presence of a caregiver or supervisor for proxy reports
You will not qualify if you...
- Family history of acute cardiac death requiring further cardiac investigation
- Cardiovascular disease in medical history (including severe hypertension, heart failure, arterial occlusive disease, life-threatening arrhythmias, angina, significant congenital heart defects, cardiomyopathy, myocardial infarction, channelopathy)
- Current or past hyperthyroidism, glaucoma, or pheochromocytoma
- Use of psychotropic or stimulant drugs that interact with methylphenidate
- History of schizophrenic or psychotic disorder
- Unstable epilepsy not controlled with medication
- History of frequent drug or alcohol abuse
- Excessive alcohol or drug use or intoxication during the study
- Pregnant or lactating women
- Inability to understand or speak Dutch
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 11 weeks
Participants receive methylphenidate or placebo in a randomized crossover design to evaluate effectiveness on ADHD-related symptoms.
Daily assessments during weeks 1, 3, 5, 7, 9, and 11
Trial Site Locations
Total: 1 location
1
Radboud University Medical Center
Nijmegen, Gelderland, Netherlands, 6500 HB
Actively Recruiting
Research Team
C
Charlotte Ockeloen, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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