Effect of Methylphenidate on KBG Syndrome in Children and Adolescents
Effectiveness of Methylphenidate in Children and Adolescents With KBG Syndrome: An N-of-1 Series
This study is testing if a medication called methylphenidate can help children and teenagers with KBG syndrome who have attention and ADHD-related challenges.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 6 Years to 20 Years |
| Sex | All |
| Sponsor | Radboud University Medical Center Academic / other |
| Locations | 1 site (Nijmegen, Gelderland) |
| Trial ID | NCT06465641 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effectiveness of methylphenidate in treating attention deficit and ADHD-related symptoms in children and adolescents diagnosed with KBG syndrome. Participants will undergo multiple treatment blocks with methylphenidate and placebo, while completing various questionnaires to assess their symptoms. KBG syndrome is a neurodevelopmental disorder that often presents with behavioral issues, including ADHD, and there is a significant need for evidence-based interventions. The study aims to provide data on optimal dosing and potential adverse effects of methylphenidate in this specific population.
Who should consider this trial
Good fit: Ideal candidates are children and adolescents aged 6-20 years with a molecularly confirmed diagnosis of KBG syndrome and significant ADHD-related symptoms.
Not a fit: Patients with a history of severe cardiovascular disease or other specified medical conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a targeted treatment option for managing ADHD-related symptoms in children with KBG syndrome.
How similar studies have performed: Preliminary findings suggest that methylphenidate may be more effective in KBG syndrome patients than in the general ADHD population, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Nijmegen, Gelderland
- Radboud University Medical Center — Nijmegen, Gelderland, Netherlands (Recruiting)
Study contacts
- Study coordinator: Charlotte Ockeloen, MD
- Email: charlotte.ockeloen@radboudumc.nl
- Phone: +31243613946
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.