Effect of Methylphenidate on KBG Syndrome in Children and Adolescents
Official title: Effectiveness of Methylphenidate in Children and Adolescents With KBG Syndrome: An N-of-1 Series
Run by Radboud University Medical Center · NCT06465641 · Data updated 2026-11-12
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 |
| Ages | 6 Years to 20 Years |
| Sex | All |
| Enrollment | 15 (estimated) |
| Sponsor | Radboud University Medical Center Academic / other |
| Locations | 1 site (Nijmegen, Gelderland) |
| Status | Recruiting |
| Started | November 13, 2024 |
| Main results expected | January 1, 2028 |
| Study ends | October 1, 2028 |
| Registry record updated | August 11, 2026 |
| Trial ID | NCT06465641 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a 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.
✕Probably not a fit
Patients with a history of severe cardiovascular disease or other specified medical conditions may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
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.
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 gone: 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.
Who can join: the full rules
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 it's running
-
Radboud University Medical Center
Nijmegen, Gelderland, Netherlands Recruiting
Study contacts
- Study coordinator: Charlotte Ockeloen, MD
- Email: charlotte.ockeloen@radboudumc.nl
- Phone: +31243613946
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
AI-assisted home-based mood diary for kids with ADHD
A home-based program uses an AI-assisted diary that turns daily mood entries into comic strips to see if it helps kids with ADHD improve emotion regulation.
-
NeuroTrainer VR cognitive program to improve academic focus
This project will test whether NeuroTrainer's VR-based cognitive and physical training can help 11–17 year-olds with or without ADHD improve attention and executive control.
-
Internet-delivered treatment for adults with ADHD
This study is testing an online therapy program for adults with ADHD to see if it helps them manage their symptoms and improve their quality of life.
-
Comparing Sertraline and Placebo for Treating Anxiety in Children with Neurodevelopmental Disorders
This study is testing if the medication sertraline can help reduce anxiety in children and teens aged 8-17 with neurodevelopmental disorders like autism and Fragile X syndrome compared to a placebo.
-
Brain imaging to study the effects of methylphenidate in adults with ADHD
This study is testing how brain scans can help us understand how adults with ADHD respond to the medication methylphenidate compared to a placebo.
-
Registry for patients with rare diseases
This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.