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New treatment option for Kbg Syndrome
Official title Methylphenidate in KBG Syndrome: N-of-1 Series
ClinicalTrials.gov ID: NCT06465641
What this study is testing
What is Methylphenidate Hydrochloride?
Methylphenidate Hydrochloride is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for kbg syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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?
- Phase 4: studies an already-approved treatment
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 20
You may be able to join if
- 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
You likely can't join if
- 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...
- 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)
See the full eligibility 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
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Nijmegen, Gelderland, Netherlands
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years to 20 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Nijmegen, Gelderland, Netherlands. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.