RecruitingPhase 1

Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response in Children With Dystonic Cerebral Palsy

United States40 participantsStarted 2024-10-15

Plain-language summary

This study looks at how a medicine called trihexyphenidyl works in children with dystonic cerebral palsy. The study aims to understand how trihexyphenidyl is broken down and used in the body of pediatric patients and whether this is impacted by a person's genetics. Information from this study will also be used to design future clinical trials.

Who can participate

Age range

5 Years – 17 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Ages 5-17 years of age * Diagnosis of cerebral palsy and dystonia causing interference * Parent/legal guardian of a child with a diagnosis of cerebral palsy and dystonia * Parent/legal guardian is willing and able to provide informed permission/assent for the study Exclusion Criteria: * Previously or currently taking trihexyphenidyl * Patients turning 18 years of age within the study period (16 weeks from Study Day 1) * A language barrier for the patient that precludes communication and/or the ability to complete study-related requirements

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Difference in Cmax between CYP2D6 and CYP2C19 phenotype groups

Timeframe: Baseline

Difference in AUC0-n between CYP2D6 and CYP2C19 phenotype groups

Timeframe: Baseline

Difference in AUC0-∞ between CYP2D6 and CYP2C19 phenotype groups

Timeframe: Baseline

Recruitment percentage

Timeframe: Through study completion, an average of 2 years

Retention percentage

Timeframe: Through study completion, an average of 2 years

Dystonia Efficacy Measures Outcome Completion

Timeframe: Through study completion, an average of 2 years

Trial details

NCT IDNCT06554288

SponsorChildren's Mercy Hospital Kansas City

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2029-11-30

Contact for this trial

Rose Gelineau-Morel, MD

816-302-3331 rngelineaumorel@cmh.edu

View on ClinicalTrials.gov More Pediatric Disorder trials

Plain-language summary

Who can participate

Age range

5 Years – 17 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Difference in Cmax between CYP2D6 and CYP2C19 phenotype groups

Timeframe: Baseline

Difference in AUC0-n between CYP2D6 and CYP2C19 phenotype groups

Timeframe: Baseline

Difference in AUC0-∞ between CYP2D6 and CYP2C19 phenotype groups

Timeframe: Baseline

Recruitment percentage

Timeframe: Through study completion, an average of 2 years

Retention percentage

Timeframe: Through study completion, an average of 2 years

Dystonia Efficacy Measures Outcome Completion

Timeframe: Through study completion, an average of 2 years