Understanding how trihexyphenidyl works in children with dystonic cerebral palsy
Pharmacogenomic Contribution to the Biotransformation of Trihexyphenidyl and Development of a Precision Dosing Model for Children With Dystonia and Cerebral Palsy
This study is testing how well the medication trihexyphenidyl works for children with dystonic cerebral palsy and if their genes affect how the drug works.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 5 Years to 17 Years |
| Sex | All |
| Sponsor | Children's Mercy Hospital Kansas City Academic / other |
| Locations | 1 site (Kansas City, Missouri) |
| Trial ID | NCT06554288 on ClinicalTrials.gov |
What this trial studies
This study investigates the effects of trihexyphenidyl in children aged 5-17 with dystonic cerebral palsy. It aims to evaluate how the medication is metabolized in the body and whether genetic factors influence its effectiveness. The study will last for 16 weeks and will help inform the design of future clinical trials based on the findings regarding pharmacokinetics and genetic variations. It is a single-arm, nonrandomized pilot study focused on understanding the drug's biotransformation and response in pediatric patients.
Who should consider this trial
Good fit: Ideal candidates are children aged 5-17 diagnosed with cerebral palsy and dystonia that interferes with their daily activities.
Not a fit: Patients who are currently taking trihexyphenidyl or will turn 18 during the study period may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to more effective treatment strategies for children with dystonic cerebral palsy.
How similar studies have performed: While pharmacogenomic approaches in pediatric populations are being explored, this specific study's focus on trihexyphenidyl and its genetic interactions is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Kansas City, Missouri
- Children's Mercy Hospital Kansas City — Kansas City, Missouri, United States (Recruiting)
Study contacts
- Principal investigator: Rose Gelineau-Morel, MD — Children's Mercy Kansas City
- Study coordinator: Rose Gelineau-Morel, MD
- Email: rngelineaumorel@cmh.edu
- Phone: 816-302-3331
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.