Actively Recruiting
Pharmacogenomic Contribution to the Biotransformation of Trihexyphenidyl and Development of a Precision Dosing Model for Children With Dystonia and Cerebral Palsy
Led by Children's Mercy Hospital Kansas City · Updated on 2025-07-18
40
Participants Needed
1
Research Sites
4 weeks
Total Duration
On this page
Sponsors
C
Children's Mercy Hospital Kansas City
Lead Sponsor
U
University of Kansas Medical Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying how the medicine trihexyphenidyl is processed in children with dystonic cerebral palsy. This study aims to understand how genetic differences affect the way children's bodies break down and use this medicine. The information gathered will help design future clinical trials to better tailor trihexyphenidyl doses. Participants will receive trihexyphenidyl in a single group over 16 weeks, starting with a dose escalation during the first 6 weeks. The dose increases from 0.05 mg/kg twice daily to 0.25 mg/kg three times daily, followed by a 9-week maintenance period at the highest dose. This study is a nonrandomized pilot trial focused on pharmacokinetics and genotype effects. During the study, children will be closely monitored for how their bodies absorb and process the medicine, with assessments of dystonia symptoms and safety. Researchers will use various scales to measure changes in dystonia and functional impact, as well as caregiver observations. The study will track recruitment, retention, and any adverse events over an average of two years.
CONDITIONS
Brief Title
Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response in Children With Dystonic Cerebral Palsy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ages 5-17 years
- Diagnosis of cerebral palsy and dystonia causing interference
- Parent or legal guardian of a child with cerebral palsy and dystonia
- Parent or legal guardian willing and able to provide informed permission/assent for the study
You will not qualify if you...
- Previously or currently taking trihexyphenidyl
- Turning 18 years old within the 16-week study period
- Language barrier preventing communication or study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 16 weeks
Participants receive trihexyphenidyl with a 6-week dose escalation followed by a 9-week maintenance period at the target dose.
Weekly visits for up to 16 weeks
Trial Site Locations
Total: 1 location
1
Children's Mercy Hospital Kansas City
Kansas City, Missouri, United States, 64108
Actively Recruiting
Research Team
R
Rose Gelineau-Morel, MD
R
Rachel Nass
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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