NCT06554288

Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response 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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: Children's Mercy Hospital Kansas City · Academic / Other
• Sex: All
• Age: 5 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a 16-week single-arm nonrandomized pilot study of trihexyphenidyl in children with dystonic cerebral palsy (DCP) to 1) evaluate the pharmacokinetics (PK) of trihexyphenidyl (THP) and variation in PK parameters between CYP2D6 and CYP2C19 genotypes and 2) evaluate the feasibility of a future exposure-controlled clinical trial of THP.

Conditions

• Pediatric Disorder
• Genetic Predisposition
• Dystonia, Secondary
• Dystonia
• Cerebral Palsy, Dystonic-Rigid
• Cerebral Palsy, Dyskinetic
• Trihexyphenidyl Adverse Reaction
• Pharmacogenomic Drug Interaction

Interventions

Type	Name	Description
DRUG	Trihexyphenidyl	6-week dose escalation up to 0.25mg/kg TID, followed by a 9-week maintenance period at this dose

Timeline

• Start date: 2024-10-15
• Primary completion: 2029-11-30
• Completion: 2029-12-31
• First posted: 2024-08-15
• Last updated: 2025-07-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study