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

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.

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.

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