Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response in Children With Dystonic Cerebral Palsy (TRIKE2)

Children's Mercy Hospital Kansas City

Status and phase: Enrolling; Phase 1

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

Treatments: Drug: Trihexyphenidyl

Study type: Interventional

Funder types: Other

Identifiers

NCT06554288

STUDY00003228

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 estimated patients

Sex: All

Ages: 5 to 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

40 participants in 1 patient group

Trihexyphenidyl

Experimental group

Description:

Participants receive trihexyphenidyl following the dose escalation schedule below: Week 1: 0.05 mg/kg BID Week 2: 0.05 mg/kg TID Week 3: 0.1 mg/kg TID Week 4: 0.15 mg/kg TID Week 5: 0.20 mg/kg TID Week 6-15: 0.25 mg/kg TID

Treatment:

Drug: Trihexyphenidyl