Recruiting
Phase 1

Trihexyphenidyl

Sponsor:

Children's Mercy Hospital Kansas City

Code:

NCT06554288

Conditions

Pediatric Disorder

Genetic Predisposition

Dystonia, Secondary

Dystonia

Cerebral Palsy, Dystonic-Rigid

Eligibility Criteria

Sex: All

Age: 5 - 17

Healthy Volunteers: Not accepted

Interventions

Trihexyphenidyl

Study Details

Brief 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.

Conditions

Pediatric Disorder

Genetic Predisposition

Dystonia, Secondary

Dystonia

Cerebral Palsy, Dystonic-Rigid

Study ID

NCT06554288

Start date

Oct 15, 2024

Status verified date

Jun, 2026

Completion date

Dec 31, 2029

Anticipated

Primary completion date

Nov 30, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 5 - 17

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

40 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Trihexyphenidyl

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

Interventions

Trihexyphenidyl

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

Primary outcome measure

  • Difference in Cmax between CYP2D6 and CYP2C19 phenotype groups [ Time Frame: Baseline ]
  • Difference in AUC0-n between CYP2D6 and CYP2C19 phenotype groups [ Time Frame: Baseline ]
  • Difference in AUC0-∞ between CYP2D6 and CYP2C19 phenotype groups [ Time Frame: Baseline ]
  • Recruitment percentage [ Time Frame: Through study completion, an average of 2 years ]
  • Retention percentage [ Time Frame: Through study completion, an average of 2 years ]
  • Dystonia Efficacy Measures Outcome Completion [ Time Frame: Through study completion, an average of 2 years ]

Central Contacts and Locations

Central contacts

Rose Gelineau-Morel, MD

816-302-3331rngelineaumorel@cmh.edu

Rachel Nass

8166011354rnass@cmh.edu

Locations

Children's Mercy Hospital Kansas City

Recruiting

Kansas City, Missouri, United States, 64108

Contacts

Rachel Nass

816-601-1354rnass@cmh.edu

Principal Investigator:

Rose Gelineau-Morel, MD

More Information

Sponsor

Children's Mercy Hospital Kansas City

Last update posted

Jun 24, 2026

Last verified

Jun, 2026

Keywords

  • Pediatric
  • Cerebral Palsy
  • Dystonia
  • Pharmacogenomics
  • Trihexyphenidyl

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Children's Mercy Hospital Kansas City on 2026-06-24.