Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response in Children With Dystonic Cerebral Palsy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Trihexyphenidyl.
- Who it may be relevant to
- Registry conditions: Pediatric Disorder, Genetic Predisposition, Dystonia, Secondary, Dystonia. Basic parameters: 5 years — 17 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Pharmacogenomic Contribution to the Biotransformation of Trihexyphenidyl and Development of a Precision Dosing Model for Children With Dystonia and Cerebral Palsy
Overview
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.
Interventions
- Drug Trihexyphenidyl
6-week dose escalation up to 0.25mg/kg TID, followed by a 9-week maintenance period at this dose
Primary outcome measures
- 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]
Secondary outcome measures (12)
- Number of participants with at least one adverse event as measured by the Safety Monitoring Uniform Report Form (SMURF) [Time frame: Through study completion, an average of 2 years]
- Change from baseline in dystonia duration as measured by the Dyskinesia Impairment Scale, Version 2 (DIS-2) (exploratory) [Time frame: Baseline, 16 weeks]
- Change from baseline in dystonia amplitude as measured by the Dyskinesia Impairment Scale (exploratory) [Time frame: Baseline, 16 weeks]
- Change from baseline in dystonia as measured by the Quality of Upper Extremity Skills Test (QUEST) (exploratory) [Time frame: Baseline, 16 weeks]
- Change in functional impact from baseline as measured by the Dyskinetic Cerebral Palsy Functional Impact Scale (D-FIS) (exploratory) [Time frame: Baseline, 16 weeks]
- Change in priority scores in functional impact from baseline as measured by the Dyskinetic Cerebral Palsy Functional Impact Scale (D-FIS) (exploratory) [Time frame: Baseline, 16 weeks]
- Change in patient-driven performance from baseline as measured by the Canadian Occupational Performance Measure (exploratory) [Time frame: Baseline, 16 weeks]
- Change in patient-driven goal satisfaction from baseline as measured by the Canadian Occupational Performance Measure (exploratory) [Time frame: Baseline, 16 weeks]
- Change in caregiver's perspective about their child in 4 domains: health status, comfort, wellbeing, functional abilities, and ease of caregiving from baseline as measured by Caregiver Priorities and Child Health Index of Life with Disabilities [Time frame: Baseline, 16 weeks]
- Measure the acceptability of outcome measures at 16 weeks as measured by the Acceptability of Intervention Measure [Time frame: 16 weeks]
- Change in disease severity from baseline as measured by the Patient Global Impression of Severity (PGI-S) (exploratory) [Time frame: Baseline and 16 weeks]
- Change in overall status as measured by the Patient Global Impression of Change (PGI-C) [Time frame: Week 16]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Children's Mercy Hospital Kansas City — Kansas City
Identifiers
NCT: NCT06554288 · STUDY00003228