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.
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
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
Single group
Trihexyphenidyl
Drug6-week dose escalation up to 0.25mg/kg TID, followed by a 9-week maintenance period at this dose
Difference in Cmax between CYP2D6 and CYP2C19 phenotype groups
Cmax will be measured in first-dose pharmacokinetic study on study day 1
Difference in AUC0-n between CYP2D6 and CYP2C19 phenotype groups
AUC0-n will be measured in first-dose pharmacokinetic study on study day 1
Difference in AUC0-∞ between CYP2D6 and CYP2C19 phenotype groups
AUC0-∞ will be measured in first-dose pharmacokinetic study on study day 1
Recruitment percentage
Measure percent of participants who were approached for the study that enrolled in the study
Number of participants with at least one adverse event as measured by the Safety Monitoring Uniform Report Form (SMURF)
Adverse events will only include those that are determined to be related to the study drug.
Change from baseline in dystonia duration as measured by the Dyskinesia Impairment Scale, Version 2 (DIS-2) (exploratory)
The Dyskinesia Impairment Scale, Version 2 (DIS-2) is an updated version of the Dyskinesia Impairment Scale used to assess dystonia across multiple body regions. Duration of dystonia using subscale DIS-D are measured in 12 body regions during activity and rest. Dystonia duration scores are measured on a 4-point scale from 0 to 4 with 0 being dystonia is absent and 4 being dystonia is always present (≥90%).
Change from baseline in dystonia amplitude as measured by the Dyskinesia Impairment Scale (exploratory)
Amplitude of dystonia using subscale DIS-D are measured in 12 body regions during activity and rest. Dystonia amplitude scores are measured on a 4-point scale from 0 to 4 with 0 being dystonia is absent and 4 being dystonia in maximal range of motion (≥90%).
Change from baseline in dystonia as measured by the Quality of Upper Extremity Skills Test (QUEST) (exploratory)
Upper extremity function in 1 domain; grasp. 13 activities with item-level scores and three items for the tester to rate: hand function, spasticity, and cooperativeness. All scores are summed, and formulas are used to calculate percentages for this domain. Domain percentage is summed with a minimum score less than 0, and the maximum score is 100.
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Children's Mercy Hospital Kansas City
Lead sponsor
University of Kansas Medical Center
Collaborator
