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

July 17, 2025 updated by: Rose Gelineau-Morel, Children's Mercy Hospital Kansas City

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
40

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

  • Name: Rachel Nass
  • Phone Number: 8166011354
  • Email: rnass@cmh.edu

Study Locations

  • United States
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Children's Mercy Hospital Kansas City
        • Contact:
        • Principal Investigator:
          • Rose Gelineau-Morel, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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
Drug: Trihexyphenidyl
6-week dose escalation up to 0.25mg/kg TID, followed by a 9-week maintenance period at this dose

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Difference in Cmax between CYP2D6 and CYP2C19 phenotype groups
Time Frame: Baseline
Cmax will be measured in first-dose pharmacokinetic study on study day 1
Baseline
Difference in AUC0-n between CYP2D6 and CYP2C19 phenotype groups
Time Frame: Baseline
AUC0-n will be measured in first-dose pharmacokinetic study on study day 1
Baseline
Difference in AUC0-∞ between CYP2D6 and CYP2C19 phenotype groups
Time Frame: Baseline
AUC0-∞ will be measured in first-dose pharmacokinetic study on study day 1
Baseline
Recruitment percentage
Time Frame: Through study completion, an average of 2 years
Measure percent of participants who were approached for the study that enrolled in the study
Through study completion, an average of 2 years
Retention percentage
Time Frame: Through study completion, an average of 2 years
Measure percent of participants enrolled who completed the study
Through study completion, an average of 2 years
Dystonia Efficacy Measures Outcome Completion
Time Frame: Through study completion, an average of 2 years
Measure percent of participants enrolled who were able to complete each dystonia efficacy measure (see secondary outcome measures)
Through study completion, an average of 2 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
Adverse events will only include those that are determined to be related to the study drug.
Through study completion, an average of 2 years
Change from baseline in dystonia duration as measured by the Dyskinesia Impairment Scale (exploratory)
Time Frame: Baseline, 16 weeks

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%).
Baseline, 16 weeks
Change from baseline in dystonia amplitude as measured by the Dyskinesia Impairment Scale (exploratory)
Time Frame: Baseline, 16 weeks

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%).
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
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.
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
Functional impact scores are measured on a 5-point scale from 0 to 4 with 0 being dyskinesia may be present but has no impact on the named activity, and 4 being dyskinesia is present and prevents child from doing a named activity, even with help. An "NA" option indicates the activity is difficult but NOT due to dyskinesia.
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
Priority scores are measured on a 4-point scale from 1 to 4 with 1 being an activity is not a priority and 4 being an activity is highest priority.
Baseline, 16 weeks
Change in patient-driven performance from baseline as measured by the Canadian Occupational Performance Measure (exploratory)
Time Frame: Baseline, 16 weeks
Performance scores are measured on a 10-point scale with 1 being not able to do an activity at all and 10 being able to do an activity extremely well.
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
Satisfaction scores are measured on a 10-point scale with 1 being not satisfied at all with the way they do an activity to 10 being extremely satisfied with the way they do an activity.
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
Scores for each domain and for the total survey are standardized and range from 0 to 100 with 0 being the worst and 100 being the best.
Baseline, 16 weeks
Measure the acceptability of outcome measures at 16 weeks as measured by the Acceptability of Intervention Measure
Time Frame: 16 weeks
Scores are measured on a 5-point scale from Completely Disagree to Completely Agree for items 1) The outcome measure meets my approval. 2) The outcome measure is appealing to me. 3) I like the outcome measure. 4) I welcome the outcome measure
16 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Children's Mercy Hospital Kansas City

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
University of Kansas Medical Center

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Rose Gelineau-Morel, MD, Children's Mercy Kansas City

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 15, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 30, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 29, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 13, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 15, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 18, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 17, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • STUDY00003228

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Pharmacokinetic data, including genotypes and THP levels, will be in publications for analysis replication. This will include participant genotypes and corresponding levels of THP, R-THP, and S-THP. Raw data and technical duplicates are available on request for result transparency and clinical trial design. CYP2D6 and CYP2C19 data will also be published; raw sequences go to NICHD's Data Hub (https://dash.nichd.nih.gov/), new haplotypes to PharmVar (https://www.pharmvar.org/). Recruitment, retention, and scientific methods outcomes will be published to guide future THP trials, though the study isn't powered for efficacy endpoints.

IPD Sharing Time Frame

The data will be made available at the time of the associated publication or at the end of the study period, whichever comes first. The datasets made available through publications in PubMed will be made available indefinitely. Sharing of raw data upon request will be accommodated for at least 10 years. Once genetic data are published or uploaded to DASH or PharmVar, they will be available indefinitely.

IPD Sharing Access Criteria

Access to raw pharmacokinetic data/samples and genetic samples will be controlled because it will only be available upon request. For DASH, a request for study data can be submitted and managed within their system.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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