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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-15

Study Completion Date

2029-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Childhood Hypertonia of Central Origin: A Trial of Anticholinergic Treatment Effects

NCT00122044

Dystonia

COMPLETED PHASE2

Intrathecal Baclofen and Pediatric Dystonia

NCT06606574

Dystonic Cerebral Palsy

RECRUITING NA

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

NCT02696044

Rett Syndrome

UNKNOWN PHASE2

Oral Baclofen Pharmacokinetics and Pharmacodynamics in Children With Spasticity

NCT00607542

Spasticity Cerebral Palsy

COMPLETED PHASE1/PHASE2

Side Effects of Bone Marrow Derived CD133 Transplantation in Cerebral Palsy

NCT01404663

Cerebral Palsy

COMPLETED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

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

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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

Group Type EXPERIMENTAL

Trihexyphenidyl

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Trihexyphenidyl

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

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are 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

Minimum Eligible Age

5 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No