Trihexyphenidyl for Cerebral Palsy

(TRIKE2 Trial)

This trial tests a medicine called trihexyphenidyl to evaluate its effects in children with dystonic cerebral palsy. Researchers aim to understand how the body processes this medicine and whether genetics influence its effects. This information will guide the design of future studies. Children aged 5 to 17 with cerebral palsy and movement issues (dystonia) that impact daily life may be suitable candidates. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

The trial information does not specify whether you need to stop taking your current medications. Please consult with the study team for guidance.

Research has shown that trihexyphenidyl can cause side effects. Some people report restlessness, constipation, dry mouth, and trouble sleeping. However, this medicine is already approved for other uses, indicating it has been studied for safety in people. Although this trial is in an early stage, doctors will closely monitor any safety concerns. Participants should always consult their doctor if they have any worries.¹²³⁴⁵

Most treatments for cerebral palsy focus on managing symptoms through therapies like physical therapy, muscle relaxants, or botox injections to reduce muscle tightness. But trihexyphenidyl offers a different approach by targeting muscle stiffness and tremors with its anticholinergic effects. Researchers are excited about trihexyphenidyl because it modulates the action of neurotransmitters, potentially offering improved control over muscle movements. This could provide a new option for symptom management, especially for those who don't respond well to current treatments.

Research on trihexyphenidyl for dystonic cerebral palsy has shown mixed results. Some studies have found that trihexyphenidyl did not reduce dystonia, which involves muscle contractions causing twisting and repetitive movements, nor did it improve arm function in children with cerebral palsy. However, another study reported significant improvements in dystonia, motor skills, and development in children using trihexyphenidyl. Additionally, combining trihexyphenidyl with clonazepam proved more effective than using trihexyphenidyl alone for treating dystonic cerebral palsy. These findings suggest that while trihexyphenidyl alone might not always work, it may be more effective when combined with other treatments. Participants in this trial will receive trihexyphenidyl following a dose escalation schedule to evaluate its effectiveness as a standalone treatment.¹²⁴⁶⁷