NCT00122044

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

Childhood Hypertonia of Central Origin: An Open Label Trial of Anticholinergic Treatment Effects

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 35 (planned)
• Sponsor: University of Southern California · Academic / Other
• Sex: All
• Age: 5 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This study is an open-label trial of trihexyphenidyl in children with upper extremity dystonia due to cerebral palsy. It is hypothesized that trihexyphenidyl in doses up to 0.75mg/kg/day would be well-tolerated and show significant changes on the Melbourne scale of upper extremity function.

Detailed description

BACKGROUND: Although trihexyphenidyl has been used to treat both primary and secondary dystonia in children, previous studies have not investigated efficacy in secondary dystonia. We describe the results of a prospective, open-label, multi-center trial of high-dose trihexyphenidyl in children with secondary dystonia of the arms due to cerebral palsy. METHODS: Twenty-six children age 4-15 years with cerebral palsy and dystonia that impairs function of the dominant upper extremity were enrolled. All children were given trihexyphenidyl at increasing doses over 9 weeks up to 0.75mg/kg/day. Trihexyphenidyl was subsequently tapered over 5 weeks. Visits occurred at baseline, 9 weeks, and 15 weeks. The primary outcome measure was the Melbourne assessment of upper extremity function, tested in the dominant arm. RESULTS: Three children withdrew due to non-serious adverse events (chorea, drug rash, hyperactivity). 3 children reduced dosage due to non-serious adverse events. The 23 children who completed the study showed a significant improvement in arm function at 15 weeks (p=0.045) but not at 9 weeks. Post-hoc analysis showed that a subgroup (N=10) with hyperkinetic dystonia worsened at 9 weeks (p=0.04) but subsequently returned to baseline following taper of the medicine. CONCLUSIONS: Trihexyphenidyl appears to be safe and effective for treatment of arm dystonia in children with cerebral palsy. Children with hyperkinetic dystonia may worsen. A larger randomized prospective trial is needed to confirm these results.

Conditions

• Dystonia

Interventions

Type	Name	Description
DRUG	trihexyphenidyl

Timeline

• Start date: 2003-01-01
• Primary completion: 2004-12-01
• Completion: 2004-12-01
• First posted: 2005-07-21
• Last updated: 2014-05-23

Locations

7 sites across 1 country: United States