Clinical Trial: Childhood Hypertonia of Central Origin: A Trial of Anticholinergic Treatment Effects
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Childhood Hypertonia of Central Origin: An Open Label Trial of Anticholinergic Treatment Effects
Brief 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 Summary:
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.
Sponsor: University of Southern California
Current Primary Outcome: Melbourne assessment of upper extremity function
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Barry-Albright Dystonia Scale
- Burke-Fahn-Marsden Dystonia Scale
- Pediatric Outcomes Data Collection Instrument
- Pediatric Quality of Life
- Gross Motor Function Measure
Original Secondary Outcome: Same as current
Information By: University of Southern California
Dates:
Date Received: July 18, 2005
Date Started: January 2003
Date Completion: December 2004
Last Updated: May 21, 2014
Last Verified: May 2014
