search
Back to results

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

Primary Purpose

Dystonia

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
trihexyphenidyl
Sponsored by
University of Southern California
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dystonia focused on measuring childhood, cerebral palsy, dystonia, secondary, trihexyphenidyl, pediatric dystonia

Eligibility Criteria

5 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Dystonia in the dominant upper extremity Exclusion Criteria: Complete absence of voluntary movement in the affected hands, wrists, and elbows Severe weakness in the dominant upper extremity (MRC grade < 4) Passive range of motion at the hand, wrist or elbow less than 80% of normal Current use of medications for dystonia (anticholinergics, L-dopa, baclofen, diazepam, tizanidine, tetrabenazine, reserpine, and others) Changes in the subject's physical therapy regimen for the duration of the 15-week study Prior use of trihexyphenidyl or other anticholinergic therapy for dystonia. History of surgery on the dominant upper extremity or cervical spine Botulinum toxin injection in the dominant upper extremity within the previous 6 months Current or prior implantation of an intrathecal baclofen pump, deep brain stimulator, or other device to treat dystonia or spasticity Concurrent acute or chronic medical condition (such as frequent seizures, heart disease, or asthma) that could adversely affect motor performance or the safety of testing Presence of diurnal fluctuations or other clinical signs and symptoms suggesting an inborn error of metabolism, a family history of dystonia suggesting a genetic dystonia, or dystonia due to injury after the neonatal period (including toxin exposure, trauma, or medication-induced) History of allergic or adverse reaction to trihexyphenidyl or other anticholinergic medications Current complaint of urinary retention requiring treatment. History of glaucoma, or family history of glaucoma with onset before age 40

Sites / Locations

  • University of Alabama School of Medicine
  • Stanford University
  • Rehabilitation Institute of Chicago
  • Kennedy Krieger Institute
  • Washington University School of Medicine
  • University of Rochester Medical Center
  • Texas Scottish Rite Hospital for Children

Outcomes

Primary Outcome Measures

Melbourne assessment of upper extremity function

Secondary Outcome Measures

Barry-Albright Dystonia Scale
Burke-Fahn-Marsden Dystonia Scale
Pediatric Outcomes Data Collection Instrument
Pediatric Quality of Life
Gross Motor Function Measure

Full Information

First Posted
July 18, 2005
Last Updated
May 21, 2014
Sponsor
University of Southern California
Collaborators
United Cerebral Palsy Foundation, Don and Linda Carter Foundation, Crowley Carter Foundation
search

1. Study Identification

Unique Protocol Identification Number
NCT00122044
Brief Title
Childhood Hypertonia of Central Origin: A Trial of Anticholinergic Treatment Effects
Official Title
Childhood Hypertonia of Central Origin: An Open Label Trial of Anticholinergic Treatment Effects
Study Type
Interventional

2. Study Status

Record Verification Date
May 2014
Overall Recruitment Status
Completed
Study Start Date
January 2003 (undefined)
Primary Completion Date
December 2004 (Actual)
Study Completion Date
December 2004 (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Southern California
Collaborators
United Cerebral Palsy Foundation, Don and Linda Carter Foundation, Crowley Carter Foundation

4. Oversight

5. Study Description

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dystonia
Keywords
childhood, cerebral palsy, dystonia, secondary, trihexyphenidyl, pediatric dystonia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
35 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
trihexyphenidyl
Primary Outcome Measure Information:
Title
Melbourne assessment of upper extremity function
Secondary Outcome Measure Information:
Title
Barry-Albright Dystonia Scale
Title
Burke-Fahn-Marsden Dystonia Scale
Title
Pediatric Outcomes Data Collection Instrument
Title
Pediatric Quality of Life
Title
Gross Motor Function Measure

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Dystonia in the dominant upper extremity Exclusion Criteria: Complete absence of voluntary movement in the affected hands, wrists, and elbows Severe weakness in the dominant upper extremity (MRC grade < 4) Passive range of motion at the hand, wrist or elbow less than 80% of normal Current use of medications for dystonia (anticholinergics, L-dopa, baclofen, diazepam, tizanidine, tetrabenazine, reserpine, and others) Changes in the subject's physical therapy regimen for the duration of the 15-week study Prior use of trihexyphenidyl or other anticholinergic therapy for dystonia. History of surgery on the dominant upper extremity or cervical spine Botulinum toxin injection in the dominant upper extremity within the previous 6 months Current or prior implantation of an intrathecal baclofen pump, deep brain stimulator, or other device to treat dystonia or spasticity Concurrent acute or chronic medical condition (such as frequent seizures, heart disease, or asthma) that could adversely affect motor performance or the safety of testing Presence of diurnal fluctuations or other clinical signs and symptoms suggesting an inborn error of metabolism, a family history of dystonia suggesting a genetic dystonia, or dystonia due to injury after the neonatal period (including toxin exposure, trauma, or medication-induced) History of allergic or adverse reaction to trihexyphenidyl or other anticholinergic medications Current complaint of urinary retention requiring treatment. History of glaucoma, or family history of glaucoma with onset before age 40
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Terence D Sanger, MD, PhD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Alabama School of Medicine
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305-5235
Country
United States
Facility Name
Rehabilitation Institute of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Washington University School of Medicine
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
University of Rochester Medical Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Texas Scottish Rite Hospital for Children
City
Dallas
State/Province
Texas
ZIP/Postal Code
75219
Country
United States

12. IPD Sharing Statement

Links:
URL
http://www.kidsmove.org
Description
Information on childhood dystonia and other movement disorders

Learn more about this trial

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

We'll reach out to this number within 24 hrs