Childhood Hypertonia of Central Origin: A Trial of Anticholinergic Treatment Effects
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Dystonia |
Drug: trihexyphenidyl |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Childhood Hypertonia of Central Origin: An Open Label Trial of Anticholinergic Treatment Effects |
- Melbourne assessment of upper extremity function
- Barry-Albright Dystonia Scale
- Burke-Fahn-Marsden Dystonia Scale
- Pediatric Outcomes Data Collection Instrument
- Pediatric Quality of Life
- Gross Motor Function Measure
| Estimated Enrollment: | 35 |
| Study Start Date: | January 2003 |
| Estimated Study Completion Date: | December 2004 |
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.
Eligibility| Ages Eligible for Study: | 5 Years to 17 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations| United States, Alabama | |
| University of Alabama School of Medicine | |
| Birmingham, Alabama, United States, 35233 | |
| United States, California | |
| Stanford University | |
| Stanford, California, United States, 94305-5235 | |
| United States, Illinois | |
| Rehabilitation Institute of Chicago | |
| Chicago, Illinois, United States, 60611 | |
| United States, Maryland | |
| Kennedy Krieger Institute | |
| Baltimore, Maryland, United States, 21205 | |
| United States, Missouri | |
| Washington University School of Medicine | |
| St. Louis, Missouri, United States, 63110 | |
| United States, New York | |
| University of Rochester Medical Center | |
| Rochester, New York, United States, 14642 | |
| United States, Texas | |
| Texas Scottish Rite Hospital for Children | |
| Dallas, Texas, United States, 75219 | |
| Principal Investigator: | Terence D Sanger, MD, PhD | Stanford University |
More Information
Additional Information:
No publications provided
| ClinicalTrials.gov Identifier: | NCT00122044 History of Changes |
| Other Study ID Numbers: | CHOCOLATE |
| Study First Received: | July 18, 2005 |
| Last Updated: | August 15, 2005 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by Stanford University:
|
childhood cerebral palsy dystonia |
secondary trihexyphenidyl pediatric dystonia |
Additional relevant MeSH terms:
|
Dystonia Dystonic Disorders Muscle Hypertonia Dyskinesias Neurologic Manifestations Nervous System Diseases Signs and Symptoms Movement Disorders Central Nervous System Diseases Neuromuscular Manifestations Trihexyphenidyl |
Cholinergic Antagonists Antiparkinson Agents Anti-Dyskinesia Agents Central Nervous System Agents Therapeutic Uses Pharmacologic Actions Muscarinic Antagonists Cholinergic Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs |
ClinicalTrials.gov processed this record on May 19, 2013