Childhood Hypertonia of Central Origin: An Open Label Trial of Anticholinergic Treatment Effects
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 35
- 试验地点
- 14
- 主要终点
- Melbourne assessment of upper extremity function
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Dystonia in the dominant upper extremity
排除标准
- •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
结局指标
主要结局
Melbourne assessment of upper extremity function
次要结局
- Barry-Albright Dystonia Scale
- Burke-Fahn-Marsden Dystonia Scale
- Pediatric Outcomes Data Collection Instrument
- Pediatric Quality of Life
- Gross Motor Function Measure
研究者
Terence Sanger
Associate Professor
University of Southern California