A Phase 4 interventional study of L- DOPA and placebo in Dystonic Cerebral Palsy, sponsored by Shaare Zedek Medical Center. Status unknown at 1 site in Israel. Open to participants aged 4 Years to 18 Years. Per ClinicalTrials.gov, last updated 2011-05-27.
Sponsored by Shaare Zedek Medical Center · Phase 4, Interventional, and Treatment
Background:
Cerebral palsy (CP) is the main cause of childhood immobility and is defined as a non progressive injury to the developing central nervous system in children younger than 3 years, resulting in neurological and musculoskeletal abnormalities. The main pathophysiological causes are encephalopathy of prematurity (periventricular leukomalacia) hypoxic ischemic encephalopathy. Infections, infracts and migration defects are other less common causes of CP. The brain injury leads to functional motor impairment impacting on daily activities commonly manifests as a movement disorder: pyramidal, leading to spasticity and extra-pyramidal leading to dystonia and chorea. In most cases extensive brain injury causes a mixed movement disorder. Dystonia is defined as involuntary muscle contractions causing twisting and abnormal postures. While the neurological underpinnings of CP remain unknown, a link between low dopamine and increased acetylcholine release has recently been reported in dystonia. Dopamine is considered the first line of treatment in children with dystonia and CP followed by anticholiergic treatment with trihexphenidyl. The recommendation of dopaminergic treatment is based on need to rule out dopamine-responsive-dystonia, a rare genetic disorder, and on single case study reporting improvement in CP. A double blind study support or refute the use of dopamine treatment for dystonic CP was never reported. Working hypothesis and
Aims:
In children with CP due to a clear underlying pathology, dopamine treatment will not improve daily function. Methods: the investigators will perform a double blinded randomized controlled crossover study. 50 children ages 4-18 years with a clear pathophysiological cause for CP will be enrolled. Each child will receive dopamine and placebo treatment for 2 weeks with a 2 week washout interval. Participants will be randomized into 2 groups; one will receive placebo followed by dopamine and the other vice versa. The primary outcome measure, goal-attainment-scale, and secondary outcome functional measures (such as box and blocks, 9 hole pegs, pronation/ supination, finger sequencing) will be assessed at the beginning and end of each treatment as well as parent questionnaires regarding satisfaction and side effects.
Expected results:
No functional improvement with dopamine treatment compared to placebo.
Importance:
supplying sufficient data to support or refute the use of dopamine treatment for dystonic CP.
Probable implications to Medicine:
this may lead to a change in medical treatment guidelines for children with CP.
1,853 studies on the registry are indexed under Cerebral Palsy; 437 are open to participants now.
This study's planned enrollment of 50 is above the median of 34 across 1,368 interventional studies indexed under Cerebral Palsy.
Browse Cerebral Palsy studies →Shaare Zedek Medical Center is the lead sponsor of 191 studies on the registry; 17 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Sinemet up to 10 mg/kg/day
Drug: L- DOPA
placebo
Drug: placebo
Sinemet up to 10 mg/kg/ day increasing gradually for 2 weeks
placebo for 2 weeks
QUEST quality of upper extremity skills test score before and after treatment
The QUEST is a measure designed to evaluate movement patterns and hand function in children with cerebral palsy. It is administered within a play context. Items are related to quality of movement, not to chronological age. There are 36 items assessing dissociated movements, grasp, protective extension, and weight bearing.
Time frame: 2 years
box and blocks, 9 hole pegs, pronation/ supination, finger sequencing
Difference between test scores before and after treatment. Namely: * of box transfered per minute time to complete the 9 hole pegs insertion. * pronation/ supination per 20 seconds time to complete 5 finger sequencing rounds
Time frame: 2 years
This study is status unknown, as verified in May 2011. You cannot join it, but the record below documents what was studied.
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Shaare Zedek Medical Center