Dopamine Treatment in Children With Cerebral Palsy With Dystonia- A Double Blind Controlled Study
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|ClinicalTrials.gov Identifier: NCT01361373|
Recruitment Status : Unknown
Verified May 2011 by Shaare Zedek Medical Center.
Recruitment status was: Recruiting
First Posted : May 26, 2011
Last Update Posted : May 27, 2011
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
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.
No functional improvement with dopamine treatment compared to placebo.
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.
|Condition or disease||Intervention/treatment||Phase|
|Dystonic Cerebral Palsy||Drug: L- DOPA Drug: placebo||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Study Start Date :||May 2010|
|Estimated Primary Completion Date :||May 2012|
|Estimated Study Completion Date :||August 2012|
U.S. FDA Resources
Active Comparator: DOPAMINE
Sinemet up to 10 mg/kg/day
Drug: L- DOPA
Sinemet up to 10 mg/kg/ day increasing gradually for 2 weeks
Placebo Comparator: Placebo
placebo for 2 weeks
- QUEST quality of upper extremity skills test score before and after treatment [ Time Frame: 2 years ]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.
- box and blocks, 9 hole pegs, pronation/ supination, finger sequencing [ Time Frame: 2 years ]
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01361373
|Shaare Zedek Medical Center||Recruiting|
|Contact: Hilla Ben- Pazi, MD Benpazi@gmail.com|