Dopamine Treatment in Children With Cerebral Palsy With Dystonia- A Double Blind Controlled Study

Dystonic Cerebral Palsy Clinical Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01361373
• Other study ID #: -L- Dopa-Cerebral Palsy
• Status: Recruiting
• Phase: Phase 4
• First received: June 6, 2010
• Last updated: May 26, 2011
• Start date: May 2010
• Est. completion date: August 2012

Study information

• Verified date: May 2011
• Source: Shaare Zedek Medical Center
• Contact: Hilla Ben- Pazi, MD
• Email: Benpazi[@]gmail.com
• Is FDA regulated: No
• Health authority: Israel: Minstry of health
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: August 2012
• Est. primary completion date: May 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 4 Years to 18 Years

Eligibility

Inclusion Criteria:

• clear pathophysiological cause for CP

• disabling dystonia in upper limbs

Exclusion Criteria:

• significant contractures

• psychiatric disorder

Study Design

Allocation: Randomized, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cerebral Palsy
• Dystonic Cerebral Palsy

Intervention

Drug:
• L- DOPA
Sinemet up to 10 mg/kg/ day increasing gradually for 2 weeks
• placebo
placebo for 2 weeks

Locations

Country	Name	City	State
Israel	Shaare Zedek Medical Center	Jerusalem

Sponsors (1)

Lead Sponsor	Collaborator
Shaare Zedek Medical Center

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	2 years	No
Secondary	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	2 years	No