Trial record 2 of 6 for:    Neuronal Migration Disorders

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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified May 2011 by Shaare Zedek Medical Center.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Shaare Zedek Medical Center
ClinicalTrials.gov Identifier:
NCT01361373
First received: June 6, 2010
Last updated: May 26, 2011
Last verified: May 2011
  Purpose

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.


Condition Intervention Phase
Dystonic Cerebral Palsy
Drug: L- DOPA
Drug: placebo
Phase 4

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

Resource links provided by NLM:


Further study details as provided by Shaare Zedek Medical Center:

Primary Outcome Measures:
  • QUEST quality of upper extremity skills test score before and after treatment [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • box and blocks, 9 hole pegs, pronation/ supination, finger sequencing [ Time Frame: 2 years ] [ Designated as safety issue: No ]

    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


Estimated Enrollment: 50
Study Start Date: May 2010
Estimated Study Completion Date: August 2012
Estimated Primary Completion Date: May 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
Drug: placebo
placebo for 2 weeks

  Eligibility

Ages Eligible for Study:   4 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • clear pathophysiological cause for CP
  • disabling dystonia in upper limbs

Exclusion Criteria:

  • significant contractures
  • psychiatric disorder
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01361373

Contacts
Contact: Hilla Ben- Pazi, MD Benpazi@gmail.com
Contact: Hilla Ben- Pazi Benpazi@gmail.com

Locations
Israel
Shaare Zedek Medical Center Recruiting
Jerusalem, Israel
Contact: Hilla Ben- Pazi, MD       Benpazi@gmail.com   
Sponsors and Collaborators
Shaare Zedek Medical Center
  More Information

No publications provided by Shaare Zedek Medical Center

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Hilla Ben Pazi, MD, Shaare Zedek Medical Center
ClinicalTrials.gov Identifier: NCT01361373     History of Changes
Other Study ID Numbers: -L- Dopa-Cerebral Palsy
Study First Received: June 6, 2010
Last Updated: May 26, 2011
Health Authority: Israel: Minstry of health

Additional relevant MeSH terms:
Cerebral Palsy
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on August 18, 2014