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

NCT ID: NCT01361373

Last Updated: 2011-05-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-05-31

Study Completion Date

2012-08-31

Brief Summary

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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.

Detailed Description

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Conditions

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Dystonic Cerebral Palsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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DOPAMINE

Sinemet up to 10 mg/kg/day

Group Type ACTIVE_COMPARATOR

L- DOPA

Intervention Type DRUG

Sinemet up to 10 mg/kg/ day increasing gradually for 2 weeks

Placebo

placebo

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type DRUG

placebo for 2 weeks

Interventions

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L- DOPA

Sinemet up to 10 mg/kg/ day increasing gradually for 2 weeks

Intervention Type DRUG

placebo

placebo for 2 weeks

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* significant contractures
* psychiatric disorder
Minimum Eligible Age

4 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shaare Zedek Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Shaare Zedek Medical Center

Locations

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Shaare Zedek Medical Center

Jerusalem, , Israel

Site Status RECRUITING

Countries

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Israel

Central Contacts

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Hilla Ben- Pazi, MD

Role: CONTACT

Hilla Ben- Pazi

Role: CONTACT

Facility Contacts

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Hilla Ben- Pazi, MD

Role: primary

References

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Pozin I, Bdolah-Abram T, Ben-Pazi H. Levodopa does not improve function in individuals with dystonic cerebral palsy. J Child Neurol. 2014 Apr;29(4):534-7. doi: 10.1177/0883073812473645. Epub 2013 Jan 24.

Reference Type DERIVED
PMID: 23349519 (View on PubMed)

Other Identifiers

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-L- Dopa-Cerebral Palsy

Identifier Type: -

Identifier Source: org_study_id

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