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

NCT01361373 | Unknown Phase 4

• 1 other identifier: -L- Dopa-Cerebral Palsy
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Dystonic Cerebral Palsy

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (1)

• box and blocks, 9 hole pegs, pronation/ supination, finger sequencing

  2 years

Study Arms (2)

DOPAMINE

ACTIVE COMPARATOR

Sinemet up to 10 mg/kg/day

Drug: L- DOPA

Placebo

PLACEBO COMPARATOR

placebo

Drug: placebo

Interventions

L- DOPA DRUG

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

DOPAMINE

placebo DRUG

placebo for 2 weeks

Placebo

Eligibility Criteria

• Age: 4 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• significant contractures
• psychiatric disorder

Sponsors & Collaborators

• Shaare Zedek Medical Center: lead

Study Sites (1)

Shaare Zedek Medical Center

Jerusalem, Israel

RECRUITING

Related Publications (1)

• 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.

  PMID: 23349519 DERIVED

MeSH Terms

Interventions

Dihydroxyphenylalanine

Intervention Hierarchy (Ancestors)

Catecholamines; Amines; Organic Chemicals; Catechols; Phenols; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Tyrosine

Central Study Contacts

Hilla Ben- Pazi, MD

CONTACT

Benpazi@gmail.com

Hilla Ben- Pazi

CONTACT

Benpazi@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: June 6, 2010
• First Posted: May 26, 2011
• Study Start: May 1, 2010
• Primary Completion: May 1, 2012
• Study Completion: August 1, 2012
• Last Updated: May 27, 2011

Record last verified: 2011-05

Locations

IL (1)