Dystonic Cerebral PalsyPhase 4NCT01361373
Dopamine Treatment in Children With Cerebral Palsy With Dystonia- A Double Blind Controlled Study
A Phase 4 study testing L- DOPA in people with Dystonic Cerebral Palsy, run by Shaare Zedek Medical Center at 1 site in Israel.
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. Read more about this study
- Who is it for?
- Ages 4 to 18 years
- Conditions the study is for
- Dystonic Cerebral Palsy
- What is being tested?
- L- DOPA
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 1 siteIsrael
- Study dates
- May 2010to Aug 2012
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- clear pathophysiological cause for CP
- disabling dystonia in upper limbs
Who cannot join
- significant contractures
- psychiatric disorder
Trial locations and contacts
1 site in 1 country.
Hilla Ben- Pazi, MD contact
Hilla Ben- Pazi contact
Israel1 site, 1 recruiting
- Recruiting
Shaare Zedek Medical Center
Jerusalem
Hilla Ben- Pazi, MD, Benpazi@gmail.com
About this study
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
Read more
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
- Being tested
- L- DOPA, placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L- DOPADrug
Sinemet up to 10 mg/kg/ day increasing gradually for 2 weeks
placeboDrug
placebo for 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| DOPAMINE | Active comparator | Sinemet up to 10 mg/kg/day Receives: Drug: L- DOPA |
| Placebo | Placebo comparator | placebo Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 years
Secondary outcomes (1)
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
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2011 (estimated)
- Study startMay 2010
- Primary completionMay 2012 (estimated)
- Study completionAug 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Dystonic Cerebral PalsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shaare Zedek Medical Center
- Sponsor type
- University, hospital or other
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01361373
- Sponsor's protocol ID
- -L- Dopa-Cerebral Palsy
- Status last verified
- May 2011
- First submitted
- 6 Jun 2010
- Last updated
- 27 May 2011
- Results posted
- No
Publications
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.
PubMed 23349519
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dopamine Treatment in Children With Cerebral Palsy With Dystonia- A Double Blind Controlled Study
- ClinicalTrials.gov ID
- NCT01361373
- Acronym / sponsor ID
- -L- Dopa-Cerebral Palsy
- Conditions
- Dystonic Cerebral Palsy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Shaare Zedek Medical CenterUniversity, hospital or other
- Study start
- May 2010
- Primary completion
- May 2012 (estimated)
- Study completion
- Aug 2012 (estimated)
- First posted
- 26 May 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| DOPAMINE | Active comparator | Drug: L- DOPASinemet up to 10 mg/kg/day |
| Placebo | Placebo comparator | Drug: placeboplacebo |
Who may qualify
- Ages
- 4 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- clear pathophysiological cause for CP
- disabling dystonia in upper limbs
Key exclusion criteria (2 listed)
- significant contractures
- psychiatric disorder
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| L- DOPASinemet up to 10 mg/kg/ day increasing gradually for 2 weeks | Drug | DOPAMINE |
| placeboplacebo for 2 weeks | Drug | Placebo |
Primary outcome
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.
1 secondary outcome
box and blocks, 9 hole pegs, pronation/ supination, finger sequencing
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2011.
- Derived from this study: PubMed 23349519Pozin 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hilla Ben- Pazi, MD contact
Hilla Ben- Pazi contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing L- DOPA in people with Dystonic Cerebral Palsy, run by Shaare Zedek Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 27 May 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Hilla Ben- Pazi, MD (Benpazi@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
QUEST quality of upper extremity skills test score before and after treatment (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01361373, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 May 2011.