Clinical trial NCT00549471: Improvement After Botulinum Toxin Injections to the Arms in Children With Cerebral Palsy
Official title: Improvement After Botulinum Toxin A Injections to the Upper Extremities in Children With Cerebral Palsy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Cerebral Palsy
Keywords: children with cerebral palsy ages eight to eleven.
Lead sponsor: Shaare Zedek Medical Center
Sponsor class: OTHER
First posted: 2007-10-25
Enrollment: 3
Sex: ALL
Minimum age (years): 8
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Hypothesis: (1) Treating upper limb hypertonia/spasticity with Botox®, in addition to reducing hypertonia/spasticity, will (a) improve global development, (b) improve function (passive \& active), (c) reduce carer burden and (d) improve quality of life. (2) Early treatment with Botox® will have a greater impact on the rate of global development when compared to late treatment.

Detailed description: Background: Upper limb function is essential for activities of daily living impacting on quality of life in children with cerebral palsy (CP). In preschool children, dysfunctional upper extremity manipulation not only leads to disability but may further delay global development and substantially increase career burden. Even modest functional improvement could have tremendous long-term benefit in activities of daily living and significantly reduce career burden. Hypertonia is the main symptom causing motor dysfunction in CP. Intramuscular Botulinum toxin injection is one way of treatment. In spite of anecdotal evidence suggesting that early intervention can lead to better outcomes, Israeli physicians are unable to prescribe this treatment for the upper extremities due to limited health insurance coverage. A paucity research evidence is often cited as the reason for limiting the insurance coverage, in particular to the upper limb. We therefore propose to study the effects of Botox® in treating children with CP.

Method: Twenty cooperative quadriplegic CP children ages 8-11 years, gross motor function level 4, will be enrolled for the study. Inclusion criteria will be troublesome hypertonia that will respond to treatment with Botox® (as identified by clinical assessment and neurophysiological measures). Since cooperation is crucial for the intensive therapy children with cognitive impairment (IQ\<70) or severe behavioural disorders will be excluded. The children will be randomized to one of two groups a Botox group (BG) and a control group (CG). CG children will undergo a program of intensive therapy and BG children will be given Botox, as clinically required, in addition to an equivalent program of intensive therapy. Botox injection will be tailored according to the specific child. Generally injection site will include biceps and brachioradialis , while flexors of the wrist and digits will be injected according to abnormal postures during function. Maximal total dose will be 23 IU per kg. The intensive therapy will be as clinically required and the therapy program will be fully documented.

Outcome measures will include the following:

1. Hypertonia- neurophysiological measures
2. Impairment measures - Grip and Pinch strength, active and passive range of motion at the writs elbow and shoulder
3. Upper extremity function - Quality of Upper Extremity Skills Test (QUEST), Box and Blocks test
4. Function and patient needs assessment - Goal Attainment Scores, Developmental Fine Motor Scale, Pediatric Evaluation and Disability Inventory (PEDI)
5. Quality of life scales (care and comfort hypertonicity questionnaire) All of these measures will be taken once before treatment and then repeated at 7 months, and at 13 months after treatment.

Eligibility criteria: Inclusion Criteria:

* cooperative quadriplegic CP children
* gross motor function level 4
* troublesome hypertonia that will respond to treatment with Botox

Exclusion Criteria:

* cognitive impairment (IQ\<70)
* severe behavioural disorder

Primary outcomes:
measure: Quality of life
timeFrame: 2 years
Secondary outcomes:
measure: 1. Hypertonia 2. Impairment measures 3. Upper extremity function 4. Function and patient needs assessment
timeFrame: 2 years
Other outcomes:
Locations:
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
Study officials:
affiliation: Shaare Zedek Medical Center
name: Hilla Ben-Pazi, MD
role: PRINCIPAL_INVESTIGATOR
References: