Cerebral PalsyPhase 1/2NCT00238641
"DOES MYOBLOC™ IMPROVE FUNCTIONAL HAND USE IN YOUNG CHILDREN WITH A HYPERTONIC UPPER EXTREMITY?"
A Phase 1/2 study testing Botulinum Toxin type B in people with Cerebral Palsy, run by Stanford University at 1 site in United States.
Trial summary
In children with cerebral palsy, stiffness of the arm can develop early and delay or prevent the acquisition of normal hand skills. Improvement in functional use of the hand may therefore be dependent upon early treatment of upper extremity hypertonia. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Botulinum Toxin type B
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2002to May 2005
- Phase
- Phase 1/2
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 1. Age 2- 17 years 2. Hypertonia affecting one or both upper extremities 3. Hypertonia caused by static encephalopathy due to prenatal or perinatal injury 4. Cognitive and motor function sufficient so that the child will reliably reach toward a target or a small toy 5. Concurrent enrollment in "standard of care" physical therapy or occupational therapy services
Who cannot join
- 1. Injection of neuromuscular blocking agents to any extremity within six months prior to enrollment 2. Dose changes of trihexyphenidyl, dantrolene, baclofen, dopaminergic agents or benzodiazepines during the course of the study.
- 3. Any use of aminoglycoside antibiotics during the study. 4. Prior surgery to either upper extremity 5. Initiation or change in physical or occupational therapy regimen within 3 months of study entry 6. Progressive or neurodegenerative disease, or suspicion of an inborn error of metabolism 7. Neuropathy, myopathy, or neuromuscular junction disease 8. Congenital deformities of either upper extremity 9. Respiratory or airway compromise, or use of respiratory medications 10. Cardiomyopathy 11. Any other medical condition that would place the child at risk for participation
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University
Stanford, California, 94305-5235
About this study
In children with cerebral palsy, stiffness of the arm can develop early and delay or prevent the acquisition of normal hand skills. Improvement in functional use of the hand may therefore be dependent upon early treatment of upper extremity hypertonia. We propose to test a series of injections of Myobloc™ in a non-randomized one-way crossover pilot clinical trial and dose-finding study, with clinical assessments and blinded video evaluations. Ten children age 2-17 years with increased tone at the elbow or wrist will be expected to complete the study. A 1-month baseline evaluation period will be followed by an injection of low-dose Myobloc™ to affected muscle(s) of the arm according to standardized per-kilogram dosing with a maximum of 25U/kg in each affected arm. Three months later, a second injection of up to 50U/kg will be performed in each affected arm. Three months after the second dose, a third dose of up to 100U/kg will be performed in each affected arm. Neurological assessments will be performed at study entry, prior to each injection, and at 1 and 3 months following each injection. Routine physical therapy and non-study medications will be continued during the study.
Read the detailed description
Subjects: 10 subjects age 2-17 years with hypertonia affecting one or both upper extremities, but without injections of neuromuscular blocking agents for six months prior to enrollment, or surgery on the upper extremity.
Intervention: Injection of Myobloc™ in 2 sites per muscle for up to two muscles of each upper extremity (biceps, brachioradialis), at a maximum dose of 25Units/kg/arm for the first injection, a maximum of 50Units/kg/arm for the second injection, and a maximum of 100Units/kg/arm for the third injection. If there is significant weakness or worsening of function following any injection, the dose will not be increased for subsequent injections. Injections will be performed after placement of topical anesthetic (ELA-MAX™ cream), using EMG guidance to identify active muscles contributing to hypertonia.
Primary outcome measures: The primary outcome measure is the time it takes to complete maximum arm extension during voluntary reaching, measured from the coraco-acromial joint to the midpoint of the dorsum of the wrist. Change scores will be calculated between intake and 1 month (baseline effect), 1 month and 2 months (first injection effect), 4 months and 5 months (second injection effect), and 7 months and 8 months (third injection effect). A device called a Shape Tape (Measureand, Inc.) will be used to measure this outcome. Shape Tape is a flexible strip of portable spring steel with optic fiber that provides instantaneous readouts to a portable computer of bends and twists and other forms of movement capture that can be conducted in a clinical setting. The Shape Tape is fastened loosely to the body part under measurement (in this case, the subject's wrist and shoulder) with either medical-grade adhesive tape or Velcro. The shapetape is connected to a portable computer, and custom software allows measurement of the average velocity of hand movement during reaching.
Secondary outcome measures: The rater will compare improvement or worsening in global arm function through the Unified Dystonia Rating Scale (UDRS), the motor subscale of the Unified Parkinson's Disease rating scale (UPDRS), and the Burke-Fahn-Marsden dystonia scale (BFM). Comparisons will be performed between intake and 1 month (baseline effect), 1 month and 2 months (first injection effect), 4 months and 5 months (second injection effect), and 7 months and 8 months (third injection effect). Other secondary measures include neurological examination, Ashworth spasticity scale, and comparisons of numerical stiffness measures as measured by the Rigidity Analyzer Device. A parent rating scale, the Pediatric Quality of Life Inventory (PedsQL), will be used to determine overall functional improvement.
Statistical analysis: The outcomes will be tested at the 0.05 level of significance using repeated-measures analysis of variance (ANOVA) and the Friedman nonparametric test applied to the 4 repeated change scores for each of the primary and secondary outcome measures. Tests of significance for the secondary measures will be corrected for multiple comparisons.
- Conditions
- Cerebral Palsy
- Being tested
- Botulinum Toxin type B
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Botulinum Toxin type BDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome measure is the time it takes to complete maximum arm extension during voluntary reaching, measured from the coraco-acromial joint to the midpoint of the dorsum of the wrist.
Secondary outcomes (1)
The rater will compare improvement or worsening in global arm function through the Unified Dystonia Rating Scale (UDRS), the motor subscale of the Unified Parkinson's Disease rating scale (UPDRS), and the Burke-Fahn-Marsden dystonia scale (BFM). Compar
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2005 (estimated)
- Study startJan 2002
- Study completionMay 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Elan Pharmaceuticals
- Investigators
- Terence Sanger, MD, PhD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00238641
- Sponsor's protocol ID
- Myobloc2002
- Status last verified
- Oct 2005
- First submitted
- 11 Oct 2005
- Last updated
- 13 Oct 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- "DOES MYOBLOC™ IMPROVE FUNCTIONAL HAND USE IN YOUNG CHILDREN WITH A HYPERTONIC UPPER EXTREMITY?"
- ClinicalTrials.gov ID
- NCT00238641
- Acronym / sponsor ID
- Myobloc2002
- Conditions
- Cerebral Palsy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Elan Pharmaceuticals
- Study start
- Jan 2002
- Study completion
- May 2005
- First posted
- 13 Oct 2005 (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.
In children with cerebral palsy, stiffness of the arm can develop early and delay or prevent the acquisition of normal hand skills. Improvement in functional use of the hand may therefore be dependent upon early treatment of upper extremity hypertonia. We propose to test a series of injections of Myobloc™ in a non-randomized one-way crossover pilot clinical trial and dose-finding study, with clinical assessments and blinded video evaluations. Ten children age 2-17 years with increased tone at the elbow or wrist will be expected to complete the study. A 1-month baseline evaluation period will be followed by an injection of low-dose Myobloc™ to affected muscle(s) of the arm according to standardized per-kilogram dosing with a maximum of 25U/kg in each affected arm. Three months later, a second injection of up to 50U/kg will be performed in each affected arm. Three months after the second dose, a third dose of up to 100U/kg will be performed in each affected arm. Neurological assessments will be performed at study entry, prior to each injection, and at 1 and 3 months following each injection. Routine physical therapy and non-study medications will be continued during the study.
Detailed description
Subjects: 10 subjects age 2-17 years with hypertonia affecting one or both upper extremities, but without injections of neuromuscular blocking agents for six months prior to enrollment, or surgery on the upper extremity.
Intervention: Injection of Myobloc™ in 2 sites per muscle for up to two muscles of each upper extremity (biceps, brachioradialis), at a maximum dose of 25Units/kg/arm for the first injection, a maximum of 50Units/kg/arm for the second injection, and a maximum of 100Units/kg/arm for the third injection. If there is significant weakness or worsening of function following any injection, the dose will not be increased for subsequent injections. Injections will be performed after placement of topical anesthetic (ELA-MAX™ cream), using EMG guidance to identify active muscles contributing to hypertonia.
Primary outcome measures: The primary outcome measure is the time it takes to complete maximum arm extension during voluntary reaching, measured from the coraco-acromial joint to the midpoint of the dorsum of the wrist. Change scores will be calculated between intake and 1 month (baseline effect), 1 month and 2 months (first injection effect), 4 months and 5 months (second injection effect), and 7 months and 8 months (third injection effect). A device called a Shape Tape (Measureand, Inc.) will be used to measure this outcome. Shape Tape is a flexible strip of portable spring steel with optic fiber that provides instantaneous readouts to a portable computer of bends and twists and other forms of movement capture that can be conducted in a clinical setting. The Shape Tape is fastened loosely to the body part under measurement (in this case, the subject's wrist and shoulder) with either medical-grade adhesive tape or Velcro. The shapetape is connected to a portable computer, and custom software allows measurement of the average velocity of hand movement during reaching.
Secondary outcome measures: The rater will compare improvement or worsening in global arm function through the Unified Dystonia Rating Scale (UDRS), the motor subscale of the Unified Parkinson's Disease rating scale (UPDRS), and the Burke-Fahn-Marsden dystonia scale (BFM). Comparisons will be performed between intake and 1 month (baseline effect), 1 month and 2 months (first injection effect), 4 months and 5 months (second injection effect), and 7 months and 8 months (third injection effect). Other secondary measures include neurological examination, Ashworth spasticity scale, and comparisons of numerical stiffness measures as measured by the Rigidity Analyzer Device. A parent rating scale, the Pediatric Quality of Life Inventory (PedsQL), will be used to determine overall functional improvement.
Statistical analysis: The outcomes will be tested at the 0.05 level of significance using repeated-measures analysis of variance (ANOVA) and the Friedman nonparametric test applied to the 4 repeated change scores for each of the primary and secondary outcome measures. Tests of significance for the secondary measures will be corrected for multiple comparisons.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- 1. Age 2- 17 years 2. Hypertonia affecting one or both upper extremities 3. Hypertonia caused by static encephalopathy due to prenatal or perinatal injury 4. Cognitive and motor function sufficient so that the child will reliably reach toward a target or a small toy 5. Concurrent enrollment in "standard of care" physical therapy or occupational therapy services
Key exclusion criteria (2 listed)
- 1. Injection of neuromuscular blocking agents to any extremity within six months prior to enrollment 2. Dose changes of trihexyphenidyl, dantrolene, baclofen, dopaminergic agents or benzodiazepines during the course of the study.
- 3. Any use of aminoglycoside antibiotics during the study. 4. Prior surgery to either upper extremity 5. Initiation or change in physical or occupational therapy regimen within 3 months of study entry 6. Progressive or neurodegenerative disease, or suspicion of an inborn error of metabolism 7. Neuropathy, myopathy, or neuromuscular junction disease 8. Congenital deformities of either upper extremity 9. Respiratory or airway compromise, or use of respiratory medications 10. Cardiomyopathy 11. Any other medical condition that would place the child at risk for participation
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 |
|---|---|---|
| Botulinum Toxin type B | Drug | – |
Primary outcome
The primary outcome measure is the time it takes to complete maximum arm extension during voluntary reaching, measured from the coraco-acromial joint to the midpoint of the dorsum of the wrist.
1 secondary outcome
The rater will compare improvement or worsening in global arm function through the Unified Dystonia Rating Scale (UDRS), the motor subscale of the Unified Parkinson's Disease rating scale (UPDRS), and the Burke-Fahn-Marsden dystonia scale (BFM). Compar
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2005.
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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.
Terence Sanger, MD, PhD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Botulinum Toxin type B in people with Cerebral Palsy, run by Stanford University at 1 site in United States.
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 1 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: finished, as of the sponsor's last update on 13 Oct 2005. 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 United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary outcome measure is the time it takes to complete maximum arm extension during voluntary reaching, measured from the coraco-acromial joint to the midpoint of the dorsum of the wrist.. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Oct 2005.