Cerebral PalsyTOXIMUS_CPNCT02853240
Effect of Botulinum Toxin on Muscles of Children With Cerebral Palsy
A study testing Muscle biopsy in people with Cerebral Palsy, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Cerebral palsy (CP) is a group of non-progressive motor dysfunction but often changing, secondary to injury or brain abnormalities that occur in early stages of development. In children with CP, the brain injury lead to a delayed motor development in the first weeks, associated with muscular spasticity. Read more about this study
- Who is it for?
- Ages 8 to 17 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Muscle biopsy
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteFrance
- Study dates
- 24 Oct 2016to 10 Jan 2020
- Phase
- Interventional
- 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 8 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age Children > 7 years and <18 years
- With spastic cerebral palsy (all grades of the combined GMFCS
- Having an orthopedic surgical indication already planned on the lower limbs
- Receiving toxin injections
- With social security coverage
- Whose parents / holders of parental authority have signed the consent form
Who cannot join
- Patients with an evolutive CP
- Children with a baclofen pump
- Children who underwent neurotomy or functional dorsal rhizotomy, or alcohol or phenol injections
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Transversal Monocentric Study of the Anatomophysiological Effect of Botulinum Toxin on the Spastic Muscle of Children With Cerebral Palsy
Cerebral palsy (CP) is a group of non-progressive motor dysfunction but often changing, secondary to injury or brain abnormalities that occur in early stages of development. In children with CP, the brain injury lead to a delayed motor development in the first weeks, associated with muscular spasticity. Drug treatments include oral treatments (baclofen and tizanidine) and injectable treatments like Botox (intramuscular injection) and neurolysis with alcohol or phenol (local injection into the nerve).
Regarding botulinum toxin, there is no study questioning its effectiveness. However, no publication on the pathophysiology of human muscle of the CP child after toxin injection was found. The action of the toxin on the neuromuscular junction (NMJ) and muscle structure is unknown in children with CP.
Read more
The primary objective of this study is to describe structural abnormalities of the CP child's muscle following multiple toxin injections in terms of NMJ fragmentation and axonal sprouting.
Secondary objectives:
To evaluate the relationship between:
- The severity of the motor impairment and muscle structural abnormalities.
- The clinical measure of spasticity and muscle structural abnormalities.
- To compare the structure spastic muscles with toxin injections and spastic muscle without toxin injections
For muscles with multiple toxin injections, assessing the relationship between :
- The number of toxin injections and muscle structural abnormalities.
- The date of the first injection and muscle structural abnormalities.
- The total dose of injected toxin in the muscle and its structural abnormalities.
- The nature of the product injected in the muscle and its structural abnormalities.
This innovative study will improve the knowledge on the effects of long-term botulinum toxin injections on the muscle (and therefore its safety in usual care), on the spastic muscle NMJ of CP children, on the pathophysiology of the CP child's muscle.
All the visits all acts will be performed according to usual patient follow-up. Only a biopsy will be performed in addition, taken from an injected muscle during a planned operation. A biopsy may also be performed on a muscle without toxin injection if the act is made possible by the planned surgery. No biopsy will be made on a muscle that would not require surgery.
- Conditions
- Cerebral Palsy
- Being tested
- Muscle biopsy
- Keywords
- Cerebral palsy, children, muscle spasticity, botulinum toxin injection
- Medical subject headings
- Cerebral Palsy, Muscle Spasticity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Muscle biopsyProcedure or surgery
A biopsy (specifically done for the study) of a muscle that has already been injected with botulinum toxin before inclusion of the patient in the study will be performed, taken during a planned surgery (for tendon transfer or muscle lengthening). A biopsy of a muscle that has never been injected with botulinum toxin may be performed during surgery. No biopsy will be made on a member that would not require surgery. Thus sampling will be conducted on a muscle requiring a surgery: 2 to 3 millimeters will be taken on 10 mm muscle before transfer or lengthening.
| Study group | Role | What they receive |
|---|---|---|
| Children with spastic CP receiving toxin injections | Experimental | Children with spastic cerebral palsy receiving toxin injections Receives: Procedure: Muscle biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Presence of neuromuscular junctions fragmentation (both qualitative and quantitative).
The biopsy is performed at the same time of a scheduled general anesthesia surgery (multisite surgery). The biopsy is 2 to 3 mm x 10 mm. It is a simple and quick gesture, usually practiced by surgeon
Measured: 6 months maximum (time of surgery)
Presence of axonal sprouting (qualitative).
Measured: 6 months maximum (time of surgery)
Secondary outcomes (7)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to patient's GMFCS grade (1 to 5).
The Gross Motor Function Classification System (GMFCS) is a 5 level clinical classification system that describes the gross motor function of people with cerebral palsy on the basis of self-initiated movement abilities.
Measured: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to Ashworth score.
The Ashworth scale is bedside tool for assessing muscle spasticity in patients with neurological conditions. Ashworth grades as follows: 0, 1, 1+, 2, 3, 4.
Measured: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to Tardieu score.
The Tardieu Scale is bedside tool for assessing muscle spasticity in patients with neurological conditions. Tardieu scale grades from 0 to 5.
Measured: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to the number of toxin injections in the muscle.
Measured: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to the delay (in months) since the first toxin injection in the muscle
Measured: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to the total volume (IU) of injected toxin since the first injection in the muscle.
Measured: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to the brand of the injected toxin (Botox® or Dysport®).
Measured: 6 months maximum (time of surgery)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2016 (estimated)
- Study start24 Oct 2016
- Primary completion10 Jan 2020
- Study completion10 Jan 2020
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 Hospices Civils de LyonThe 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 N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02853240
- Sponsor's protocol ID
- 69HCL15_0540
- Other IDs
- 2016-001610-24, 2016
- Acronym
- TOXIMUS_CP
- Status last verified
- Jan 2020
- First submitted
- 19 Jul 2016
- Last updated
- 18 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Transversal Monocentric Study of the Anatomophysiological Effect of Botulinum Toxin on the Spastic Muscle of Children With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT02853240
- Acronym / sponsor ID
- TOXIMUS_CP / 69HCL15_0540
- Conditions
- Cerebral Palsy
- Keywords
- Cerebral palsy, children, muscle spasticity, botulinum toxin injection
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 24 Oct 2016 (actual)
- Primary completion
- 10 Jan 2020 (actual)
- Study completion
- 10 Jan 2020 (actual)
- First posted
- 2 Aug 2016 (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.
Cerebral palsy (CP) is a group of non-progressive motor dysfunction but often changing, secondary to injury or brain abnormalities that occur in early stages of development. In children with CP, the brain injury lead to a delayed motor development in the first weeks, associated with muscular spasticity. Drug treatments include oral treatments (baclofen and tizanidine) and injectable treatments like Botox (intramuscular injection) and neurolysis with alcohol or phenol (local injection into the nerve).
Regarding botulinum toxin, there is no study questioning its effectiveness. However, no publication on the pathophysiology of human muscle of the CP child after toxin injection was found. The action of the toxin on the neuromuscular junction (NMJ) and muscle structure is unknown in children with CP.
The primary objective of this study is to describe structural abnormalities of the CP child's muscle following multiple toxin injections in terms of NMJ fragmentation and axonal sprouting.
Secondary objectives:
To evaluate the relationship between:
- The severity of the motor impairment and muscle structural abnormalities.
- The clinical measure of spasticity and muscle structural abnormalities.
- To compare the structure spastic muscles with toxin injections and spastic muscle without toxin injections
For muscles with multiple toxin injections, assessing the relationship between :
- The number of toxin injections and muscle structural abnormalities.
- The date of the first injection and muscle structural abnormalities.
- The total dose of injected toxin in the muscle and its structural abnormalities.
- The nature of the product injected in the muscle and its structural abnormalities.
This innovative study will improve the knowledge on the effects of long-term botulinum toxin injections on the muscle (and therefore its safety in usual care), on the spastic muscle NMJ of CP children, on the pathophysiology of the CP child's muscle.
All the visits all acts will be performed according to usual patient follow-up. Only a biopsy will be performed in addition, taken from an injected muscle during a planned operation. A biopsy may also be performed on a muscle without toxin injection if the act is made possible by the planned surgery. No biopsy will be made on a muscle that would not require surgery.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Children with spastic CP receiving toxin injections | Experimental | Procedure: Muscle biopsyChildren with spastic cerebral palsy receiving toxin injections |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Age Children > 7 years and <18 years
- With spastic cerebral palsy (all grades of the combined GMFCS
- Having an orthopedic surgical indication already planned on the lower limbs
- Receiving toxin injections
- With social security coverage
Key exclusion criteria (3 listed)
- Patients with an evolutive CP
- Children with a baclofen pump
- Children who underwent neurotomy or functional dorsal rhizotomy, or alcohol or phenol injections
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 |
|---|---|---|
| Muscle biopsyA biopsy (specifically done for the study) of a muscle that has already been injected with botulinum toxin before inclusion of the patient in the study will be performed, taken during a planned surgery (for tendon transfer or muscle lengthening). A biopsy of a muscle that has never been injected with botulinum toxin may be performed during surgery. No biopsy will be made on a member that would not require surgery. Thus sampling will be conducted on a muscle requiring a surgery: 2 to 3 millimeters will be taken on 10 mm muscle before transfer or lengthening. | Procedure or surgery | Children with spastic CP receiving toxin injections |
Primary outcome
Presence of neuromuscular junctions fragmentation (both qualitative and quantitative).
Time frame: 6 months maximum (time of surgery)
The biopsy is performed at the same time of a scheduled general anesthesia surgery (multisite surgery). The biopsy is 2 to 3 mm x 10 mm. It is a simple and quick gesture, usually practiced by surgeon
Presence of axonal sprouting (qualitative).
Time frame: 6 months maximum (time of surgery)
7 secondary outcomes
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to patient's GMFCS grade (1 to 5).
Time frame: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to Ashworth score.
Time frame: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to Tardieu score.
Time frame: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to the number of toxin injections in the muscle.
Time frame: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to the delay (in months) since the first toxin injection in the muscle
Time frame: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to the total volume (IU) of injected toxin since the first injection in the muscle.
Time frame: 6 months maximum (time of surgery)
Proportion of muscles with neuromuscular junctions fragmentation (both qualitative and quantitative) and/or axonal sprouting (qualitative) according to the brand of the injected toxin (Botox® or Dysport®).
Time frame: 6 months maximum (time of surgery)
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 18 Jan 2020.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Muscle biopsy in people with Cerebral Palsy, run by Hospices Civils de Lyon at 1 site in France.
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 6 inclusion and 3 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 18 Jan 2020. 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 France. 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.
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?
Presence of neuromuscular junctions fragmentation (both qualitative and quantitative). (time frame: 6 months maximum (time of surgery)). The study lists 2 primary outcomes in all. 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 18 Jan 2020.