Cerebral PalsyCPBiopsyBTXNCT05497609
The Muscle in Children With Cerebral Palsy - Longitudinal Exploration of Microscopic Muscle Structure.
An observational study of Cerebral Palsy and Muscle Contraction, run by Eva Ponten at 1 site in Sweden.
Trial summary
Cerebral palsy (CP) is a motor impairment due to a brain malformation or a brain lesion before the age of two. Spasticity, hypertonus in flexor muscles, dyscoordination and an impaired sensorimotor control are cardinal symptoms. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Cerebral Palsy, Muscle Contraction, Muscle Spasticity
- How many people
- 50 participants
- Where
- 1 siteSweden
- Study dates
- 15 Jan 2006to 15 Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
Study population: Children with cerebral palsy and aquired brain injury who, as a part of care as usual, gets botulinum toxin injections in muscles. Children are recruited consecutively.
Who may be able to join
- Cerebral Palsy, Aquired Brain Injury
Who cannot join
- Progressive neural disease
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Alexandra Palmcrantz contact
Sweden1 site, 1 recruiting
- Recruiting
Karolinska University Hospital
Stockholm, 17176
Eva M Pontén, MD PhD, 0706303052, eva.ponten@ki.se
Alexandra Palmcrantz, PT, alexandra.palmcrantz@ki.se
About this study
Official title: The Muscle in Children Cerebral Palsy - Longitudinal Exploration of Microscopic Muscle Structure in Gastrocnemius and Biceps Brachii During Two Years of Care as Usual, Including Training With Botulinum Toxin Injections as Adjuvant Therapy.
Cerebral palsy (CP) is a motor impairment due to a brain malformation or a brain lesion before the age of two. Spasticity, hypertonus in flexor muscles, dyscoordination and an impaired sensorimotor control are cardinal symptoms. The brain lesion is non-progressive, but the flexor muscles of the limbs will during adolescence become relatively shorter and shorter (contracted), forcing the joints into a progressively flexed position. This will worsen the positions of already paretic and malfunctioning arms and legs. Due to bending forces across the joints, bony malformations will occur, worsening the function even further. Since about 25 years a combination treatment with intramuscular botulinum toxin injections, braces and training has had a tremendous and increasing popularity, although lasting long-term clinical advantage is not yet proven.
Muscle morphology of the biceps brachii and the gastrocnemius muscles:
- The hypothesis is that care as usual, i.e. training and splinting sessions with botulinum toxin as adjuvant treatment, will reduce (normalize) the expression of the fast fatigable myosin heavy chain MyHC IIx and increase the expression of developmental myosin, as a possible sign of growth. As the biceps in the arm is used irregularly and voluntarily, and the gastrocnemius is activated during automated gait, the adaptations of those muscles will be different. Methods: Baseline muscle biopsies: Percutaneous biopsies are taken just before the first intramuscular botulinum toxin injection is given. The doses and the intervals for the botulinum toxin treatment will follow clinical routines. Biopsies 4-6 months, 12 months and 24 months after the first botulinum toxin injection: The exact same procedure as above will be performed, but the biopsies will be taken 2 cm distant, medial or lateral, from previous biopsy sites
- Significance:. More knowledge is warranted regarding the actual molecular process in the muscle leading to a contracture, and its relation to the constant communication with the injured central nervous system. This study will give answers that could result in new, early prophylactic treatment of joint movement restrictions and motor impairment in children with CP.
Read the detailed description
Cerebral palsy (CP) is a motor impairment due to a brain malformation or a brain lesion before the age of two. Spasticity, hypertonus in flexor muscles, dyscoordination and an impaired sensorimotor control are cardinal symptoms. The brain lesion is non-progressive, but the flexor muscles of the limbs will during adolescence become relatively shorter and shorter (contracted), forcing the joints into a progressively flexed position. This will worsen the positions of already paretic and malfunctioning arms and legs. Due to bending forces across the joints, bony malformations will occur, worsening the function even further. Currently, the initial treatment of choice is the use of braces, which diminishes the shortening somewhat. Since about 25 years a combination treatment with intramuscular botulinum toxin injections, braces and training has had a tremendous and increasing popularity, although lasting long-term clinical advantage is not yet proven.
Muscle morphology of the biceps brachii and the gastrocnemius muscles:
- The hypothesis is that care as usual, i.e. training and splinting sessions with botulinum toxin as adjuvant treatment, will reduce (normalize) the expression of the fast fatigable myosin heavy chain MyHC IIx and increase the expression of developmental myosin, as a possible sign of growth. As the biceps in the arm is used irregularly and voluntarily, and the gastrocnemius is activated during automated gait, the adaptations of those muscles will be different. Methods: Baseline muscle biopsies: Percutaneous biopsies from the biceps brachii and the gastrocnemius muscles are taken just before the first intramuscular botulinum toxin injection is given. The doses and the intervals for the botulinum toxin treatment will follow clinical routines. Training of the leg and arm will after the injections be performed with the help of physiotherapists and occupational therapists. Biopsies 4-6 months, 12 months and 24 months after the first botulinum toxin injection: The exact same procedure as above will be performed, but the biopsies will be taken 2 cm distant, medial or lateral, from previous biopsy sites. The muscle specimens are snap frozen and stored at -80°C until analyzed. The expression of different myosin heavy chain (MyHC) isoforms is assessed by using the monoclonal antibodies (mAb) N2.261, mAb A4.840 against slow MyHC I, mAb F1.652 against embryonic MyHC, and mAb NCL-MHCn against fetal (=neonatal) MyHC)(Tiger, Champliaud et al. 1997; Wewer, Thornell et al. 1997). Satellite cells will be identified with mAb against N-CAM (neural cell adhesion molecule). The fibers are typed according to the content of MyHCs.
Significance: Children with cerebral palsy have a motor impairment and progressive contractures that we often treat late; when tendon and bony surgery are the only options to realign the joints. Our aim is to treat the muscles early, so that the contractures and the bony malformations won't occur in the first place. Training and splints, with botulinum toxin as adjuvant treatment, is a very popular regime with this aim, but the long-term effect on muscle tissue and function is not yet known. This study will elucidate the effect during a 2-year period, and no such studies have yet been published. More knowledge is warranted regarding the actual molecular process in the muscle leading to a contracture, and its relation to the constant communication with the injured central nervous system. This study will give answers that could result in new, early prophylactic treatment of joint movement restrictions and motor impairment in children with CP.
- Conditions
- Cerebral Palsy, Muscle Contraction, Muscle Spasticity
- Being tested
- no intervention. Care as ususal
- Keywords
- Cerebral Palsy, Upper Limb, Lower Limb, Muscle Biopsy, Botulinum Toxin Type A
- 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.
no intervention. Care as ususalOther
No intervention
What is being measured
The primary outcome is the main question the study is designed to answer.
Muscle fiber area
um2, square micrometers, measured on specimens
Measured: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
Muscle fiber types based on myosin heavy chain isoforms
Percentage of all fibers
Measured: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
Extracellular matrix, area
um2, square micrometers, measured on specimens
Measured: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
Capillaries per fiber area
number of capillaries/um2
Measured: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
Mitochondria: NADH staining, morphology
Scoring 1, 2, 3
Measured: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
Secondary outcomes (1)
Clinical assessment, contracture
°, degrees
Measured: At 0 months, 6 months, 12 months and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2022
- Study start15 Jan 2006
- Primary completion15 Dec 2026 (estimated)
- Study completion15 Dec 2027 (estimated)
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.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eva Ponten
- Sponsor type
- Other government
- Responsible party
- Sponsor-investigator, Eva Ponten, Region Stockholm
- Investigators
- Eva M Pontén, MD PhD, Principal investigator, Karolinska Institutet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05497609
- Sponsor's protocol ID
- BTX muskel CP
- Acronym
- CPBiopsyBTX
- Status last verified
- Jul 2022
- First submitted
- 27 Jul 2022
- Last updated
- 11 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Muscle in Children Cerebral Palsy - Longitudinal Exploration of Microscopic Muscle Structure in Gastrocnemius and Biceps Brachii During Two Years of Care as Usual, Including Training With Botulinum Toxin Injections as Adjuvant Therapy.
- ClinicalTrials.gov ID
- NCT05497609
- Acronym / sponsor ID
- CPBiopsyBTX / BTX muskel CP
- Conditions
- Cerebral Palsy, Muscle Contraction, Muscle Spasticity
- Keywords
- Cerebral Palsy, Upper Limb, Lower Limb, Muscle Biopsy, Botulinum Toxin Type A
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Eva PontenOther government
- Study start
- 15 Jan 2006 (actual)
- Primary completion
- 15 Dec 2026 (estimated)
- Study completion
- 15 Dec 2027 (estimated)
- First posted
- 11 Aug 2022 (actual)
- 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 motor impairment due to a brain malformation or a brain lesion before the age of two. Spasticity, hypertonus in flexor muscles, dyscoordination and an impaired sensorimotor control are cardinal symptoms. The brain lesion is non-progressive, but the flexor muscles of the limbs will during adolescence become relatively shorter and shorter (contracted), forcing the joints into a progressively flexed position. This will worsen the positions of already paretic and malfunctioning arms and legs. Due to bending forces across the joints, bony malformations will occur, worsening the function even further. Since about 25 years a combination treatment with intramuscular botulinum toxin injections, braces and training has had a tremendous and increasing popularity, although lasting long-term clinical advantage is not yet proven.
Muscle morphology of the biceps brachii and the gastrocnemius muscles:
- The hypothesis is that care as usual, i.e. training and splinting sessions with botulinum toxin as adjuvant treatment, will reduce (normalize) the expression of the fast fatigable myosin heavy chain MyHC IIx and increase the expression of developmental myosin, as a possible sign of growth. As the biceps in the arm is used irregularly and voluntarily, and the gastrocnemius is activated during automated gait, the adaptations of those muscles will be different. Methods: Baseline muscle biopsies: Percutaneous biopsies are taken just before the first intramuscular botulinum toxin injection is given. The doses and the intervals for the botulinum toxin treatment will follow clinical routines. Biopsies 4-6 months, 12 months and 24 months after the first botulinum toxin injection: The exact same procedure as above will be performed, but the biopsies will be taken 2 cm distant, medial or lateral, from previous biopsy sites
- Significance:. More knowledge is warranted regarding the actual molecular process in the muscle leading to a contracture, and its relation to the constant communication with the injured central nervous system. This study will give answers that could result in new, early prophylactic treatment of joint movement restrictions and motor impairment in children with CP.
Detailed description
Cerebral palsy (CP) is a motor impairment due to a brain malformation or a brain lesion before the age of two. Spasticity, hypertonus in flexor muscles, dyscoordination and an impaired sensorimotor control are cardinal symptoms. The brain lesion is non-progressive, but the flexor muscles of the limbs will during adolescence become relatively shorter and shorter (contracted), forcing the joints into a progressively flexed position. This will worsen the positions of already paretic and malfunctioning arms and legs. Due to bending forces across the joints, bony malformations will occur, worsening the function even further. Currently, the initial treatment of choice is the use of braces, which diminishes the shortening somewhat. Since about 25 years a combination treatment with intramuscular botulinum toxin injections, braces and training has had a tremendous and increasing popularity, although lasting long-term clinical advantage is not yet proven.
Muscle morphology of the biceps brachii and the gastrocnemius muscles:
- The hypothesis is that care as usual, i.e. training and splinting sessions with botulinum toxin as adjuvant treatment, will reduce (normalize) the expression of the fast fatigable myosin heavy chain MyHC IIx and increase the expression of developmental myosin, as a possible sign of growth. As the biceps in the arm is used irregularly and voluntarily, and the gastrocnemius is activated during automated gait, the adaptations of those muscles will be different. Methods: Baseline muscle biopsies: Percutaneous biopsies from the biceps brachii and the gastrocnemius muscles are taken just before the first intramuscular botulinum toxin injection is given. The doses and the intervals for the botulinum toxin treatment will follow clinical routines. Training of the leg and arm will after the injections be performed with the help of physiotherapists and occupational therapists. Biopsies 4-6 months, 12 months and 24 months after the first botulinum toxin injection: The exact same procedure as above will be performed, but the biopsies will be taken 2 cm distant, medial or lateral, from previous biopsy sites. The muscle specimens are snap frozen and stored at -80°C until analyzed. The expression of different myosin heavy chain (MyHC) isoforms is assessed by using the monoclonal antibodies (mAb) N2.261, mAb A4.840 against slow MyHC I, mAb F1.652 against embryonic MyHC, and mAb NCL-MHCn against fetal (=neonatal) MyHC)(Tiger, Champliaud et al. 1997; Wewer, Thornell et al. 1997). Satellite cells will be identified with mAb against N-CAM (neural cell adhesion molecule). The fibers are typed according to the content of MyHCs.
Significance: Children with cerebral palsy have a motor impairment and progressive contractures that we often treat late; when tendon and bony surgery are the only options to realign the joints. Our aim is to treat the muscles early, so that the contractures and the bony malformations won't occur in the first place. Training and splints, with botulinum toxin as adjuvant treatment, is a very popular regime with this aim, but the long-term effect on muscle tissue and function is not yet known. This study will elucidate the effect during a 2-year period, and no such studies have yet been published. More knowledge is warranted regarding the actual molecular process in the muscle leading to a contracture, and its relation to the constant communication with the injured central nervous system. This study will give answers that could result in new, early prophylactic treatment of joint movement restrictions and motor impairment in children with CP.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children with cerebral palsy and aquired brain injury who, as a part of care as usual, gets botulinum toxin injections in muscles. Children are recruited consecutively.
Key inclusion criteria (1 listed)
- Cerebral Palsy, Aquired Brain Injury
Key exclusion criteria (1 listed)
- Progressive neural disease
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 |
|---|---|---|
| no intervention. Care as ususalNo intervention | Other | – |
Primary outcome
Muscle fiber area
Time frame: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
um2, square micrometers, measured on specimens
Muscle fiber types based on myosin heavy chain isoforms
Time frame: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
Percentage of all fibers
Extracellular matrix, area
Time frame: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
um2, square micrometers, measured on specimens
Capillaries per fiber area
Time frame: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
number of capillaries/um2
Mitochondria: NADH staining, morphology
Time frame: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215
Scoring 1, 2, 3
1 secondary outcome
Clinical assessment, contracture
Time frame: At 0 months, 6 months, 12 months and 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2022.
- Individual participant dataSharing planned: Undecided
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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.
Alexandra Palmcrantz contact
Eva M Pontén, MD PhD principal investigator
Karolinska Institutet
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cerebral Palsy and Muscle Contraction, run by Eva Ponten at 1 site in Sweden.
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 1 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: looking for participants now, as of the sponsor's last update on 11 Aug 2022. 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 Sweden. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Eva M Pontén, MD PhD (+46706303052, eva.ponten@ki.se), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Muscle fiber area (time frame: Biopsies are collected and stored in -80° freezer and then analyzed. Analyses will take place up to 20271215). The study lists 5 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 11 Aug 2022.