Ignite Creation Date:
2024-05-06 @ 6:53 PM
Last Modification Date:
2024-10-26 @ 2:56 PM
Study NCT ID:
NCT05817409
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-04-18
First Post:
2023-03-21
Brief Title:
Predictive Factors for Succes of ITB in CP
Sponsor:
Amsterdam UMC location VUmc
Organization:
Amsterdam UMC location VUmc
Study Overview
Official Title:
Predictive Factors for Treatment Success of Intrathecal Baclofen Therapy in Children With Cerebral Palsy - The Netherlands CP Register as a Unique Model for Practice Based Long Term Data From Children With Cerebral Palsy
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
For children with severe cerebral palsy CP in whom other options are insufficient intrathecal baclofen ITB treatment could be an option Literature provides some evidence for the effect of ITB however we notice that not all children benefit equally Furthermore long term effects of ITB in a prospective cohort of individuals with CP have not yet been described in literature To improve selection for ITB physicians need to know which factors influence outcome on the long term In order to accomplish these goals prospectively real life data over a long period of time and from a large cohort are needed
The investigators will therefore conduct a prospective observational cohort study using the Netherlands CP register to collect data over a period of 45 years The Netherlands CP register is an innovative register for data collection which was developed between 2016 and 2020 in a unique collaboration between children with CP their parents researchers and health care professionals
All children and adolescents with CP who start ITB treatment in the Netherlands under the age of 16 diagnosed with spastic or dyskinetic CP are eligible for inclusion in the study
The primary outcome measure is goal attainment scaling GAS in which achievement of individual goals can be quantified Factors that potentially influence outcome including spasticity dystonia scoliosis and complications will be secondary outcome measures
Measurements will be done before initiation of ITB after 3 months after 1 year and after 2 years
Statistical analysis will include effect size Cohens d for all outcome measures To assess which factors are possibly associated with GAS T-score outcome at follow-up multiple regression analysis will be conducted
The investigators will therefore conduct a prospective observational cohort study using the Netherlands CP register to collect data over a period of 45 years The Netherlands CP register is an innovative register for data collection which was developed between 2016 and 2020 in a unique collaboration between children with CP their parents researchers and health care professionals
All children and adolescents with CP who start ITB treatment in the Netherlands under the age of 16 diagnosed with spastic or dyskinetic CP are eligible for inclusion in the study
The primary outcome measure is goal attainment scaling GAS in which achievement of individual goals can be quantified Factors that potentially influence outcome including spasticity dystonia scoliosis and complications will be secondary outcome measures
Measurements will be done before initiation of ITB after 3 months after 1 year and after 2 years
Statistical analysis will include effect size Cohens d for all outcome measures To assess which factors are possibly associated with GAS T-score outcome at follow-up multiple regression analysis will be conducted
Detailed Description:
Backgroundrational
Cerebral palsy CP is the most common cause of childhood disability with a prevalence of 211 per 1000 live births in high income countries CP enfolds a group of developmental disorders of movement and posture attributed to non-progressive disturbances which occurred in the developing foetal or infant brain
An advanced treatment option for individuals with severe CP is Intrathecal Baclofen ITB treatment This treatment can be considered when other options such as oral medication are insufficient or cause side effects Previous studies have shown mostly low level evidence for the effect of ITB on spasticity in children with spastic CP For children with dyskinetic CP The Amsterdam UMC and Maastricht UMC previously conducted a randomized placebo-controlled trial which provides evidence for the effect of ITB on attaining personal treatment goals after three months of ITB treatment It was furthermore found that after one year of ITB treatment personal treatment goals were at least partially achieved in 97 of children and adolescents
Not all children show similar response to ITB some benefit more than others in attaining treatment goals The investigators previous research on the effect of ITB in children with dyskinetic CP found that a higher level of dystonia prior to ITB and reduction of pain during ITB were associated for a small part with attainment of treatment goals Sample size was not sufficient to look at specific factors associated with specific types of treatment goals For example goals on the level of personal care could be influenced by other factors than goals related to sitting or transfers
Long-term effects of ITB in a prospective cohort of individuals with spastic and dyskinetic CP have not yet been described in literature Furthermore knowing which factors influence outcome on the long term preferably related to specific types of treatment goals can be used to inform and select children and adolescents with CP better before initiating ITB In order to accomplish these goals prospectively real life data over a long period of time and from a large cohort are needed
The Netherlands CP register is an innovative register for data collection which was developed between 2016 and 2020 in a unique collaboration between children with CP their parents researchers and health care professionals The overall aim of the register is to provide personalized value-based care for children with CP In the Netherlands CP register a follow-up registration program is combined with treatment registration
Prospective data collection in the treatment registration will allow us to implement standardised assessment protocols in the two academic medical centres in the Netherlands where baclofen pump implantations are performed the Amsterdam University Medical Center Amsterdam UMC in Amsterdam and Maastricht UMC MUMCin Maastricht
Objectives
The primary research question of this study is
1 What is the short- 3 months medium- 1 year and long-term 2 years effect of ITB in children with CP on personal treatment goals
Secondary objectives are
1 What is the association between the effect on personal treatment goals and on spasticity andor dystonia
2 Which factors are associated with treatment success of ITB in children with CP
Study design A prospective observational study will be conducted in Amsterdam UMC and the MUMC using the Netherlands CP register to collect data over a period of 45 years It is anticipated that in these centres 15 to 20 pumps will be implanted in children and adolescents annually Over the total study period of 45 years we aim to include a total of 67 to 90 children
The Medical Ethics Review Committee MEC of the Amsterdam UMC location VU University Medical Center IRB00002991 has stated that the Medical Research Involving Human Subjects Act WMO does not apply to the study and that further official approval by the MEC is waived
Participants Inclusion criteria
All children with CP
Who start ITB treatment in the Netherlands under the age of 16 Exclusion criteria
no consent is given by the patient andor caregiver for participation
Subject follow up schedule In the investigators previous randomised controlled trial the IDYS trial on the effect of ITB in dyskinetic CP we found that after three months of ITB most goals were at least partially attained A study by Albright et al additionally implies that dosage increases mostly in the first months then shows some increase between 12 to 24 months and is fairly stable thereafter Considering this information data are collected and entered in the register at the following time points before initiation of ITB after 3 months after 1 year after 2 years All centres participating in the register and performing ITB pump implantations enter data directly into the CP register Data collection ceases if the patient dies if ITB therapy is terminated or once the patient transitions to adult services at the age of 18
Outcome measures The primary outcome measure in this study is goal attainment scaling GAS in which achievement of individual goals can be quantified Secondary outcome measures include patient characteristics treatment history Range of motion spasticity Dystonia scoliosis selectivity functional strength gait analysis questionnaires on activities and participation treatment characteristics ITB including dosage complications
Statistical methodrationale A linear mixed-model analysis will be used to determine the development over time for the primary and secondary outcome measures using time as a categorical variable All analyses will be adjusted for possible covariates
Effect size ES Cohens d will be calculated for all outcome measures Univariable linear regression analysis for each potential variable influencing outcome will be conducted Factors showing a p-value of 020 are then selected for input in a final model in which a multiple regression analysis using backward stepwise selection will be performed to identify the predicting factors
A p-value 005 is considered statistically significant Correction for multiple testing will be done by Holm correction of p-values
Cerebral palsy CP is the most common cause of childhood disability with a prevalence of 211 per 1000 live births in high income countries CP enfolds a group of developmental disorders of movement and posture attributed to non-progressive disturbances which occurred in the developing foetal or infant brain
An advanced treatment option for individuals with severe CP is Intrathecal Baclofen ITB treatment This treatment can be considered when other options such as oral medication are insufficient or cause side effects Previous studies have shown mostly low level evidence for the effect of ITB on spasticity in children with spastic CP For children with dyskinetic CP The Amsterdam UMC and Maastricht UMC previously conducted a randomized placebo-controlled trial which provides evidence for the effect of ITB on attaining personal treatment goals after three months of ITB treatment It was furthermore found that after one year of ITB treatment personal treatment goals were at least partially achieved in 97 of children and adolescents
Not all children show similar response to ITB some benefit more than others in attaining treatment goals The investigators previous research on the effect of ITB in children with dyskinetic CP found that a higher level of dystonia prior to ITB and reduction of pain during ITB were associated for a small part with attainment of treatment goals Sample size was not sufficient to look at specific factors associated with specific types of treatment goals For example goals on the level of personal care could be influenced by other factors than goals related to sitting or transfers
Long-term effects of ITB in a prospective cohort of individuals with spastic and dyskinetic CP have not yet been described in literature Furthermore knowing which factors influence outcome on the long term preferably related to specific types of treatment goals can be used to inform and select children and adolescents with CP better before initiating ITB In order to accomplish these goals prospectively real life data over a long period of time and from a large cohort are needed
The Netherlands CP register is an innovative register for data collection which was developed between 2016 and 2020 in a unique collaboration between children with CP their parents researchers and health care professionals The overall aim of the register is to provide personalized value-based care for children with CP In the Netherlands CP register a follow-up registration program is combined with treatment registration
Prospective data collection in the treatment registration will allow us to implement standardised assessment protocols in the two academic medical centres in the Netherlands where baclofen pump implantations are performed the Amsterdam University Medical Center Amsterdam UMC in Amsterdam and Maastricht UMC MUMCin Maastricht
Objectives
The primary research question of this study is
1 What is the short- 3 months medium- 1 year and long-term 2 years effect of ITB in children with CP on personal treatment goals
Secondary objectives are
1 What is the association between the effect on personal treatment goals and on spasticity andor dystonia
2 Which factors are associated with treatment success of ITB in children with CP
Study design A prospective observational study will be conducted in Amsterdam UMC and the MUMC using the Netherlands CP register to collect data over a period of 45 years It is anticipated that in these centres 15 to 20 pumps will be implanted in children and adolescents annually Over the total study period of 45 years we aim to include a total of 67 to 90 children
The Medical Ethics Review Committee MEC of the Amsterdam UMC location VU University Medical Center IRB00002991 has stated that the Medical Research Involving Human Subjects Act WMO does not apply to the study and that further official approval by the MEC is waived
Participants Inclusion criteria
All children with CP
Who start ITB treatment in the Netherlands under the age of 16 Exclusion criteria
no consent is given by the patient andor caregiver for participation
Subject follow up schedule In the investigators previous randomised controlled trial the IDYS trial on the effect of ITB in dyskinetic CP we found that after three months of ITB most goals were at least partially attained A study by Albright et al additionally implies that dosage increases mostly in the first months then shows some increase between 12 to 24 months and is fairly stable thereafter Considering this information data are collected and entered in the register at the following time points before initiation of ITB after 3 months after 1 year after 2 years All centres participating in the register and performing ITB pump implantations enter data directly into the CP register Data collection ceases if the patient dies if ITB therapy is terminated or once the patient transitions to adult services at the age of 18
Outcome measures The primary outcome measure in this study is goal attainment scaling GAS in which achievement of individual goals can be quantified Secondary outcome measures include patient characteristics treatment history Range of motion spasticity Dystonia scoliosis selectivity functional strength gait analysis questionnaires on activities and participation treatment characteristics ITB including dosage complications
Statistical methodrationale A linear mixed-model analysis will be used to determine the development over time for the primary and secondary outcome measures using time as a categorical variable All analyses will be adjusted for possible covariates
Effect size ES Cohens d will be calculated for all outcome measures Univariable linear regression analysis for each potential variable influencing outcome will be conducted Factors showing a p-value of 020 are then selected for input in a final model in which a multiple regression analysis using backward stepwise selection will be performed to identify the predicting factors
A p-value 005 is considered statistically significant Correction for multiple testing will be done by Holm correction of p-values
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None