Ignite Creation Date:
2024-10-26 @ 3:40 PM
Last Modification Date:
2024-10-26 @ 3:40 PM
Study NCT ID:
NCT06606574
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-09-18
Brief Title:
Intrathecal Baclofen and Pediatric Dystonia
Sponsor:
None
Organization:
None
Study Overview
Official Title:
PREDICT-ITB Predicting Response in Children With Dystonic Cerebral Palsy to Intrathecal Baclofen
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PREDICT-ITB
Brief Summary:
The goal of this clinical trial is to better understand the effects of intrathecal baclofen ITB on children with dystonic cerebral palsy CP
The main questions this study aims to answer are
1 Determine if ITB reduces dystonia while identifying other potential benefits 2 Identify the characteristics of children with the best response to ITB 3 Develop a holistically representative composite outcome measure for dystonic CP
This study will evaluate patient improvement by using a standardized titration or medication management protocol to gradually increase the childs ITB dosages over a 12-month period until they achieve maximum benefit with minimal to no side effects This titration protocol mimics what is currently done through routine care but with more precision This study will also directly measure the global effects of ITB taking into account spasticity known dystonia triggers eg pain and patterns of CNS injury that cause dystonia
Participants will
1 Complete a total of 4 additional clinic visits outside usual care These appointments will be with physical and occupational therapists as well as the study PI to complete evaluations for dystonia spasticity and function
2 Complete several questionnaires at these visits The total duration of the study for an individual child will be 12 months
The main questions this study aims to answer are
1 Determine if ITB reduces dystonia while identifying other potential benefits 2 Identify the characteristics of children with the best response to ITB 3 Develop a holistically representative composite outcome measure for dystonic CP
This study will evaluate patient improvement by using a standardized titration or medication management protocol to gradually increase the childs ITB dosages over a 12-month period until they achieve maximum benefit with minimal to no side effects This titration protocol mimics what is currently done through routine care but with more precision This study will also directly measure the global effects of ITB taking into account spasticity known dystonia triggers eg pain and patterns of CNS injury that cause dystonia
Participants will
1 Complete a total of 4 additional clinic visits outside usual care These appointments will be with physical and occupational therapists as well as the study PI to complete evaluations for dystonia spasticity and function
2 Complete several questionnaires at these visits The total duration of the study for an individual child will be 12 months
Detailed Description:
Cerebral palsy CP is the most common cause of physical disability in childhood and affects 1 in 323 in the US per the CDC The lifetime medical costs for an individual with CP was expected to be 14 million inflation adjusted An effective way to improve quality of life and decrease disease burden both in childhood and adulthood is to significantly reduce or eliminate abnormal tone As muscle tone subsides it is easier to move the body both passively and actively A reduction in tone can improve quality of life reduce pain ease the burden of daily care and performance of day-to-day activities and reduce healthcare costs Intrathecal baclofen ITB is used widely to manage dystonia in CP even though the data on exact efficacy is mixed Therefore the goal of this study is to understand the effects of ITB on a heterogenous population of children with dystonic CP
The goal of the proposed research is to complete a longitudinal prospective cohort study investigating the effects of intrathecal baclofen ITB on children with dystonic cerebral palsy CP This study builds on new preliminary data and capitalizes on the expertise of an established transdisciplinary team a high-resource academic clinical environment and a diverse Parent-Patient Council The studys findings are likely to have high impact to improve future treatment of children with dystonic CP including identifying key patient characteristics associated with more or less positive outcomes and developing a sensitive efficient multidimensional composite measure to assess treatment responsiveness Specifically this protocol aims to determine the 12-month effects of ITB in a cohort of 65 clinically representative ITB naïve children with dystonic CP using a standardized titration protocol Additionally this protocol aims to complete a detailed characterization of brain malformation and injury patterns in children with dystonic CP and explore potential differences in outcome patterns as related to patient characteristics and use findings to guide development of a sensitive multidimensional or composite measure that captures clinically important areas of change for the child and family
Our study site will be Texas Childrens Hospital TCH in Houston TX our PI Sruthi Thomas home institution TCH has over 860 active patients with CP and just over 200 patients with ITB Houston TX is the most diverse city in the United States allowing us to recruit a very diverse patient population based on ethnicity and socioeconomic groups
The goal of the proposed research is to complete a longitudinal prospective cohort study investigating the effects of intrathecal baclofen ITB on children with dystonic cerebral palsy CP This study builds on new preliminary data and capitalizes on the expertise of an established transdisciplinary team a high-resource academic clinical environment and a diverse Parent-Patient Council The studys findings are likely to have high impact to improve future treatment of children with dystonic CP including identifying key patient characteristics associated with more or less positive outcomes and developing a sensitive efficient multidimensional composite measure to assess treatment responsiveness Specifically this protocol aims to determine the 12-month effects of ITB in a cohort of 65 clinically representative ITB naïve children with dystonic CP using a standardized titration protocol Additionally this protocol aims to complete a detailed characterization of brain malformation and injury patterns in children with dystonic CP and explore potential differences in outcome patterns as related to patient characteristics and use findings to guide development of a sensitive multidimensional or composite measure that captures clinically important areas of change for the child and family
Our study site will be Texas Childrens Hospital TCH in Houston TX our PI Sruthi Thomas home institution TCH has over 860 active patients with CP and just over 200 patients with ITB Houston TX is the most diverse city in the United States allowing us to recruit a very diverse patient population based on ethnicity and socioeconomic groups
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None