PREDICT-ITB: Predicting Response in Children With Dystonic Cerebral Palsy to Intrathecal Baclofen

Status: Recruiting
Location: See location...
Intervention Type: Other
Study Type: Interventional
Study Phase: Not Applicable
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 (e.g. 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.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: f
View:

• Child must be large enough to have an ITB pump implanted (typically \

⁃ 18 kg/4 years old)

• They have a diagnosis of CP

• Presence of dystonia verified with a documented Hypertonia Assessment Tool (HAT) and they must have a Barry-Albright Dystonia Scale (BADS) score \>15

• They have been identified by a physician for ITB treatment for tone management and the family/child have agreed to proceed with implantation; note: an ITB trial dose is not necessary to qualify

• Child and family are willing to participate in full schedule of formal assessments, spanning pre-implantation, every 2 weeks until ideal intrathecal baclofen pump dosing is reached, and follow-up assessments

Locations
United States
Texas
Texas Childrens Hospital
RECRUITING
Houston
Contact Information
Primary
Sruthi Thomas, MD, PhD
spthomas@texaschildrens.org
832-824-4945
Backup
Rachel Arp
rearp@texaschildrens.org
(936) 267-7707
Time Frame
Start Date: 2024-11-01
Estimated Completion Date: 2029-08-31
Participants
Target number of participants: 65
Treatments
Other: Titration protocol
This is a single arm study where each subject will be started with a standardized ITB protocol. The dosing will be increased or decreased stepwise based on parameters that have been described in detail in the intervention section.
Sponsors
Leads: Baylor College of Medicine
Collaborators: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

This content was sourced from clinicaltrials.gov