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Intrathecal Baclofen and Pediatric Dystonia

NCT06606574 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 65

Last updated 2025-07-30

No results posted yet for this study

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.

Conditions

  • Dystonic Cerebral Palsy

Interventions

OTHER

Titration protocol

A standardized titration protocol for ITB will be used and the ITB dose will be increased to the next step if 1) there is persistent hypertonia identified on the dystonia screener or ASAS total spasticity, or 2) there is room for improvement on the D-FIS, and 3) there are no detrimental side effects (e.g., worsening head control). This will be continued until 1) dystonia and spasticity have been eliminated or no longer deemed as functionally impairing by the medical team, 2) side effects prevent further titration, or 3) a maximum dose of 2000 mcg/day has been reached. Once steady state is reached, this new dose becomes the maintenance dose for the rest of the trial. If there are detrimental side effects, the child will be seen weekly to confirm resolution of side effects. If side effects persist, the child will be reduced at each visit to the previous step. Once side effects are controlled, this new dose becomes the child's maintenance dose for the rest of the trial.

Sponsors & Collaborators

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

    collaborator NIH

  • Baylor College of Medicine

    lead OTHER

Principal Investigators

  • Sruthi P Thomas, MD, PhD · Baylor College of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-01
Primary Completion
2029-06-30
Completion
2029-08-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06606574 on ClinicalTrials.gov