Intrathecal Baclofen and Pediatric Dystonia

Part of paid clinical trials in Houston, Texas.

Sponsor
Baylor College of Medicine
Study ID
NCT06606574
Status
Recruiting
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Conditions

  • Dystonic Cerebral Palsy

Eligibility Criteria

Sex
ALL
Age
N/A - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • Titration protocol — OTHER
    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.

Study Details

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.

Key Dates

Start date
Nov 1, 2024
Status verified
Jul 2025
Primary completion
Jun 30, 2029
Completion
Aug 31, 2029

Study Design

Enrollment
65 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • 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.

Primary Outcome Measure

Barry-Albright Dystonia Scale (BADS) [ Time Frame: Baseline (pre-ITB initiation) , 3 months, 6 months, 12 months post ITB initiation ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Texas Childrens HospitalHoustonTexas77030
Principal Investigator, MD, PhD
[email protected]
832-824-4945

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By research site
Texas Childrens Hospital· Houston, TX

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