Recruiting

Baclofen

Sponsor:

Baylor College of Medicine

Code:

NCT06606574

Conditions

Dystonic Cerebral Palsy

Eligibility Criteria

Sex: All

Age: 0 - 17

Healthy Volunteers: Not accepted

Interventions

Titration protocol

Study Details

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 (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

Study ID

NCT06606574

Start date

Nov 1, 2024

Status verified date

Jul, 2025

Completion date

Aug 31, 2029

Anticipated

Primary completion date

Jun 30, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 17

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Emergent need for ITB such as status dystonicus or paroxysmal sympathetic hyperactivity
  • Botulinum injections within 3 months or phenol injections within 6 months of enrollment
  • Foster care placement or incarceration

Study Design

Enrollment

65 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

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.

Interventions

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.

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 and Locations

Central contacts

Sruthi Thomas, MD, PhD

832-824-4945spthomas@texaschildrens.org

Rachel Arp

(936) 267-7707rearp@texaschildrens.org

Locations

Texas Childrens Hospital

Recruiting

Houston, Texas, United States, 77030

Contacts

Principal Investigator, MD, PhD

832-824-4945spthomas@texaschildrens.org

More Information

Sponsor

Baylor College of Medicine

Last update posted

Jul 30, 2025

Last verified

Jul, 2025

Keywords

  • Intrathecal baclofen
  • Spasticity
  • Dystonia
  • Cerebral Palsy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Baylor College of Medicine on 2025-07-30.