Intrathecal Baclofen and Pediatric Dystonia

NCT ID: NCT06606574

Last Updated: 2025-07-30

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 65 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-11-01
• Study Completion Date: 2029-08-31

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.

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 ~$1.4 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 study's 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 Children's 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.

Conditions

Dystonic Cerebral Palsy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: OTHER

Titration protocol

Intervention Type: 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.

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.

Intervention Type: OTHER

Eligibility Criteria

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

• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

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

NIH

Sponsor Role: collaborator

Baylor College of Medicine

OTHER

Sponsor Role: lead

Responsible Party

Sruthi P. Thomas, MD, PhD

Associate Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Sruthi P Thomas, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Baylor College of Medicine

Locations

Texas Childrens Hospital

Houston, Texas, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Sruthi Thomas, MD, PhD

Role: CONTACT

spthomas@texaschildrens.org

832-824-4945

Rachel Arp

Role: CONTACT

rearp@texaschildrens.org

(936) 267-7707

Facility Contacts

Principal Investigator, MD, PhD

Role: primary

spthomas@texaschildrens.org

832-824-4945

References

Centers for Disease Control and Prevention (CDC). Economic costs associated with mental retardation, cerebral palsy, hearing loss, and vision impairment--United States, 2003. MMWR Morb Mortal Wkly Rep. 2004 Jan 30;53(3):57-9.

Reference Type: BACKGROUND

PMID: 14749614 (View on PubMed)

Lungu C, Ozelius L, Standaert D, Hallett M, Sieber BA, Swanson-Fisher C, Berman BD, Calakos N, Moore JC, Perlmutter JS, Pirio Richardson SE, Saunders-Pullman R, Scheinfeldt L, Sharma N, Sillitoe R, Simonyan K, Starr PA, Taylor A, Vitek J; participants and organizers of the NINDS Workshop on Research Priorities in Dystonia. Defining research priorities in dystonia. Neurology. 2020 Mar 24;94(12):526-537. doi: 10.1212/WNL.0000000000009140. Epub 2020 Feb 25.

Reference Type: BACKGROUND

PMID: 32098856 (View on PubMed)

Bohn E, Goren K, Switzer L, Falck-Ytter Y, Fehlings D. Pharmacological and neurosurgical interventions for individuals with cerebral palsy and dystonia: a systematic review update and meta-analysis. Dev Med Child Neurol. 2021 Sep;63(9):1038-1050. doi: 10.1111/dmcn.14874. Epub 2021 Mar 27.

Reference Type: BACKGROUND

PMID: 33772789 (View on PubMed)

Bonouvrie LA, Becher JG, Vles JSH, Vermeulen RJ, Buizer AI; IDYS Study Group. The Effect of Intrathecal Baclofen in Dyskinetic Cerebral Palsy: The IDYS Trial. Ann Neurol. 2019 Jul;86(1):79-90. doi: 10.1002/ana.25498. Epub 2019 May 21.

Reference Type: BACKGROUND

PMID: 31050023 (View on PubMed)

Thomas SP, Fehlings D, Ramey S, Conaway M, Kralik S, Raskin J. PREDICT-ITB: Predicting response in children with dystonic CP to ITB - study protocol. medRxiv [Preprint]. 2025 Jul 8:2025.07.08.25331080. doi: 10.1101/2025.07.08.25331080.

Reference Type: DERIVED

PMID: 40672491 (View on PubMed)

Other Identifiers

R01HD112563

Identifier Type: NIH

Identifier Source: secondary_id

View Link

H-54449

Identifier Type: -

Identifier Source: org_study_id