Intrathecal Baclofen and Pediatric Dystonia

NCT06606574 | Recruiting DMC

• 2 other identifiers: H-54449R01HD112563
• Study Type: interventional
• Target: 65
• Locations: 1 country
• Sites: 1

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. 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. 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

Keywords

Intrathecal baclofen; Spasticity; Dystonia; Cerebral Palsy

Outcome Measures

Primary Outcomes (1)

• Barry-Albright Dystonia Scale (BADS)

  The BADS was created based on the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS), a tool originally designed to detect cervical dystonia in adults.The BADS tailors this scale for detecting dystonia in CP and acquired brain injury in individuals with limitations in cognition and physical ability. Scoring focuses on posturing and involuntary dystonic movements to detect potential changes in dystonia that affect ease of care and comfort. It does not assess functional tasks. It has moderate internal consistency and inter-rater reliability. It has effectively detected change with ITB. The BADS was also found to have the greatest clinical utility for children with dystonic CP

  Baseline (pre-ITB initiation) , 3 months, 6 months, 12 months post ITB initiation

Secondary Outcomes (10)

• Dyskinesia Impairment Scale-II (DIS-II)

  Baseline (pre-ITB initiation), 3 months, 6 months, 12 months post ITB initiation

• Australian Spasticity Assessment Scale (ASAS) and Composite Measure

  Baseline (pre-ITB initiation), 3 months, 6 months, 12 months post ITB initiation

• Modified Ashworth Scale (MAS)

  Baseline (pre-ITB initiation), 3 months, 6 months, 12 months post ITB initiation

• Gross Motor Function Measure (GMFM)

  Baseline (pre-ITB initiation) , 3 months, 6 months, 12 months post ITB initiation

• Box and Blocks Test

  Baseline (pre-ITB initiation) , 3 months, 6 months, 12 months post ITB initiation

Study Arms (1)

Titration protocol

OTHER

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.

Other: Titration protocol

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.

Titration protocol

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Baylor College of Medicine: lead
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): collaborator

Study Sites (1)

Texas Childrens Hospital

Houston, Texas, 77030, United States

RECRUITING

Related Publications (5)

• 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.

  PMID: 14749614 BACKGROUND

• 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.

  PMID: 32098856 BACKGROUND

• 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.

  PMID: 33772789 BACKGROUND

• 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.

  PMID: 31050023 BACKGROUND

• 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.

  PMID: 40672491 DERIVED

MeSH Terms

Conditions

Muscle Spasticity; Dystonia; Cerebral Palsy

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Muscle Hypertonia; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Dyskinesias; Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases

Study Officials

• Sruthi P Thomas, MD, PhD

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sruthi Thomas, MD, PhD

CONTACT

832-824-4945; spthomas@texaschildrens.org

Rachel Arp

CONTACT

(936) 267-7707; rearp@texaschildrens.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Masking Details: Therapists completing the assessments will be blinded to the patient's baseline characteristics, imaging findings, and current ITB dosage.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: September 18, 2024
• First Posted: September 23, 2024
• Study Start: November 1, 2024
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): August 31, 2029
• Last Updated: July 30, 2025

Record last verified: 2025-07

Locations

US (1)