Intrathecal Baclofen for Cerebral Palsy

(PREDICT-ITB Trial)

Overseen By Rachel Arp

Age: < 18

Sex: Any

Trial Phase: Academic

Sponsor: Baylor College of Medicine

Must be taking: Intrathecal baclofen

Must not be taking: Botulinum, Phenol

Disqualifiers: Foster care, Incarceration, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 4

Duration: 12 months

65 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Sruthi P Thomas, MD, PhD

Principal Investigator

Baylor College of Medicine

Eligibility Criteria

This trial is for children with dystonic cerebral palsy who have significant muscle tightness, as shown by specific scores on medical scales. They must be willing to attend several assessments and be considered suitable for intrathecal baclofen pump treatment. Children should typically weigh around 18 kg or be at least 4 years old.

Inclusion Criteria

My doctor and I agree on proceeding with ITB implantation for muscle tone management.

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

They have a diagnosis of CP

Exclusion Criteria

I haven't had botulinum injections in the last 3 months or phenol injections in the last 6 months.

Foster care placement or incarceration

I need immediate treatment for severe muscle contractions or extreme nervous system reactions.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intrathecal baclofen (ITB) with a standardized titration protocol over a 12-month period to achieve maximum benefit with minimal side effects.

12 months

4 additional clinic visits outside usual care

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

Treatment Details

Interventions

• Intrathecal Baclofen

Trial Overview The study tests a titration protocol of intrathecal baclofen (ITB) in kids with dystonia from CP over a year. It aims to find the right ITB dosage that reduces dystonia without side effects, considering factors like spasticity and pain triggers.

Participant Groups

Group I: Titration protocol Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Baylor College of Medicine

Lead Sponsor

Trials

1,044

Recruited

6,031,000+

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

Collaborator

Trials

2,103

Recruited

2,760,000+