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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand how intrathecal baclofen, a medication delivered directly to the spine, affects children with dystonic cerebral palsy, a condition that causes muscle stiffness and movement difficulties. Researchers seek to determine if this treatment reduces muscle tightness and identify which children benefit the most. The study involves gradually adjusting medication doses over a year to find the best balance of benefit and minimal side effects. Children who weigh at least 18 kg (about 4 years old) with cerebral palsy and muscle tightness verified by a doctor might be suitable for this trial. Participants will attend additional clinic visits and complete questionnaires throughout the study. As an unphased trial, this study offers a unique opportunity to contribute to understanding how this treatment can help children with cerebral palsy.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it does mention that botulinum injections within 3 months or phenol injections within 6 months before enrollment are not allowed, which might suggest some restrictions on certain treatments.

What prior data suggests that intrathecal baclofen is safe for children with dystonic cerebral palsy?

Research has shown that intrathecal baclofen (ITB) is generally safe and well-received by people with cerebral palsy. In one study, about 73.9% of patients reported being very satisfied with ITB treatment. However, some patients experienced side effects, such as muscle weakness or drowsiness. Another study found that ITB can help reduce muscle stiffness and improve movement, which are important issues for people with cerebral palsy. Long-term studies have confirmed the safety of ITB in both adults and children. While some side effects may occur, the treatment is considered safe for many people. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Intrathecal Baclofen is unique because it delivers medication directly into the spinal fluid, which can provide more targeted relief for individuals with cerebral palsy. Unlike oral medications, which can cause widespread side effects and might not effectively reach the central nervous system, this method allows for smaller doses to have a greater impact where it's needed most. Researchers are excited about this treatment because it offers the potential for better muscle control and reduced spasticity with fewer side effects, improving quality of life for patients.

What evidence suggests that intrathecal baclofen might be an effective treatment for cerebral palsy?

Research has shown that intrathecal baclofen (ITB) can help manage symptoms of cerebral palsy (CP), particularly by reducing muscle tightness. In one study, 86.5% of patients experienced less muscle stiffness. Another study found that ITB improved movement skills by an average of 9.62%. Younger patients often see more benefits from ITB treatment. Although some risks exist, the advantages in easing muscle stiffness are significant. Participants in this trial will follow a titration protocol to adjust ITB dosing based on individual responses. ^{6 7 8 9 10}

Who Is on the Research Team?

Sruthi P Thomas, MD, PhD

Principal Investigator

Baylor College of Medicine

Are You a Good Fit for This Trial?

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

They have a diagnosis of CP

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

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

Exclusion Criteria

Foster care placement or incarceration

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

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

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11055284/

Intrathecal baclofen efficacy for managing motor function and ... ITB therapy also improved motor function in patients with CP, as measured by the GMFM score. The average difference percentage was 9.62%, ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S0213485324001099

Review article Effectiveness of the intrathecal baclofen ... According to our findings, ITB treatment is more effective in younger patients. In line with these results, a clinical trial corroborates that ITB would allow ...

3. journals.lww.com journals.lww.com/md-journal/fulltext/2017/08250/outcomes_of_intrathecal_baclofen_therapy_in.5.aspx

Outcomes of intrathecal baclofen therapy in patients with... After ITB bolus injection, 32 patients (86.5%) (CP 84.2% versus acquired brain injury 88.9%) showed a positive response of reducing spasticity. However, 8 ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38678195/

Intrathecal baclofen efficacy for managing motor function ... Conclusion: Despite the risk of complications, ITB has a significant impact on the reduction of spasticity. A small but statistically ...

5. thejns.org thejns.org/pediatrics/view/journals/j-neurosurg-pediatr/35/3/article-p298.xml

Intraventricular baclofen for the treatment of pediatric ... This study reported on 15 cases of mixed or isolated spasticity and showed a relative improvement in the MAS and BADS scores after IVB pump placement.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5571990/

Outcomes of intrathecal baclofen therapy in patients with ... Seventeen patients or their caregivers (73.9%) were very satisfied, whereas 5 patients (21.7%) suffered from adverse events showed no subjective ...

7. sciencedirect.com sciencedirect.com/science/article/pii/S1090379825000509

Long-term assessment of continuous intrathecal baclofen ... From childhood to adulthood: Long-term assessment of continuous intrathecal baclofen therapy in non-ambulant spastic cerebral palsy - ScienceDirect.

8. bmcneurol.biomedcentral.com bmcneurol.biomedcentral.com/articles/10.1186/s12883-024-03647-7

Intrathecal baclofen efficacy for managing motor function and ... Long-term outcomes of ITB therapy in patients with CP are also needed, as short-term studies have shown improvements in spasticity and motor ...

9. thejns.org thejns.org/pediatrics/view/journals/j-neurosurg-pediatr/33/2/article-p179.xml

Safety and efficacy of intrathecal baclofen trials for the ... Outcome Measures and Data Analysis ... Outcomes of intrathecal baclofen therapy in patients with cerebral palsy and acquired brain injury.

10. neuromodulationjournal.org neuromodulationjournal.org/article/S1094-7159(21)00205-1/fulltext

Intrathecal Baclofen for Severe Spasticity The PSR provides the largest adult and pediatric patient cohort to date with results on the long-term safety and device performance of ITB in the treatment of ...