Predictive Factors for Succes of ITB in CP

NCT05817409 | Not Yet Recruiting FDA Drug

• 1 other identifier: 2018.444
• Study Type: observational
• Target: 75
• Locations: 0 countries
• Sites: N/A

Brief Summary

For children with severe cerebral palsy (CP) in whom other options are insufficient, intrathecal baclofen (ITB) treatment could be an option. Literature provides some evidence for the effect of ITB, however we notice that not all children benefit equally. Furthermore long term effects of ITB in a prospective cohort of individuals with CP have not yet been described in literature. To improve selection for ITB physicians need to know which factors influence outcome on the long term. In order to accomplish these goals prospectively, real life data over a long period of time, and from a large cohort are needed. The investigators will therefore conduct a prospective observational cohort study using the Netherlands CP register to collect data over a period of 4,5 years. The Netherlands CP register is an innovative register for data collection which was developed between 2016 and 2020 in a unique collaboration between children with CP, their parents, researchers and health care professionals. All children and adolescents with CP, who start ITB treatment in the Netherlands under the age of 16, diagnosed with spastic or dyskinetic CP, are eligible for inclusion in the study. The primary outcome measure is goal attainment scaling (GAS) in which achievement of individual goals can be quantified. Factors that potentially influence outcome including spasticity, dystonia, scoliosis and complications will be secondary outcome measures. Measurements will be done before initiation of ITB, after 3 months, after 1 year and after 2 years. Statistical analysis will include effect size Cohen's d for all outcome measures. To assess which factors are possibly associated with GAS T-score outcome at follow-up multiple regression analysis will be conducted.

Conditions

Cerebral Palsy; Intrathecal Baclofen

Keywords

cerebral palsy; intrathecal baclofen

Outcome Measures

Primary Outcomes (1)

• Change in Goal Attainment Scaling (GAS)

  Attainment of personal treatment goals are scored on a standardised scale where -2 is the baseline score and 0 is the goal. Scores can be given for deterioration (-3), improvement but not attainment (-1) and more (+1) or much more (+2) than goal achieved). change in these outcomes between the different time points is assessed.

  3 months, 1 year, 2 years

Secondary Outcomes (14)

• range of motion

  baseline, 3 months, 1 year, 2 years

• spasticity

  baseline, 3 months, 1 year, 2 years

• Barry-Albright-Dystonia Scale (BADS)

  baseline, 3 months, 1 year, 2 years

• Scoliosis

  baseline, 3 months, 1 year, 2 years

• Selective motor control testing

  baseline, 3 months, 1 year, 2 years

Interventions

Intrathecal Baclofen Pump DRUG

intrathecal baclofen treatment

Also known as: intrathecal baclofen treatment

Eligibility Criteria

• Age: Up to 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

all patients with CP who start ITB under the age of 16 are eligible. They are recruited from the Amsterdam UMC or Maastricht UMC. Those are the only two centers in the Netherlands where ITB pump are implanted in children.

You may qualify if:

• All children and adolescents diagnosed with CP,
• who start ITB treatment in the Netherlands under the age of 16,

You may not qualify if:

• no consent is given by the patient and/or caregiver for participation

Sponsors & Collaborators

• Amsterdam UMC, location VUmc: lead
• Medtronic: collaborator

Related Links

• Dutch CP register

MeSH Terms

Conditions

Cerebral Palsy

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Annemieke Buizer, MD PhD

  Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Laura Bonouvrié, MD PhD

CONTACT

+31204440763; l.bonouvrie@amsterdamumc.nl

Aukje Andringa, PhD

CONTACT

+31204440763; a.andringa@amsterdamumc.nl

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD PhD

Study Record Dates

• First Submitted: March 21, 2023
• First Posted: April 18, 2023
• Study Start: June 1, 2023
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: April 18, 2023

Record last verified: 2023-04