Clinical trial • Not applicable • Neurology

BACLOFEN for Cerebral palsy

Not applicable trial of BACLOFEN for Cerebral palsy.

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Cerebral palsy
Trial Stage
Not applicable
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
03-12-2025
First CTIS Authorization Date
27-01-2026

Trial design

Randomised, open-label, intrathecal baclofen pump (itb): implantation of an intrathecal baclofen pump delivering baclofen directly to the intrathecal space. products listed: lioresal® intrathecal infusion 10mg/5ml, lioresal® intrathecal infusion 10mg/20ml, baclofen aguettant 2 mg/ml solution for infusion; maximum reported daily dose 2000 µg. selective dorsal rhizotomy (sdr): selective sectioning of lumbosacral dorsal rootlets to reduce spasticity. standard care is continued for both arms.-controlled Not applicable trial across 7 sites in Netherlands, Belgium.

Randomised
Yes
Open Label
Yes
Comparator
Intrathecal Baclofen Pump (ITB): implantation of an intrathecal baclofen pump delivering baclofen directly to the intrathecal space. Products listed: Lioresal® Intrathecal Infusion 10mg/5ml, Lioresal® Intrathecal Infusion 10mg/20ml, Baclofen Aguettant 2 mg/mL solution for infusion; maximum reported daily dose 2000 µg. Selective Dorsal Rhizotomy (SDR): selective sectioning of lumbosacral dorsal rootlets to reduce spasticity. Standard care is continued for both arms.
Target Sample Size
48
Trial Duration For Participant
2920

Eligibility

Recruits 48 paediatric patients.

Vulnerable Population
Participants are children aged ≥4 and <18 years (paediatric population). Consent must be provided by the participant or their legally authorised representative; informed assent is obtained from the child in a manner appropriate to their age and understanding. Age-specific subject information and informed consent forms are provided (parents/guardians forms, adolescents 12-16/12-17, children under 12, and forms for adults who become adult during the study). Documents are available in country-specific languages (Netherlands Dutch and Belgium Dutch/French are provided in the submission).

Inclusion criteria

  • {"criterion_text":"- Voluntary written informed consent of the participant or their legally authorized representative has been obtained prior to any screening procedures. Informed assent has been obtained by the child in a manner that is appropriate for their age and understanding.\n- Male or female patients\n- Aged ≥ 4 and <18 years\n- With spastic CP\n- Gross motor function classification system (GFMCS) grade 4 or 5\n- Spasticity insufficiently controlled with conservative therapy and/or oral baclofen or intolerable side effects. Patients are allowed to continue or change their usual medication during the study. We will register concomitant medication throughout the study so we can evaluate whether this can be reduced over time. Cross-over neurosurgical procedures (ITB after SDR, or SDR after ITB) are not allowed during the first year in order to not influence the impact of our techniques. Orthopedic interventions and/or botulinum to"}

Exclusion criteria

  • {"criterion_text":"- Dystonia\n- Basal ganglia/thalamus lesions or normal brain MRI\n- <15kg body weight\n- Previous ITB/SDR surgery\n- Evidence of genetic illness\n- Evidence of neurological progressive illness"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change in CP-CHILD caregiver version (in absolute value) in the first year postoperatively vs. immediately preoperatively (non-inferiority margin = clinically meaningful difference = 5/100).","definition_or_measurement_approach":"Measured as change in the CP-CHILD caregiver version absolute score from immediately preoperative baseline to 1 year postoperatively; non-inferiority margin specified as 5/100."}

Secondary endpoints

  • {"endpoint_text":"- Adverse events\n- Cost-effectiveness\n- Cost-utility\n- Spasticity\n- Range of motion\n- Trunk control\n- Communication\n- Orobulbar function\n- Sleep\n- Urinary symptoms\n- Spinal/hip deformities\n- Medication use\n- Quality of life\n- Treatment goals\n- Parental distress\n- Patient satisfaction\n- Healthcare costs\n- Complications","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
48
Recruitment Window Months
159
Consent Approach
Voluntary written informed consent is required from the participant or their legally authorised representative prior to any screening procedures. Informed assent is obtained from the child appropriate to their age and understanding. Age-specific information and consent documents are provided (parents/guardians; adolescents 12-16/12-17; children under 12; adults who become adult during the study). Country-specific language versions are provided (Netherlands Dutch; Belgium Dutch and French). A dedicated 'Informed Consent Procedure' document and sponsor statements are included in the submission.

Geography

Total Number Of Sites
7
Total Number Of Participants
48

Netherlands

Earliest CTIS Part Ii Submission Date
07-01-2026
Latest Decision Or Authorization Date
27-01-2026
Processing Time Days
20
Number Of Sites
2
Number Of Participants
27

Sites

Site Name
Amsterdam UMC Stichting
Department Name
Rehabilitation medicine
Contact Person Name
Annemieke Buizer
Contact Person Email
ai.buizer@amsterdamumc.nl
Site Name
Academisch Ziekenhuis Maastricht
Department Name
Pediatric Neurology
Contact Person Name
Jeroen Vermeulen
Contact Person Email
jeroen.vermeulen@mumc.nl

Belgium

Earliest CTIS Part Ii Submission Date
27-03-2026
Latest Decision Or Authorization Date
03-04-2026
Processing Time Days
7
Number Of Sites
5
Number Of Participants
21

Sites

Site Name
Universitair Ziekenhuis Antwerpen
Department Name
Pediatric Neurology
Contact Person Name
Sandra Kenis
Contact Person Email
Sandra.Kenis@uza.be
Site Name
UZ Leuven
Department Name
Neurosurgery
Contact Person Name
Philippe De Vloo
Contact Person Email
philippe.devloo@uzleuven.be
Site Name
Cliniques Universitaires Saint-Luc
Department Name
Médecine physique et réadaptation
Contact Person Name
Anne Renders
Site Name
Universitair Ziekenhuis Gent
Department Name
Orthopedie en Traumatologie
Contact Person Name
Frank Plasschaert
Contact Person Email
frank.plasschaert@uzgent.be
Site Name
UZ Brussel
Department Name
Pediatric Neurology
Contact Person Name
Nathalie Smeets
Contact Person Email
nathalie.smeets@uzbrussel.be

Sponsor

Primary sponsor

Full Name
UZ Leuven
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Belgium

Third parties

  • {"country":"","full_name":"Belgian Health Care Knowledge Centre (KCE)","duties_or_roles":"Monetary support/funder","organisation_type":""}
  • {"country":"","full_name":"The Netherlands Organisation for Health Research and Development (ZonMw)","duties_or_roles":"Monetary support/funder","organisation_type":""}

Investigational products

Investigational Product Name
Baclofen Aguettant 2 mg/mL solution for infusion
Active Substance
BACLOFEN
Modality
Small molecule
Routes Of Administration
INTRATHECAL USE
Route
Intrathecal
Authorisation Status
Marketing authorisation present (marketingAuthNumber: PL 14434/0026)
Maximum Dose
2000 µg
Investigational Product Name
Lioresal® Intrathecal Infusion 10mg/20ml
Active Substance
BACLOFEN
Modality
Small molecule
Routes Of Administration
INTRATHECAL USE
Route
Intrathecal
Authorisation Status
Marketing authorisation present (marketingAuthNumber: PL 00101/0501)
Maximum Dose
2000 µg
Investigational Product Name
Lioresal® Intrathecal Infusion 10mg/5ml
Active Substance
BACLOFEN
Modality
Small molecule
Routes Of Administration
INTRATHECAL USE
Route
Intrathecal
Authorisation Status
Marketing authorisation present (marketingAuthNumber: PL 00101/0502)
Maximum Dose
2000 µg

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