Comparison of two treatments (intrathecal baclofen pump and selective dorsal rhizotomy) for children with cerebral palsy who cannot walk

Overview

Sponsor-declared trial summary

Key facts

Sponsor

UZ Leuven

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2026-04-03

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Belgian Health Care Knowledge Centre (KCE) · The Netherlands Organisation for Health Research and Development (ZonMw)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To compare the health-related quality of life, safety profile and cost effectiveness and cost utility 1 year after SDR and ITB in non-ambulatory children with spastic Cerebral palsy.

Secondary objectives 1

1. To compare quality of life, safety, cost effectiveness and cost utility, spasticity, passive range of motion, trunk control, fine motor function, communication, orobulbar function (swallowing), lower urinary tract symptoms, sleep, use of antispasmodic drugs and analgesics, parental distress, attainment of treatment goals, and patient satisfaction over the first 8 years after SDR and ITB in non-ambulatory children with spastic Cerebral palsy.

Conditions and MedDRA coding

Cerebral Palsy

Study design 1 period

Regulatory references

Plan to share IPD

No

IPD plan description

Individual participant data will not be shared publicly. Only aggregated results will be made available through scientific publications and clinical trial registries, in accordance with the protocol and publication policy.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Participants eligible for inclusion in this Trial must meet all of the following criteria: 1. 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. 2. Male or female patients 3. Aged ≥ 4 and <18 years 4. With spastic CP 5. Gross motor function classification system (GFMCS) grade 4 or 5 6. 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 1

1. Participants eligible for this Trial must not meet any of the following criteria: 1. Dystonia 2. Basal ganglia/thalamus lesions or normal brain MRI 3. <15kg body weight 4. Previous ITB/SDR surgery 5. Evidence of genetic illness 6. Evidence of neurological progressive illness

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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).

Secondary endpoints 1

1. Key secondary endpoints: Adverse events; cost-effectiveness and cost-utility. Additional secondary endpoints: Spasticity; range of motion; trunk control; communication; orobulbar function; sleep; urinary symptoms; spinal/hip deformities; medication use; quality of life; treatment goals; parental distress; patient satisfaction; healthcare costs; complications.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

UZ Leuven

Sponsor organisation

UZ Leuven

Address

Herestraat 49

City

Leuven

Postcode

3000

Country

Belgium

Scientific contact point

Organisation

UZ Leuven

Contact name

Philippe De Vloo

Public contact point

Organisation

UZ Leuven

Contact name

Amal El Kaddouri

Locations

2 EU/EEA countries · 7 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 51 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications