Clinical trial • Phase IV • Neurology

VIGABATRIN for Infantile spasms (infantile spasm syndrome) | Tuberous sclerosis complex | Hypoxic-ischemic encephalopathy | Intraventricular haemorrhage (grade IV) | Periventricular leukomalacia

Phase IV trial of VIGABATRIN for Infantile spasms (infantile spasm syndrome) | Tuberous sclerosis complex | Hypoxic-ischemic encephalopathy | Intraventric…

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Infantile spasms (infantile spasm syndrome) | Tuberous sclerosis complex | Hypoxic-ischemic encephalopathy | Intraventricular haemorrhage (grade IV) | Periventricular leukomalacia
Trial Stage
Phase IV
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
31-08-2024
First CTIS Authorization Date
13-09-2024

Trial design

Phase IV trial across 15 sites in Finland.

Target Sample Size
140

Eligibility

Recruits 140 paediatric patients.

Vulnerable Population
Participants are infants (<1 year old), identified as a vulnerable population. Consent is provided by parents/legal guardians. Parents must have sufficient Finnish, Swedish or English (or the native language of the research centre) language skills. Subject information and informed consent forms are available (documents include Finnish and English versions), and parental/legal guardian consent procedures are described in the study documentation.

Inclusion criteria

  • {"criterion_text":"-All children aged <1yr old with tuberous sclerosis (but not anymore after 11/2023), or large vascular cortical injury (large infarc-tion/hemorrhage/infection/trauma, HIE II-III including MRI criteria 2b and 3 involving cortical lesion or early stage MRI, imaged within first 5 days, general swelling of the cerebrum hemispheres and simultaneous extensive cortical/subcortical restricted diffusion finding in the ADC map). Large cortical injury is defined as almost the whole area of a. cerebri anterior or posterior, or about half of the area of a. cerebri media. Additionally, a grade IV intraventricular hemorrhage (including hemorrhagic infarction in the parenchyma) or grade III-IV PVL (containing porencephalic cyst) in premature infants. Brain MRI must have been done earlier as a part of normal clinical diagnostics.Parents must have sufficient Finnish, Swedish or English (or the native language of the research center country) language skills."}

Exclusion criteria

  • {"criterion_text":"-Patient has not allowed to have received VGB for any reason before. A patient will not be recruited if he/she is already participating in another medication intervention study, or the cause of the cortical injury is child abuse. Or child has had infantile spasms before."}

Endpoints

Primary endpoints

  • {"endpoint_text":"-Efficacy: whether patient gets infantile spasm syndrome or not","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"-Is there a difference between groups in neurocognitive development? Can eye tracking analysis predict EEG changes, neurocognitive development or onset of infantile spasm syndrome?","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
140
Recruitment Window Months
187
Consent Approach
Informed consent is provided by the parents/legal guardians (participants are infants under 1 year). Study documentation includes subject information and informed consent forms; available document titles indicate Finnish and English versions (and parents must have sufficient Finnish, Swedish or English or the native language of the research centre). Assent is not applicable due to participant age; parental/legal guardian consent procedures are described in the study documents.

Geography

Total Number Of Sites
15
Total Number Of Participants
140

Finland

Earliest CTIS Part Ii Submission Date
23-08-2024
Latest Decision Or Authorization Date
12-03-2026
Processing Time Days
566
Number Of Sites
15
Number Of Participants
140

Sites

Site Name
Lapland Central Hospital
Department Name
-
Contact Person Name
we have study permission, but due to lack of PI this site is not active -
Contact Person Email
kirsi.h.mikkonen@hus.fi
Site Name
Central Hospital in Kemi (Länsi-Pohja Central Hospital)
Department Name
Pediatric Neurology
Contact Person Name
Katariina Mankinen
Site Name
Oulu University Hospital
Department Name
Pediatric Neurology
Contact Person Name
Päivi Vieira
Contact Person Email
paivi.vieira@oulu.fi
Site Name
Kymenlaakso Central Hospital
Department Name
Pediatric Neurology
Contact Person Name
Merimaaria Espo
Contact Person Email
merimaaria.espo@kymsote.fi
Site Name
Tampere University Hospital
Department Name
Pediatric Neurology
Contact Person Name
Sini Erme
Contact Person Email
sini.erme@pirha.fi
Site Name
Central Finland Hospital District Central Finland Hospital Nova
Department Name
Pediatric Neurology
Contact Person Name
Tonja Johnson-Ferguson
Site Name
HUS-Yhtymae
Department Name
P
Contact Person Name
Sanna Wickman
Contact Person Email
sanna.wickman@hus.fi
Site Name
South Karelia Central Hospital
Department Name
Pediatric Neurology
Contact Person Name
Urpu Sandini
Contact Person Email
urpu.sandini@ekvha.fi
Site Name
Kanta-Hame Central Hospital
Department Name
Pediatri
Contact Person Name
Kati Pietilä
Site Name
Seinäjoki Central Hospital
Department Name
Pediatric Neurology
Contact Person Name
Eija Yli-Rahnasto
Contact Person Email
eija.yli-rahnasto@hyvaep.fi
Site Name
HUS-Yhtymae (Helsinki University Hospital/The New Children's hospital)
Department Name
Pediatric Neurology
Contact Person Name
Kirsi Mikkonen
Contact Person Email
kirsi.h.mikkonen@hus.fi
Site Name
Lahti Central Hospital
Department Name
Pediatric Neurology
Contact Person Name
Heli Sätilä
Contact Person Email
heli.satila@paijatha.fi
Site Name
Kainuu Central Hospital
Department Name
Pediatric Neurology
Contact Person Name
Maarit Erola
Contact Person Email
maarit.erola@kainuu.fi
Site Name
Kuopio University Hospital
Department Name
Pediatric Neurology
Contact Person Name
Jarkko Kirjavainen
Site Name
Turku University Hospital
Department Name
Pediatric Neurology
Contact Person Name
Tuire Lähdesmäki
Contact Person Email
tuire.lahdesmaki@varha.fi

Sponsor

Primary sponsor

Full Name
HUS-Yhtymae
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Finland

Investigational products

Investigational Product Name
Sabrilex 500 mg rakeet oraaliliuosta varten
Active Substance
VIGABATRIN
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Authorised (marketing authorisation FI/H/0139/002)
Maximum Dose
150 mg/kg

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