Overview
Sponsor-declared trial summary
Patients in high risk for infantile spasm syndrome (age under 1 yr old): -tuborous sclerosis (not after 11/2023) -large vascular cortical injury (large infarction/hemorrhage/infection/trauma, HIE II-III including MRI-criteria 2b and 3, with also cortical lesions -grade IV intraventricular hemorrhage (including hemorrhagic infarct in brain parenchyma) -grade III-IV PVL (including porencephalic cyst) in preterms
This study aims to prevent infantile spasm syndrome in high risk children (tuberous sclerosis or large vascular injury) under 1-yr old by vigabatrin prophylactic medication.
Key facts
- Sponsor
- HUS-Yhtymae
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 28 May 2018 → ongoing
- Decision date (initial)
- 2024-09-13
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-517488-22-00
- EudraCT number
- 2017-004775-30
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Prophylaxis
This study aims to prevent infantile spasm syndrome in high risk children (tuberous sclerosis or large vascular injury) under 1-yr old by vigabatrin prophylactic medication.
Conditions and MedDRA coding
Patients in high risk for infantile spasm syndrome (age under 1 yr old): -tuborous sclerosis (not after 11/2023) -large vascular cortical injury (large infarction/hemorrhage/infection/trauma, HIE II-III including MRI-criteria 2b and 3, with also cortical lesions -grade IV intraventricular hemorrhage (including hemorrhagic infarct in brain parenchyma) -grade III-IV PVL (including porencephalic cyst) in preterms
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- 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 1
- 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 and secondary outcome measures (English text)
Primary endpoints 1
- Efficacy: whether patient gets infantile spasm syndrome or not
Secondary endpoints 1
- Is there a difference between groups in neurocognitive development? Can eye tracking analysis predict EEG changes, neurocognitive development or onset of infantile spasm syndrome?
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Sabrilex 500 mg rakeet oraaliliuosta varten
PRD482281 · Product
- Active substance
- Vigabatrin
- Pharmaceutical form
- GRANULES FOR ORAL SOLUTION
- Route of administration
- ORAL
- Max daily dose
- 150 mg/kg milligram(s)/kilogram
- Max total dose
- 150 mg/kg milligram(s)/kilogram
- Max treatment duration
- 11 Month(s)
- Authorisation status
- Authorised
- ATC code
- N03AG04 — VIGABATRIN
- Marketing authorisation
- 11148
- MA holder
- SANOFI OY
- MA country
- Finland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
HUS-Yhtymae
- Sponsor organisation
- HUS-Yhtymae
- Address
- Stenbackinkatu 9
- City
- Helsinki
- Postcode
- 00290
- Country
- Finland
Scientific contact point
- Organisation
- HUS-Yhtymae
- Contact name
- HUS Helsinki University Hospital, New Children’s Hospital, pediatric neurology
Public contact point
- Organisation
- HUS-Yhtymae
- Contact name
- HUS Helsinki University Hospital, New Children’s Hospital, pediatric neurology
Locations
1 EU/EEA country · 15 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Finland | Ongoing, recruiting | 140 | 15 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Finland | 2018-05-28 | 2018-09-15 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 13 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_ Protocol EU CT 2024-517488-22-00_version 1_6_clean version to be version 1_7 | 1.6 |
| Protocol (for publication) | Tutkimussuunnitelma _PREVIS | 1.7 |
| Recruitment arrangements (for publication) | Liite 7 Selvitys suostumuksen saamiseksi noudatettavasta menettelysta | 1 |
| Subject information and informed consent form (for publication) | L1_ICF_Finnish_versio 1_2_clean version to be version 1_3 | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS_english | 1.5 |
| Subject information and informed consent form (for publication) | L1_SIS_english_version 1_4 | 1.4 |
| Subject information and informed consent form (for publication) | L1_SIS_Finnish_version 1_4_clean version to be version 1_5 | 1.4 |
| Subject information and informed consent form (for publication) | L1_SIS_Finnish_version 1_4_TSC patients_only redacted | 1.4 |
| Subject information and informed consent form (for publication) | L1-ICF_English_versio 1_3_TC | 1.3 |
| Subject information and informed consent form (for publication) | L1-ICF-English_version 1_2 | 1.2 |
| Subject information and informed consent form (for publication) | suostumuskaavake | 1 |
| Subject information and informed consent form (for publication) | Tiedote tutkittavalle aivokuorivaurio PREV-IS | 1.5 |
| Synopsis of the protocol (for publication) | DI_Protocol synopsis FIN 2024-517488-22-00 | 1 |
Application history
4 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-08-31 | Finland | Acceptable with conditions 2024-09-13
|
2024-09-13 |
| 2 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-10-24 | Finland | Acceptable 2025-12-22
|
2026-01-02 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2026-03-12 | Finland | Acceptable 2025-12-22
|
2026-03-12 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-03-29 | Finland | Acceptable 2025-12-22
|
2026-03-29 |