Overview
Sponsor-declared trial summary
Perinatal Arterial Ischemic Stroke (PAIS)
To perform a double-blind randomized placebo controlled multicenter study with darbepoetin in infants with MRI confirmed PAIS and to investigate whether darbepoetin can reduce brain injury in neonates who suffered from perinatal arterial ischemic stroke (PAIS). The ultimate goal of this study is therefore to develo…
Key facts
- Sponsor
- Universitair Medisch Centrum Utrecht
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 8 Aug 2024 → ongoing
- Decision date (initial)
- 2024-08-08
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- CP Alliance
External identifiers
- EU CT number
- 2024-513042-12-01
- EudraCT number
- 2015-002997-18
- ClinicalTrials.gov
- NCT03171818
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Efficacy, Safety
To perform a double-blind randomized placebo controlled multicenter
study with darbepoetin in infants with MRI confirmed PAIS and to
investigate whether darbepoetin can reduce brain injury in neonates
who suffered from perinatal arterial ischemic stroke (PAIS). The
ultimate goal of this study is therefore to develop a therapy using
erythropoiesis-stimulating-agents (ESAs) such as darbepoetin to reduce
or even prevent lifelong consequences of PAIS-related brain injury in
this group of term newborns.
Conditions and MedDRA coding
Perinatal Arterial Ischemic Stroke (PAIS)
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-513042-12-00 | Darbepoetin for Ischemic Neonatal Stroke to Augment Regeneration | Universitair Medisch Centrum Utrecht |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- 1. Newborns ≥ 36 weeks gestation, both male and female 2. MRI confirmed diagnosis of acute PAIS , with involvement. 4. Written informed consent from custodial parent(s) of the cortical spinal tract (e.g. PLIC or peduncles) 3. Less than 4 days after the onset of clinical symptoms
Exclusion criteria 1
- - Moderate –severe HIE with or without hypothermia therapy ; - Any proven or suspected major congenital anomaly, chromosomal disorder, metabolic disorder; - Presence of a serious infection of the central nervous system; - No realistic prospect of survival, (e.g. severe brain injury), at the discretion of the attending physician; - Infant for whom withdrawal of supportive care is being considered.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Our primary objective is to determine whether there is a difference in the degree in stroke tissue loss between darbepoetin and placebo treatment, which will be measured by the change in lesion size and brain growth between the time of onset of the insult and 6-8 weeks of age. Additionally we will assess whether there are differences between darbepoetin and placebo treatment in DTI parameters of selected regions of interest.
Secondary endpoints 1
- We will assess development of USCP, and cognitive development at 18 months of age using the BSID-III and PSOM scores as well as a full neurological assessment including Gross Motor Function Classification system (GMFCS) and several handfunction tests such as Manual Ability Classification System (MACS), the Hand Assessment of Infants (HAI) and Assisting Hand Assessment (AHA) and compare them between groups (darbepoetin vs placebo).
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Aranesp 10 micrograms solution for injection in pre-filled syringe
PRD539962 · Product
- Active substance
- Darbepoetin Alfa
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 10 µg/Kg microgram(s)/kilogram
- Max total dose
- 20 µg/Kg microgram(s)/kilogram
- Max treatment duration
- 1 Week(s)
- Authorisation status
- Authorised
- ATC code
- B03XA02 — DARBEPOETIN ALFA
- Marketing authorisation
- EU/1/01/185/033
- MA holder
- AMGEN EUROPE B.V.
- MA country
- EU
- Paediatric formulation
- Yes
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- repackaging
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Universitair Medisch Centrum Utrecht
- Sponsor organisation
- Universitair Medisch Centrum Utrecht
- Address
- Heidelberglaan 100
- City
- Utrecht
- Postcode
- 3584 CX
- Country
- Netherlands
Scientific contact point
- Organisation
- Universitair Medisch Centrum Utrecht
- Contact name
- MJNL Benders
Public contact point
- Organisation
- Universitair Medisch Centrum Utrecht
- Contact name
- MJNL Benders
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Netherlands | Ongoing, recruiting | 40 | 1 |
| Rest of world
Canada
|
— | 40 | — |
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 |
|---|---|---|---|---|---|
| Netherlands | 2024-08-08 | 2024-08-08 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 4 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-513042-12-00_redacted | 2.4 |
| Recruitment arrangements (for publication) | K1_blank | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Dutch_redacted | 2.4 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_smPC Aranesp | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-07-19 | Netherlands | Acceptable with conditions 2024-08-08
|
2024-08-08 |