A Phase 2/3 interventional study of MSC, marrow and Placebo in Perinatal Arterial Ischemic Stroke, Perinatal Asphyxia and Hypoxic-Ischemic Brain Injury, sponsored by UMC Utrecht. Not yet recruiting. Open to participants aged 0 Days to 8 Days. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by UMC Utrecht · Phase 2/3, Interventional, and Treatment
Hypoxic-ischemic brain injury (HIBI) is a leading cause of death and lifelong disability in near-term and term infants, most often caused by perinatal arterial ischemic stroke (PAIS, incidence approximately 1:5000 live births) or perinatal asphyxia (PA, incidence 1-2:1000 live births). No treatment currently exists to repair or limit HIBI, and many affected infants go on to develop cerebral palsy, cognitive impairment, epilepsy, or visual or hearing deficits. Intranasally administered bone marrow-derived mesenchymal stromal cells (IN-MSC) are a candidate regenerative therapy for HIBI. Preclinical studies show that IN-MSC migrate to injured brain regions, dampen neuroinflammation, and stimulate endogenous neural repair, improving both structural and functional outcomes in animal models of HIBI. A first-in-human phase I trial (PASSIoN) in 10 neonates with PAIS showed that intranasal MSC administration was feasible and well tolerated, with no serious adverse events and promising early efficacy signals at 2-year follow-up. iSTOP-CP is a phase II, single-center (UMC Utrecht, the Netherlands), double-blind, randomized, placebo-controlled trial evaluating whether IN-MSC therapy can prevent or reduce motor and other disabilities in infants with MRI-confirmed HIBI due to PAIS or PA. Eligible infants are born at 35.0 weeks of gestation or later, have HIBI confirmed on brain MRI or MRS in predefined brain regions predictive of poor outcome, and are diagnosed with PAIS or PA, with or without prior therapeutic hypothermia. A total of 162 infants will be randomized 1:1 to IN-MSC or matching placebo, stratified by the cause of HIBI and, within the PAIS group, by stroke territory. Infants with suspected chromosomal, metabolic, or congenital central nervous system anomalies, intracranial hemorrhage as the main injury, or contraindications to intranasal administration are excluded, as are infants for whom the clinical team has decided to withdraw intensive care. Participants receive a single intranasal dose of bone marrow-derived MSC or a visually indistinguishable placebo within 7 days after birth. Participants are followed for 24 months, with neurodevelopmental follow-up visits at 3-4, 6, 9-12, and 24 months of age and a brain MRI at 52 weeks postmenstrual age. The primary endpoint is the motor composite score of the Dutch version of the Bayley Scales of Infant and Toddler Development, 4th edition (Bayley-IV-NL), assessed at 24 months corrected age. Key secondary endpoints include cognitive development, the incidence of sensorineural disability, neuroregenerative imaging biomarkers, safety, health-related quality of life for infants and their parents, and the cost-effectiveness of IN-MSC treatment, evaluated through a health technology assessment.
261 studies on the registry are indexed under Hypoxia-Ischemia, Brain; 95 are open to participants now.
This study's planned enrollment of 162 is above the median of 50 across 156 interventional studies indexed under Hypoxia-Ischemia, Brain.
Browse Hypoxia-Ischemia, Brain studies →UMC Utrecht is the lead sponsor of 364 studies on the registry; 82 are open to participants now.
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Diagnosed with HIBI on MRI, caused either by PAIS (ORPHA 439175) or PA (ORPHA 137577) diagnosis (will be randomized separately)
o PAIS: a neurologic condition characterized by focal cerebral ischemia and infarction following blockage of a brain artery with impairment of blood supply and oxygenation of brain tissue, clinically characterized by seizures (clinical or subclinical), respiratory difficulties, or both (unilateral or bilateral).
o PA: defined as at least one out of the following: 5-min Apgar score ≤ 5
Showing signs of hypoxic-ischemic injury in one or more of the following predefined brain areas (indicated by DWI restriction and/or abnormal ADC values and/or 1H-MRS lactate/N-acetyl aspartate (NAA) and/or NAA/Choline ratio abnormalities):
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Administration fluid (=physiological saline (0.9% NaCl; RVG 051680) with 10% Human Serum Albumin (RVG 103594))
Drug: Placebo
Bone-marrow derived mesenchymal stromal cells (BM-MSCs) intranasally administered once within 7 days after birth.
Drug: MSC, marrow
Allogeneic BM-MSCs
Also known as: mesenchymal stromal cells, MSC
Placebo is intranasally administered within 7 days.
Bayley Scales of Infant and Toddler Development 4th Edition (Bayley-IV-NL)
The motor composite score of the Bayley Scales of Infant and Toddler Development, Fourth Edition, Dutch version (Bayley-IV-NL), assessed at 24 months of age. The motor composite combines the Fine Motor and Gross Motor subdomains into a standard score with a mean of 100 and standard deviation of 15, with higher scores indicating better motor development (a score below 70 indicates substantial developmental delay).
Time frame: The assessment will be done when the participant is around 24 months of age.
The cognitive composite score of the Bayley-IV-NL.
he cognitive composite score of the Bayley Scales of Infant and Toddler Development, Fourth Edition, Dutch version (Bayley-IV-NL), assessed at 24 months of age, used to evaluate neurodevelopmental outcome beyond motor outcome. The cognitive composite is a standard score with a mean of 100 and standard deviation of 15, with higher scores indicating better cognitive development (a score below 70 indicates substantial developmental delay).
Time frame: The assessment will be done when the participant is around 24 months of age.
Sensorineural disability at 2 years
The incidence of one or more of the following components: development of cerebral palsy (CP) with a Gross Motor Function Classification System (GMFCS) of at least 1, neuromotor delay, cognitive neurodevelopmental delay, epilepsy, moderate-severe visual impairment, moderate-severe hearing impairment.
Time frame: These components will be assessed when the participant is around 24 months of age.
Incidence of Treatment-Related Adverse Events Following Intranasal MSC Administration
Patients will be regularly monitored until 24 months of age for adverse events related to intranasal MSC treatment in (near-)term infants with HIBI.
Time frame: Until the participant is 24 months of age.
Neuroregenerative effects seen on MRI
Volumetric analyses, white matter integrity of the major white matter tracts, metabolic profile of central gray and white matter, network-level function connectivity using resting-state functional MRI
Time frame: At approximately 3 months post-term equivalent age
Impact on Health Related Quality of Life (HRQoL)
The impact of intranasal MSC compared to standard supportive care on HRQoL by means of utility and disease specific measures. Primary at 3, 9-12 and 24 months: differences in utility between the treated and non-treated groups using the EuroQol Toddler and Infant Populations (EQ-TIPS) as a proxy measure for HRQoL in infants and the EuroQol 5D 5-level version (EQ-5D-5L) in at least one parent/caregiver. Secondary at 3, 9-12 and 24 months: Differences in disease and population specific HRQoL between the treated and non-treated groups measures via questionnaires in the infant (Pediatric Quality of Life Inventory (PedsQL)-Infant scale) and at least one parent/caregiver (depression: post-traumatic stress disorder checklist PCL-5, Burden: LTO, Pain: KLIK questionnaires Pain, fatigue: Checklist Individual Strength (CIS-4) and resilience: Warwick-Edinburgh Mental Well-being Scale (WEMWBS)).
Time frame: At 3, 9-12 and 24 months.
Health Technology Assessment
The economic impact of intranasal MSCs compared to standard supportive care at 24 months of age, assessed through Health Technology Assessment (HTA).
Time frame: When the participant is around 24 months of age.
Pharmacodynamic biomarker assessments
Blood plasma will be used to quantify circulating markers reflecting mechanistic pathways identified in preclinical studies. In addition, plasma samples may be used for exploratory high-sensitivity analyses to further characterize neuroregenerative activity.
Time frame: Bloodsamples will be collected at: 1. Baseline (before administration), 2. 48 hours after administration and if possible 3. If participant is still in the hospital and blood can be drawn with regular samplin, at 96 hours after administration
No study locations are listed for this record.
Start is estimated for Apr 2027; primary completion for Sep 2032, 65 months later.
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This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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