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Intranasal remestemcel therapy for newborns with hypoxic‑ischemic brain injury: a phase II placebo‑controlled trial

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What is this trial about?

A plain-language summary of the goals, design and what participants do

A rare condition called hypoxic-ischemic brain injury can occur when a newborn’s brain does not receive enough oxygen and blood, often because of perinatal asphyxia or a perinatal arterial ischemic stroke. This lack of oxygen can damage brain tissue and may later result in cerebral palsy, which affects movement and coordination. The study investigates a new treatment that delivers bone‑marrow derived mesenchymal stromal cells straight into the nose as tiny drops; the active product is identified as remestemcel. These stem cells are thought to help repair damaged brain areas and support better development.

The main goal of the trial is to find out whether this intranasal stem cell therapy can safely reduce brain injury and improve motor abilities in affected infants. Babies with a confirmed diagnosis on a brain scan are randomly assigned to receive either the stem cell drops or a placebo that looks the same but contains no active cells. Over the next two years, the children are monitored for safety, including regular check‑ups and brain imaging using MRI, a technique that creates detailed pictures of the brain without surgery.

Developmental progress is measured with the Bayley-IV-NL motor score at 24 months, which evaluates how well a child can move and control muscles. Additional assessments look at thinking skills, the presence of cerebral palsy, seizures, and vision or hearing problems. Families also complete questionnaires about quality of life, and any serious health events are recorded throughout the study period.

The research process

The trial runs in 9 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    After enrollment, a baseline magnetic resonance imaging (mri) will be performed to confirm the brain injury and to document the initial condition.

    Clinical evaluation of motor function and overall health will be recorded before any study medication is given.

  2. Step 2

    Study medication administration

    You will receive a single dose of 0.6 ml intranasal stem cell therapy (remestemcel) as nasal drops, or a matching placebo (potassium chloride or 10% human serum albumin) delivered in the same volume and route.

    The drops are placed in each nostril by a trained professional; the administration occurs only once at the start of the trial.

  3. Step 3

    Early safety monitoring (first 3 months)

    For the first three months after the dose, you will be observed for any adverse events, and any reactions will be recorded by the study team.

    A safety visit will be scheduled at 3‑4 months to review clinical status and to prepare for the follow‑up mri.

  4. Step 4

    Follow‑up mri at 52 weeks postmenstrual age

    Approximately 12 months after birth (52 weeks postmenstrual age), a repeat magnetic resonance imaging (mri) will be performed to assess brain development and to check for any abnormal growth such as tumor formation.

  5. Step 5

    Developmental assessment at 3‑4 months

    Motor function will be evaluated using standardized scales, including the general movements assessment (gma) with the motor optimal score (mos‑r) and the hammersmith infant neurologic examination (hine).

    The assessment also contributes to ongoing safety evaluation.

  6. Step 6

    Ongoing safety monitoring (3‑24 months)

    From three months up to twenty‑four months, serious adverse events such as seizures, unplanned hospital visits, and the use of additional medications will be tracked.

    Periodic neurological examinations will be performed according to the study schedule.

  7. Step 7

    Primary and secondary outcome assessment at 24 months

    The primary outcome is the bayley‑iv‑nl motor score measured at twenty‑four months of age.

    Secondary outcomes include the bayley‑iv‑nl cognitive score, evaluation for cerebral palsy, neuromotor delay, epilepsy, and moderate to severe visual or hearing impairment.

  8. Step 8

    Quality‑of‑life questionnaires

    At 3 months, 9‑12 months, and 24 months, you will be asked to complete health‑related quality‑of‑life questionnaires for yourself and for your family.

  9. Step 9

    Economic evaluation

    Data collected up to twenty‑four months will be used for a health technology assessment, including cost‑effectiveness and budget impact analyses.

Who can join the trial?

4 criteria

  • Baby must be born at a gestational age of at least 35 weeks (meaning the pregnancy lasted 35 weeks or more).
  • Baby must have been diagnosed with either perinatal arterial ischemic stroke (PAIS) (a stroke that occurs around the time of birth because a brain artery is blocked) or perinatal asphyxia (PA) (a lack of oxygen at birth) as shown by one or more of the following: a 5‑minute Apgar score of 5 or lower, need for resuscitation, need for a breathing machine for 10 minutes or more after birth, a blood pH less than 7.0, a base excess lower than –16 mmol/L, or a lactate level higher than 10 mmol/L in the umbilical cord or early blood test.
  • Baby must show evidence of brain injury in specific areas on a special MRI scan, such as the posterior limb of the internal capsule, the basal ganglia or thalamus (deep brain regions), the rolandic cortex, the cerebral peduncles, or the white‑matter pathways called the corticospinal tract. The injury is identified by particular patterns on the scan called DWI restriction, abnormal ADC values, or abnormal levels of certain chemicals measured by magnetic resonance spectroscopy (MRS) such as lactate, N‑acetyl‑aspartate (NAA), or the NAA/choline ratio.
  • Parent(s) or legal guardian must sign a written informed consent form agreeing to let the baby take part in the study.

Who cannot join the trial?

3 criteria

  • Suspected chromosomal anomaly (abnormal number or structure of chromosomes), metabolic disorder (problem with the body’s chemical processes), genetic syndrome, birth‑related central nervous system (CNS) malformation (abnormal brain or spinal cord formation), birth‑related CNS infection, or major bleeding inside the skull (intracranial haemorrhage).
  • If the medical team decides to stop intensive NICU care because the infant has very severe brain injury on MRI, needs a breathing machine (ventilation support), is expected to have many severe disabilities (severe multiple handicaps), or has little chance of survival, the infant cannot join the study.
  • Any condition that makes giving medicine through the nose unsafe, such as a blocked nose (nasal obstruction, e.g., choanal atresia where a passage never forms), abnormal nasal septum, nose injury, nosebleeds (epistaxis), excessive mucus or blood in the nose, or damage inside the nose (intranasal damage).
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Investigated drugs

MSC, marrow (bone-marrow derived mesenchymal stromal cell) is a therapy that uses special cells taken from bone‑marrow. These cells are called mesenchymal stromal cells and they have the ability to help repair damaged tissue. In this trial they are given as nasal drops to newborn babies who have suffered a lack of oxygen to the brain (hypoxic‑ischemic brain injury). The goal of the treatment is to reduce brain damage and improve the baby's ability to move and develop normally.

What is already known about the treatment

  • Remestemcel

    This investigational product is made from bone‑marrow derived mesenchymal stromal cells and is given as 0.6 ml of nasal drops. It is currently being studied in clinical trials and is not yet approved for regular medical use. The therapy is intended for newborns with hypoxic‑ischemic brain injury to help protect brain cells and improve motor function. It works by releasing natural growth and anti‑inflammatory signals that support brain repair, and it is classified as a cell‑based regenerative therapy.

  • Potassium Chloride (Placebo)

    In this study potassium chloride is used only as a harmless filler and is administered in the same way as the active product, but it contains no therapeutic cells. It is an already approved electrolyte supplement, but here it serves as a control to compare the effects of the stem cell treatment. The substance has no intended therapeutic effect for brain injury in this trial. It belongs to the class of mineral salts and is used purely as a placebo.

  • 10% Human Serum Albumin (Placebo)

    This protein solution is given as 0.6 ml of nasal drops and acts as a neutral vehicle for the study, containing no active stem cells. Human serum albumin is a well‑known medical product used for volume support, but in this trial it functions only as a placebo. It does not aim to treat the brain injury and is included to keep the administration method the same for all participants. The product is classified as a human plasma protein solution.

Investigated diseases

Hypoxic‑ischemic brain injury - It is damage to a newborn’s brain caused by a lack of oxygen and blood flow around the time of birth. The injury often follows a period of breathing difficulty (perinatal asphyxia) or a stroke in the brain’s arteries (perinatal arterial ischemic stroke). Affected brain tissue may swell, cells can die, and connections between areas are lost. In the following weeks the injured regions may shrink and form scar tissue, while the infant may show delayed motor skills and reduced movement control. As the child grows, the early injury can influence the pattern of brain development seen on imaging.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2025-521506-17-01Estimated enrolment162 patientsSponsorUniversitair Medisch Centrum Utrecht

sourced from the EU Clinical Trials Register and site verification

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