2025-521506-17-01招募中2 期
iSTOP-CP: intranasal Stem Cells to treat Perinatal brain injury to combat Cerebral Palsy
Universitair Medisch Centrum Utrecht1 个研究点 分布在 1 个国家目标入组 162 人开始时间: 2026年10月1日最近更新:
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 162
- 试验地点
- 1
- 主要终点
- Bayley-IV-NL motor score assessed at 24 months of age.
研究概览
简要总结
The main objective of this study is to determine whether intranasal stem cell therapy (IN-MSC) can effectively reduce brain damage and improve motor outcomes in newborns with hypoxic-ischemic brain injury (HIBI). By conducting a placebo-controlled phase II clinical trial in infants with MRI confirmed HIBI, this trial wants to see if the treatment is effective and safe with the goal to eventually incorporate the therapy in standard clinical care.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Neonate with a gestational age ≥35.0 weeks at birth
- •Either a PAIS (a neurologic condition characterized by focal cerebral ischemia and infarction following blockage of a brain artery with impairment of blood supply and oxy-genation of brain tissue, clinically characterized by seizures (clinical or subclini-cal), respiratory difficulties, or both) or PA (defined as at least one out of the following: 5-min Apgar score ≤ 5, Resuscitation, Mechanical ventilation following resuscitation ≥ 10 minutes after birth, pH <7.0, BE <-16 mmol/L, or lactate > 10.0 mmol/L in umbilical cord blood sample, or in arterial, venous or capillary blood gas sample obtained within 1 hour after birth) diagnosis
- •Showing signs of hypoxic-ischemic injury in 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): PLIC (posterior limb of the internal capsule), central gray matter (BGT: basal ganglia and/or thalami), rolandic cortex, cerebral peduncles, white matter involving CST (corticospinal tract)
- •Written informed consent from custodial parent(s)
排除标准
- •Suspicion of chromosomal anomaly, metabolic disorder, genetic syndrome, congenital central nervous system (CNS) malformation, congenital CNS infection and main injury intracranial haemorrhage
- •Once a clinical team decides on withdrawal of NICU care, the patient is not eligible for inclusion: infants with very severe brain injury on MRI and need for ventilation support (not breathing independently), who have a prognosis of severely multiple handicaps and/or no realistic prospect of survival at the discretion of the attending physician, will not be eligible to iSTOP-CP, to avoid unnecessary suffering and an unacceptable quality of life
- •Contraindications for intranasal administration of medication, including nasal obstruction (e.g. choanal atresia), nasal septal abnormalities, nasal trauma, epistaxis, excessive nasal mucus or blood, and intranasal damage
结局指标
主要结局
Bayley-IV-NL motor score assessed at 24 months of age.
Bayley-IV-NL motor score assessed at 24 months of age.
次要结局
- Cognitive development at 2 years, assessed using the cognitive composite Score of the Bayley-IV-NL
- At 2 years of age, sensorineural disability will be assessed based on the presence of at least one of the following: cerebral palsy (GMFCS ≥1), neuromotor delay (Bayley-IV-NL motor score < -1 SD), cognitive delay (Bayley-IV-NL cognitive score < -1 SD), epilepsy (per ILAE definition), or moderate to severe visual or hearing impairment due to PA or PAIS.
- At 3–4 months of age, safety will be evaluated by tracking adverse events occurring between IN-MSC administration and 3 months, and by assessing potential effects on the brain, including tumor formation, through MRI at 52 weeks postmenstrual age.
- Between 3 and 24 months of age, safety will be monitored by tracking serious adverse events (e.g., seizures, unplanned hospital visits), use of additional medications, and any unexpected deviations in neurological development based on earlier MRI findings, using standardized assessments (GMA with MOS-R and HINE)
- For infants with HIBI due to PA, the effect of IN-MSCs on white matter integrity will be assessed at 52 weeks PMA using DTI-MRI and tract-based FA analysis. For infants with PAIS, changes in brain tissue volume between the early neonatal MRI (<8 days) and MRI at 52 weeks PMA will be evaluated.
- Differences in health-related quality of life (HRQoL) for patients and families will be assessed at 3, 9–12, and 24 months using questionnaires: EQ-5D-5L, PCL-5, LTO, KLIK Pain, CIS-4, and WEMWBS for parents/caregivers, and EQ-TIPS and PedsQL-Infant for infants
- The economic impact at 24 months will be evaluated through a Health Technology Assessment, including a cost of illness study, societal return on investment, cost-effectiveness analysis, and budget impact analysis.
研究者
Clinical trial contact
Scientific
Universitair Medisch Centrum Utrecht
研究点 (1)
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