NL-OMON51988尚未招募2 期
A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy) (GALax-C) - Study of Safety, Tolerability and Efficacy of PBKR03
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 1（—）
入选标准
- •1. >= 1 month and <9 months of age at enrollment, and are presymptomatic or
- •symptomatic with first symptoms of Krabbe disease at <=6 months of age
- •2. Leukocyte GALC activity <=lower limit of normal (LLN)
- •3. Whole blood (dried blood spot) psychosine > 10 nM
- •4. Biallelic pathogenic GALC gene variants associated with early infantile
- •Krabbe disease or variants classified as likely pathogenic (testing must be
- •done at a Clinical Laboratory Improvement Amendments [CLIA] or CLIA-equivalent
- •laboratory certified per local standard. If the GALC gene analysis is performed
- •in the UK or the European Union (EU) a Conformité Européenne (CE) marked test
- •will be used). See also Appendix 2 of Protocol, Classification of GALC Gene
- •Note: Subjects without documentation of two pathogenic or likely pathogenic
- •GALC variants but who meet all other inclusion criteria, including low GALC
- •activity and high psychosine level, may be considered eligible for the study.
- •In this case the totality of available data, including relevant family history,
- •must be consistent with a diagnosis of early infantile Krabbe disease. For the
- •full list of inclusion criteria see chapter 6.1 of the protocol
排除标准
- •1. Any clinically significant neurocognitive deficit not attributable to Krabbe
- •2. An acute illness requiring hospitalization within 30 days of enrollment that
- •in the opinion of the investigator would interfere with the evaluation of the
- •investigational product or interpretation of subject safety or study results.
- •3. History of chronic ventilation assisted respiratory support (defined as use
- •of more than 12 hours/day of bilevel positive airway pressure, continuous
- •positive airway pressure, or ventilator) or a need for tracheostomy as a result
- •of their disease. Note: This does not exclude subjects who use respiratory
- •4. Intractable seizure or uncontrolled epilepsy defined as having had an
- •episode of status epilepticus, or seizures requiring hospitalization.
- •a. This does not exclude subjects who have a history of staring spells that
- •have not been associated with EEG findings
- •5. Family history of seizure disorders or epilepsy of infantile or childhood
- •onset, other than febrile seizures
- •a. This does not exclude subjects with a family history of Krabbe disease
- •6. Any contraindication to ICM administration procedure, including
- •contraindications to fluoroscopic imaging, IT contrast and anesthesia, or any
- •condition that would increase the risk of adverse outcomes from the ICM
- •procedure, including but not limited to the presence of space occupying lesion
- •causing mass effect or signs of increased intracranial pressure,
- •non-communicating hydrocephalus, space-occupying lesion in the posterior fossa
- •or foramen magnum, aberrant vascular anatomy such as a large midline posterior
- •inferior cerebellar artery, venous anomaly such as a large midline cerebellar
- •vein or occipital sinus, congenital anatomical abnormalities such as Chiari
- •malformation.For the full list of exclusion criteria see chapter 6.2 of the
研究者
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