NL-OMON53530尚未招募不适用
A Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms of Multiple Individually Titrated Doses of Radiprodil in Children with GRIN-related Disorder. - RAD-GRIN-101 study
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 4
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 15（—）
入选标准
- •For Part A and Part B (unless stated otherwise):
- •1. For Part A, pediatric participants aged >=6 months to <=12 years with GRIN 1,
- •2A, 2B, or 2D gene variants known to result in GoF of the NMDA receptor. For
- •Part B, pediatric participants aged >=6 months with GRIN 1, 2A, 2B, or 2D gene
- •variants known to result in GoF of the NMDA receptor.
- •2. Participant to be enrolled in the first cohort experiences the following
- •(Part A only):
- •a) At least 1 observable motor seizure per week and >=4 observable motor
- •seizures (generalized or focal) during the prospective 4-week Observation
- •b) Has failed to obtain adequate seizure control with at least 2 ASMs used at
- •appropriate dose and duration with assured medication adherence (if applicable).
- •3. Participant to be enrolled in the second cohort experiences the following
- •(Part A only):
- •a) Significant behavioral and/or motor symptoms based on caregiver report with
- •a CGI-S score >=4 at the Screening Visit and Day -1 of Visit T1.
- •4. For part A, current therapies need to be on a stable dose for at least 4
- •weeks prior to Screening and should be maintained stable throughout the whole
- •study duration, nonpharmacological treatments such as ketogenic diet should be
- •kept as stable as possible during screening and participation in the study.
- •Changes in antiseizure medication should be discussed with the sponsor in
- •consultation with the investigator.
- •For all inclusion criteria see Protocol.
- •Rescreening criteria for Part B only:
- •1. Participant has received at least 8 weeks of treatment (combined Titration
- •and Maintenance Period) with radiprodil during Part A.
- •2. The benefit-risk of continuing radiprodil treatment remains favorable as
- •determined by the investigator*s clinical assessment and is eligible to
- •continue treatment according to the judgement of the investigator.
排除标准
- •Exclusion criteria (at initial Screening and following completion of the
- •Maintenance Period of Part A, unless stated otherwise): 1. Participant with any
- •other clinically relevant medical, neurologic, or psychiatric condition and/or
- •behavioral disorder unrelated to GRIN related disorders that would preclude or
- •jeopardize participant*s safe participation or administration of study drug or
- •the conduct of the study according to the judgement of the investigator. 2.
- •Participant with a body weight <10 kg on Day -1 of Visit T1 for whom a gastric
- •tube is the only possibility for radiprodil dosing (during treatment with the
- •first dose in Part A only). 3. Participant with any clinically significant
- •laboratory or ECG abnormalities. 4. Participant has severe hepatic dysfunction
- •(Child-Pugh grade C). 5. Participant has a history of brain surgery for
- •epilepsy or any other reason. For all exclusion criteria see Protocol.
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