EUCTR2022-000317-14-NL进行中（未招募）1 期
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
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
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
- 性别
- All
入选标准
- •1.Pediatric participants aged =6 months to =12 years with GRIN2B gene variants known to result in GoF of the NMDA receptor.
- •2.Participant to be enrolled in the first cohort experiences the following:
- •a)At least 1 observable motor seizure per week and =4 observable motor seizures (generalized or focal) during the prospective 4 week Observation Period.
- •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:
- •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.Current antiseizure therapies should be maintained stable throughout the whole study duration, and nonpharmacological treatments such as ketogenic diet should be kept as stable as possible during the participation in the study.
- •5.Participant’s caregivers have signed informed consent and participant has signed assent (if applicable).
- •6.Participant’s caregivers are willing and able to complete entries in the eDiary on a daily basis.
- •7.Participant is 1 of the following:
- •a.Not of childbearing potential (premenarchal or male/not in possession of a uterus).
- •b.If of childbearing potential, is nonpregnant (negative serum pregnancy test results at Screening), nonlactating, and practicing 1 of the following medically acceptable methods of birth control:
- •Abstinence as a lifestyle choice.
- •Hormonal methods such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant’s usual menstrual cycle period) before IP administration.
- •Intrauterine device.
- •c.If male, is willing to use a highly effective method of contraception throughout the study period.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 12
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Participant with any other clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder unrelated to GRIN2B that would preclude or jeopardize participant’s safe participation or the conduct of the study according to the judgement of the investigator.
- •2.Participant with any clinically significant laboratory or ECG abnormalities.
- •3. Participant has severe hepatic dysfunction (Child-Pugh grade C).
- •4.Participant has a history of brain surgery for epilepsy or any other reason.
- •5.Participant with any contraindications to radiprodil or with known hypersensitivity to the active substance or the excipients or other chemically closely related substances.
- •6.Participant receiving treatment with contraindicated concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to felbamate, memantine, and perampanel.
- •7.Participant is on treatment with hormonal therapy such as adrenocorticotrophic hormone or prednisolone.
- •8.Participant receiving treatment with marijuana or tetrahydrocannabinol or derivatives (the use of Epidiolex® is permitted).
- •9.Participant has participated in any other investigational clinical study within 3 months of Screening.
- •10.Participant has previously been enrolled in the current study
研究者
相似试验
招募中
1 期
A Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics,and Effect on Seizures and Behavioral Symptoms of Multiple IndividuallyTitrated Doses of Radiprodil in Children with GRIN-related DisorderCTIS2023-509672-42-00Grin Therapeutics Inc.25
进行中（未招募）
1 期
Study with Radiprodil in Children with GRIN-related DisorderEUCTR2022-000317-14-DEGRIN Therapeutics, Inc.24
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.NL-OMON53530GRIN Therapeutics, Inc.4
进行中（未招募）
1 期
Safety and tolerability during open-label treatment with LCZ696 in patients with chronic heart failure and reduced ejection fractioHeart failure with reduced ejection fractionMedDRA version: 17.1Level: LLTClassification code 10019279Term: Heart failureSystem Organ Class: 100000004849EUCTR2014-001971-30-ITovartis Farma SPA5,000
进行中（未招募）
1 期
Safety and tolerability during open-label treatment with LCZ696 in patients with chronic heart failure and reduced ejection fractioEUCTR2014-001971-30-FIovartis Pharma services AG5,000