CTIS2023-509672-42-00招募中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 - RAD-GRIN-101
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 25
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 17（—）
- 性别
- All
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 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 (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. 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 from heterosexual intercourse 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. 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 GRINrelated 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. 6. Participant with any contraindications to radiprodil or with known hypersensitivity to the active substance or the excipients or other chemically closely related substances. 7. Participant receiving treatment with contraindicated concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to lamotrigine, felbamate, memantine, and perampanel. 8. Participant is on treatment with hormonal therapy such as adrenocorticotrophic hormone or prednisolone (Part A only). 9. Participant has participated in any other investigational clinical study within 3 months of Screening (Part A only).
研究者
相似试验
进行中（未招募）
1 期
Study with Radiprodil in Children with GRIN-related DisorderEUCTR2022-000317-14-DEGRIN Therapeutics, Inc.24
进行中（未招募）
1 期
Study with Radiprodil in Children with GRIN-related Disordergain of function (GoF) variants in the GRIN2B geneMedDRA version: 21.1Level: PTClassification code 10064062Term: Neurodevelopmental disorderSystem Organ Class: 10037175 - Psychiatric disordersEUCTR2022-000317-14-NLGRIN Therapeutics, Inc.12
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