140. ドラベ症候群 Dorabe syndrome Clinical trials / Disease details
臨床試験数 : 104 / 薬物数 : 61 - (DrugBank : 14) / 標的遺伝子数 : 48 - 標的パスウェイ数 : 63
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2016-000474-38-ES (EUCTR) | 31/05/2017 | 07/04/2017 | A Multicenter, Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 Oral Solution When Added to Standard of Care, Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to standar treatment in Children and Young Adults with Dravet Syndrome | A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2) | Dravet's syndrome MedDRA version: 19.1;Level: LLT;Classification code 10073682;Term: Dravet syndrome;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE | Zogenix International Limited | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | France;United States;Canada;Spain;Netherlands;Germany;United Kingdom | ||
| 2 | EUCTR2016-000474-38-GB (EUCTR) | 06/04/2017 | 17/01/2017 | A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2) | A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2) | Dravet's syndrome MedDRA version: 20.0;Level: LLT;Classification code 10073682;Term: Dravet syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE | Zogenix International Limited | NULL | Not Recruiting | Female: yes Male: yes | 110 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Spain;Netherlands;Germany;United Kingdom | ||
| 3 | EUCTR2016-000474-38-DE (EUCTR) | 31/03/2017 | 27/12/2016 | A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2) | A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2) | Dravet's syndrome MedDRA version: 20.0;Level: LLT;Classification code 10073682;Term: Dravet syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE | Zogenix International Limited | NULL | Not Recruiting | Female: yes Male: yes | 110 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Spain;Netherlands;Germany;United Kingdom | ||
| 4 | EUCTR2016-000474-38-NL (EUCTR) | 13/09/2016 | 30/03/2016 | A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2) | A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2) | Dravet's syndrome MedDRA version: 18.1;Level: LLT;Classification code 10073682;Term: Dravet syndrome;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE Product Name: Fenfluramine hydrochloride Product Code: ZX008 INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE | Zogenix International Limited | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | Spain;Germany;Netherlands;United Kingdom | ||
| 5 | NCT02926898 (ClinicalTrials.gov) | September 2016 | 10/8/2016 | A Two-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children =2 Years Old and Young Adults With Dravet Syndrome | A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care , Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults With Dravet Syndrome | Dravet Syndrome | Drug: ZX008 - 0.2 mg/kg/day;Drug: ZX008 - 0.4 mg/kg/day;Drug: ZX008 - 20 mg/day maximum dose;Drug: Matching Placebo | Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. | NULL | Completed | 2 Years | 18 Years | All | 105 | Phase 3 | United States;Canada;France;Germany;Netherlands;Spain;United Kingdom |
| 6 | NCT02239276 (ClinicalTrials.gov) | September 2014 | 10/9/2014 | Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies | Expanded Access Use of Stiripentol in Participants With Dravet Syndrome or Epileptic Encephalopathies Associated With Sodium Channel Mutations | Dravet Syndrome;Epileptic Encephalopathies Associated With SCN1A Mutations | Drug: Stiripentol | Cook Children's Health Care System | NULL | No longer available | 6 Months | 18 Years | All | United States | ||
| 7 | NCT01983722 (ClinicalTrials.gov) | October 2013 | 7/11/2013 | Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome | Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome | Dravet Syndrome | Drug: Stiripentol | Children's Hospital Medical Center, Cincinnati | NULL | Approved for marketing | 6 Months | N/A | All | United States | ||
| 8 | NCT01835314 (ClinicalTrials.gov) | May 2013 | 15/4/2013 | Compassionate Use of Stiripentol in Dravet Syndrome | Compassionate Use of Stiripentol in Dravet Syndrome | Dravet Syndrome | Drug: Stiripentol | University of Colorado, Denver | NULL | No longer available | 1 Year | 21 Years | All | United States | ||
| 9 | JPRN-JapicCTI-205180 | 29/11/2012 | 25/02/2020 | Post-marketing surveillance of DIACOMITCapsule, Powder for Oral Suspension | Post-marketing surveillance of DIACOMIT (Capsule: 250 mg or 500 mg, Powder for Oral Suspension: 250 mg or 500 mg) | Dravet Syndrome | Intervention name : DIACOMIT (Stiripentol) INN of the intervention : stiripentol Dosage And administration of the intervention : Usually, for patients aged 1 year or older, 50 mg/kg of stiripentol per day is orally administered in divided doses two to three times a day during or immediately after a meal. Dosing starts at 20 mg/kg daily and is increased by 10 mg/kg at intervals of at least one week. However, for patients weighing 50 kg or more, start taking 1000 mg of stiripentol daily, and increase the dosage by 500 mg at intervals of one week or more. The maximum daily dose shall not exceed 50 mg/kg or 2500 mg, whichever is lower. Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : - | Meiji Seika Pharma Co., Ltd. | NULL | recruiting | BOTH | 300 | NA | Japan | ||
| 10 | NCT01533506 (ClinicalTrials.gov) | February 2012 | 9/2/2012 | Stiripentol in Dravet Syndrome | Compassionate Use of Stiripentol in Intractable Epilepsy Due to Dravet Syndrome | Dravet Syndrome | Drug: stiripentol | Mayo Clinic | NULL | No longer available | 11 Years | N/A | Female | Phase 4 | United States | |
| 11 | JPRN-JapicCTI-101116 | 01/4/2010 | 28/04/2010 | Open Phase III Study of ME2080 in Patients with Dravet Syndrome | Open Phase III Study of ME2080 in Patients with Dravet Syndrome | Dravet Syndrome | Intervention name : ME2080 (stiripentol) Dosage And administration of the intervention : 50mg/kg/day administered twice or 3 times a day (maximum 2500mg/day) | Meiji Seika Pharma Co., Ltd. (formerly MEIJI SEIKA KAISHA Co., LTD.) | NULL | 1 | 30 | BOTH | 25 | Phase 3 | NULL | |
| 12 | JPRN-JMA-IIA00014 | 01/10/2007 | 06/11/2007 | Use of the antiepileptic drug stiripentol for the treatment of severe myoclonic epilepsy in infancy (SMEI) | Clinical evaluation of stiripentol in Dravet syndrome | Dravet syndrome (severe myoclonic epilepsy in infancy: SMEI) | Intervention type:DRUG. Intervention1:Stiripentol, Dose form:FINE GRANULES, Route of administration:ORAL, intended dose regimen:Stiripentol will be started at 50 mg/kg (or 1000 mg for patients weighing 20 kg or more) and will be titrated up to 100 mg/kg (maximum of 4.0 g).. | Yushi InoueNational Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorders | Shunya IkedaDepartment of Pharmaceutical Sciences, International University of Health and WelfareYoko OhtsukaOkayama University, Graduate School of Medicine, Dentistry and Pharmaceutical SciencesHirokazu OguniDepartment of Pediatrics, Tokyo Women's Medical UniversityYukitoshi TakahashiNational Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological DisordersJun TohyamaNational Hospital Organization Nishi-Niigata Chuo National HospitalHiroshi BabaNational Hospital Organization Nagasaki Medical CenterKatsuyuki FukushimaNational Hospital Organization Yakumo Hospital | Completed | >=1 YEARS | BOTH | 10 | NOT APPLICABLE | Japan | |
| 13 | EUCTR2007-001784-30-FR (EUCTR) | 19/07/2007 | 01/06/2007 | Population pharmacokinetic study in Dravet's syndrome patients treated with stiripentol, valproate and clobazam. Open pharmacokinetic multicenter study. | Population pharmacokinetic study in Dravet's syndrome patients treated with stiripentol, valproate and clobazam. Open pharmacokinetic multicenter study. | Antiepileptic | Trade Name: DIACOMIT Product Name: DIACOMIT Trade Name: DIACOMIT Product Name: DIACOMIT | BIOCODEX | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | France | ||||
| 14 | EUCTR2007-003702-95-IT (EUCTR) | 03/07/2007 | 07/09/2007 | A MULTICENTRE RANDOMIZED CONTROLLED TRIAL COMPARING TOPIRAMATE, STIRIPENTOL AND CLOBAZAM AS ADJUNCTIVE THERAPY TO VALPROATE AND CLOBAZAM IN PAEDIATRIC PATIENTS WITH DRAVET'S SYNDROME (SMEI) NOT ADEQUATELY CONTROLLED WITH CLOBAZAM AND VALPROATE, AND AUXILIARY PHARMACOGENETIC STUDY - ND | A MULTICENTRE RANDOMIZED CONTROLLED TRIAL COMPARING TOPIRAMATE, STIRIPENTOL AND CLOBAZAM AS ADJUNCTIVE THERAPY TO VALPROATE AND CLOBAZAM IN PAEDIATRIC PATIENTS WITH DRAVET'S SYNDROME (SMEI) NOT ADEQUATELY CONTROLLED WITH CLOBAZAM AND VALPROATE, AND AUXILIARY PHARMACOGENETIC STUDY - ND | Dravet Syndrome Level: PTClassification code 10054859 | Trade Name: stiripentolo Trade Name: TOPAMAX INN or Proposed INN: Topiramate Trade Name: FRISIUM*30CPS 10MG INN or Proposed INN: Clobazam | ISTITUTO C. MONDINO | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Italy | |||
| 15 | EUCTR2007-002198-30-IT (EUCTR) | 03/07/2007 | 17/01/2012 | A MULTICENTRE RANDOMIZED CONTROLLED TRIAL COMPARING TOPIRAMATE, STIRIPENTOL AND CLOBAZAM AT THE MAXIMAL TOLERATED DOSAGE, AS ADJUNCTIVE THERAPY TO VALPROATE AND CLOBAZAM IN PAEDIATRIC PATIENTS WITH DRAVET`S SYNDROME (SMEI), AND AUXILIARY PHARMACOGENETIC STUDY | A MULTICENTRE RANDOMIZED CONTROLLED TRIAL COMPARING TOPIRAMATE, STIRIPENTOL AND CLOBAZAM AT THE MAXIMAL TOLERATED DOSAGE, AS ADJUNCTIVE THERAPY TO VALPROATE AND CLOBAZAM IN PAEDIATRIC PATIENTS WITH DRAVET`S SYNDROME (SMEI), AND AUXILIARY PHARMACOGENETIC STUDY | Dravet Syndrome MedDRA version: 14.1;Level: PT;Classification code 10054859;Term: Myoclonic epilepsy;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: stiripentolo INN or Proposed INN: Other antiepileptics Trade Name: TOPAMAX INN or Proposed INN: Topiramate Trade Name: FRISIUM*30CPS 10MG INN or Proposed INN: Clobazam | Inserm-ISP Pole Recherches cliniques et thérapeutiques | NULL | Not Recruiting | Female: yes Male: yes | 90 | Italy |