152. PCDH19関連症候群
［臨床試験数：9，薬物数：7（DrugBank：1），標的遺伝子数：16，標的パスウェイ数：7］
Searched query = "PCDH19 related syndrome", "PCDH19 Epilepsy", "Epilepsy and mental retardation limited to females", "PCDH19 female pediatric epilepsy", "PCDH19-related epilepsy", "Protocadherin 19 (PCDH19)-related epilepsy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | EUCTR2018-004496-12-GB (EUCTR) | 14/01/2020 | 18/11/2019 | A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsy | A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment | protocadherin 19 (PCDH19)-related epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10032061;Term: Other forms of epilepsy;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Ganaxolone Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT3162 INN or Proposed INN: GANAXOLONE | Marinus Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: no | 70 | Phase 3 | United States;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Belgium;Poland;Australia;Denmark;Germany;Netherlands | ||
| 2 | EUCTR2018-004496-12-HU (EUCTR) | 13/01/2020 | 04/11/2019 | A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsy | A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment | protocadherin 19 (PCDH19)-related epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10032061;Term: Other forms of epilepsy;System Organ Class: 100000004852 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Marinus Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: no | 70 | Phase 3 | United States;Spain;Israel;United Kingdom;Italy;France;Hungary;Czech Republic;Poland;Belgium;Australia;Denmark;Germany;Netherlands | |||
| 3 | EUCTR2018-004496-12-FR (EUCTR) | 26/09/2019 | 29/04/2019 | A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsy | A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment | protocadherin 19 (PCDH19)-related epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10032061;Term: Other forms of epilepsy;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Ganaxolone Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT316 INN or Proposed INN: GANAXOLONE | Marinus Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: no | 50 | Phase 3 | United States;France;Hungary;Poland;Spain;Australia;Netherlands;Italy;United Kingdom | ||
| 4 | EUCTR2018-004496-12-NL (EUCTR) | 23/09/2019 | 05/06/2019 | A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsy | A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment | protocadherin 19 (PCDH19)-related epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10032061;Term: Other forms of epilepsy;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Ganaxolone Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT3162 INN or Proposed INN: GANAXOLONE | Marinus Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: no | 30 | Phase 2 | United States;Czechia;Spain;Israel;United Kingdom;Italy;France;Hungary;Belgium;Poland;Australia;Denmark;Netherlands;Germany | ||
| 5 | NCT03865732 (ClinicalTrials.gov) | May 17, 2019 | 5/3/2019 | Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study) | A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy Followed by Long-term Open-label Treatment. | PCDH19-Related Epilepsy | Drug: Ganaxolone;Drug: Placebo | Marinus Pharmaceuticals | NULL | Active, not recruiting | 1 Year | 17 Years | Female | 25 | Phase 2 | United States;Hungary;Italy;Netherlands;Poland;Australia;France |
| 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 |
| 6 | EUCTR2015-001324-36-IT (EUCTR) | 05/06/2015 | 16/04/2015 | Pilot, multicenter study to evaluate the efficacy of the ganaxolone in female children with PCDH19 Pediatric Epilepsy. | A Multicenter, Open-Label Proof-of-Concept Trial of Ganaxolone in Children with PCDH19 Female Pediatric Epilepsy | PCDH19 Female Pediatric Epilepsy (FPE) MedDRA version: 18.0;Level: LLT;Classification code 10032062;Term: Other forms of epilepsy, with intractable epilepsy;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: ganaxolone Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT316 INN or Proposed INN: GANAXOLONE Other descriptive name: Ganaxolone, SPT3162, MD 9150000, CCD-1042, Mepalon 1042, (3a-hydroxy-3ß-methyl-5a-pregnan-20-one) Product Name: ganaxolone Product Code: CCD 1042 INN or Proposed INN: GANAXOLONE Other descriptive name: Ganaxolone, SPT3162, MD 9150000, CCD-1042, Mepalon 1042, (3a-hydroxy-3ß-methyl-5a-pregnan-20-one) | Marinus Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: no | 10 | United States;Italy | |||
| 7 | NCT02358538 (ClinicalTrials.gov) | March 2015 | 30/1/2015 | A Multicenter, Open-Label Proof-of-Concept Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies | A Multicenter, Open-Label Proof-of-Concept Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies Followed by 52 Week Open-Label Treatment | Epilepsy | Drug: Ganaxolone | Marinus Pharmaceuticals | NULL | Active, not recruiting | 2 Years | 18 Years | All | 30 | Phase 2 | United States;Italy |
| 8 | EUCTR2018-004496-12-DK (EUCTR) | 07/11/2019 | A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsy | A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment | protocadherin 19 (PCDH19)-related epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10032061;Term: Other forms of epilepsy;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Ganaxolone Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT316 INN or Proposed INN: GANAXOLONE | Marinus Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: no | 70 | Phase 3 | United States;Spain;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Poland;Belgium;Denmark;Australia;Germany;Netherlands | |||
| 9 | EUCTR2018-004496-12-PL (EUCTR) | 01/08/2019 | A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsy | A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment | protocadherin 19 (PCDH19)-related epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10032061;Term: Other forms of epilepsy;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Ganaxolone Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT3162 INN or Proposed INN: GANAXOLONE | Marinus Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: no | 30 | Phase 2 | United States;Czechia;Spain;Ireland;Italy;United Kingdom;France;Hungary;Poland;Belgium;Australia;Denmark;Germany;Netherlands |