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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
9 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-004496-12-GB
(EUCTR)
14/01/202018/11/2019A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsyA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: no
70Phase 3United States;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Belgium;Poland;Australia;Denmark;Germany;Netherlands
2EUCTR2018-004496-12-HU
(EUCTR)
13/01/202004/11/2019A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsyA 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.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: no
70Phase 3United States;Spain;Israel;United Kingdom;Italy;France;Hungary;Czech Republic;Poland;Belgium;Australia;Denmark;Germany;Netherlands
3EUCTR2018-004496-12-FR
(EUCTR)
26/09/201929/04/2019A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsyA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: no
50Phase 3United States;France;Hungary;Poland;Spain;Australia;Netherlands;Italy;United Kingdom
4EUCTR2018-004496-12-NL
(EUCTR)
23/09/201905/06/2019A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsyA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: no
30Phase 2United States;Czechia;Spain;Israel;United Kingdom;Italy;France;Hungary;Belgium;Poland;Australia;Denmark;Netherlands;Germany
5NCT03865732
(ClinicalTrials.gov)
May 17, 20195/3/2019Study 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 EpilepsyDrug: Ganaxolone;Drug: PlaceboMarinus PharmaceuticalsNULLActive, not recruiting1 Year17 YearsFemale25Phase 2United States;Hungary;Italy;Netherlands;Poland;Australia;France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2015-001324-36-IT
(EUCTR)
05/06/201516/04/2015Pilot, 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: no
10United States;Italy
7NCT02358538
(ClinicalTrials.gov)
March 201530/1/2015A Multicenter, Open-Label Proof-of-Concept Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic EpilepsiesA 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 TreatmentEpilepsyDrug: GanaxoloneMarinus PharmaceuticalsNULLActive, not recruiting2 Years18 YearsAll30Phase 2United States;Italy
8EUCTR2018-004496-12-PL
(EUCTR)
01/08/2019A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsyA 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.NULLNAFemale: yes
Male: no
30Phase 2United States;Czechia;Spain;Ireland;Italy;United Kingdom;France;Hungary;Poland;Belgium;Australia;Denmark;Germany;Netherlands
9EUCTR2018-004496-12-DK
(EUCTR)
07/11/2019A clinical study with an investigational drug called ganaxolone in female children with protocadherin 19 (PCDH19)-related epilepsyA 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.NULLNAFemale: yes
Male: no
70Phase 3United States;Spain;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Poland;Belgium;Denmark;Australia;Germany;Netherlands