152. ＰＣＤＨ19関連症候群 ［臨床試験数：3，薬物数：5（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 of the data. Export date: 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03865732 | May 17, 2019 | 11 November 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 | Recruiting | 1 Year | 17 Years | Female | 70 | Phase 3 | United States;Australia;France;Italy | |
| 2 | EUCTR2015-001324-36-IT | 05/06/2015 | 10 July 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 Pharmaceutical Form: Oral suspension INN or Proposed INN: GANAXOLONE CAS Number: 38398-32-2 Current Sponsor code: CCD 1042 Other descriptive name: Ganaxolone, SPT3162, MD 9150000, CCD-1042, Mepalon 1042, (3a-hydroxy-3ß-methyl-5a-pregnan-20-one) Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- Product Name: ganaxolone Product Code: CCD 1042 Pharmaceutical Form: Capsule INN or Proposed INN: GANAXOLONE CAS Number: 38398-32-2 Current Sponsor code: CCD 1042 Other descriptive name: Ganaxolone, SPT3162, MD 9150000, CCD-1042, Mepalon 1042, (3a-hydroxy-3ß-methyl-5a-pregnan-20-one) Concentration unit: mg milligram(s) Concentration type: range Concentration number: 200-225 | Marinus Pharmaceuticals, Inc. | Authorised | Female: yes Male: no | 10 | United States;Italy | ||||
| 3 | NCT02358538 | March 2015 | 15 July 2019 | 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 | Not recruiting | 2 Years | 18 Years | All | 30 | Phase 2 | United States;Italy |