DDrare: Database of Drug Development for Rare Diseases

Plasma from healthy young people treatment + riluzole (DrugBank: Riluzole)

8 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
2	筋萎縮性側索硬化症	1
3	脊髄性筋萎縮症	0
5	進行性核上性麻痺	0
8	ハンチントン病	0
13	多発性硬化症／視神経脊髄炎	0
17	多系統萎縮症	0
18	脊髄小脳変性症（多系統萎縮症を除く。）	0
206	脆弱X症候群	0

2. 筋萎縮性側索硬化症　［臨床試験数：508，薬物数：530（DrugBank：146），標的遺伝子数：170，標的パスウェイ数：221］

Searched query = "Amyotrophic lateral sclerosis", "ALS"
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.

1 / 508 trial found

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	NCT04454840 (ClinicalTrials.gov)	May 1, 2016	29/6/2020	Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis	A Clinical Study on Safety and Effectiveness of Intravenous Infusion Plasma From Healthy Young People to Treat Amyotrophic Lateral Sclerosis	Amyotrophic Lateral Sclerosis	Biological: Plasma from healthy young people treatment + Riluzole;Drug: Riluzole	Peking University Third Hospital	NULL	Completed	N/A	N/A	All	20	Early Phase 1	China

3. 脊髄性筋萎縮症　［臨床試験数：179，薬物数：102（DrugBank：26），標的遺伝子数：52，標的パスウェイ数：78］

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 179 trial found

5. 進行性核上性麻痺　［臨床試験数：82，薬物数：107（DrugBank：36），標的遺伝子数：60，標的パスウェイ数：90］

Searched query = "Progressive supranuclear palsy", "PSP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 82 trial found

8. ハンチントン病　［臨床試験数：197，薬物数：171（DrugBank：54），標的遺伝子数：82，標的パスウェイ数：144］

Searched query = "Huntington disease", "Huntington chorea"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 197 trial found

13. 多発性硬化症／視神経脊髄炎　［臨床試験数：3,050，薬物数：2,147（DrugBank：348），標的遺伝子数：244，標的パスウェイ数：228］

Searched query = "Multiple sclerosis/Neuromyelitis optica", "Multiple sclerosis", "Neuromyelitis optica", "MS", "NMOSD", "Devic disease", "Balo concentric sclerosis", "Baló concentric sclerosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 3,050 trial found

17. 多系統萎縮症　［臨床試験数：107，薬物数：153（DrugBank：48），標的遺伝子数：59，標的パスウェイ数：104］

Searched query = "Multiple system atrophy", "MSA-C", "MSA-P", "Olivopontocerebellar atrophy", "OPCA", "Striatonigral degeneration", "Shy-Drager syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 107 trial found

18. 脊髄小脳変性症（多系統萎縮症を除く。）　［臨床試験数：59，薬物数：78（DrugBank：28），標的遺伝子数：44，標的パスウェイ数：59］

Searched query = "Spinocerebellar degeneration", "SCD", "Spinocerebellar ataxia type I", "SCA1", "Spinocerebellar ataxia type II", "SCA2", "Spinocerebellar ataxia type III", "SCA3", "Machado-Joseph disease", "Spinocerebellar ataxia type VI", "SCA6", "Spinocerebellar ataxia type VII", "SCA7", "Spinocerebellar ataxia type X", "SCA10", "Spinocerebellar ataxia type XII", "SCA12", "Dentatorubural pallidoluysian atrophy", "Dentatorubropallidoluysial atrophy", "DRPLA", "Naito-Koyanagi disease", "Friedreich ataxia", "FRDA", "Ataxia with vitamin E deficiency", "AVED", "Early-onset ataxia with ocular motor ataxia and hypoalbuminemia", "EOAH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 59 trial found

206. 脆弱X症候群　［臨床試験数：98，薬物数：73（DrugBank：27），標的遺伝子数：46，標的パスウェイ数：57］

Searched query = "Fragile X syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 98 trial found