DDrare: Database of Drug Development for Rare Diseases

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

8 diseases

ID	Disease name (Link within this page)	Number of trials
2	Amyotrophic lateral sclerosis	1
3	Spinal muscular atrophy	0
5	Progressive supranuclear palsy	0
8	Huntington disease	0
13	Multiple sclerosis/Neuromyelitis optica	0
17	Multiple system atrophy	0
18	Spinocerebellar degeneration	0
206	Fragile X syndrome	0

2. Amyotrophic lateral sclerosis [ 508 clinical trials, 530 drugs, (DrugBank: 146 drugs), 170 drug target genes, 221 drug target pathways]

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. Spinal muscular atrophy [ 179 clinical trials, 102 drugs, (DrugBank: 26 drugs), 52 drug target genes, 78 drug target pathways]

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. Progressive supranuclear palsy [ 82 clinical trials, 107 drugs, (DrugBank: 36 drugs), 60 drug target genes, 90 drug target pathways]

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. Huntington disease [ 197 clinical trials, 171 drugs, (DrugBank: 54 drugs), 82 drug target genes, 144 drug target pathways]

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. Multiple sclerosis/Neuromyelitis optica [ 3,050 clinical trials, 2,147 drugs, (DrugBank: 348 drugs), 244 drug target genes, 228 drug target pathways]

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. Multiple system atrophy [ 107 clinical trials, 153 drugs, (DrugBank: 48 drugs), 59 drug target genes, 104 drug target pathways]

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. Spinocerebellar degeneration [ 59 clinical trials, 78 drugs, (DrugBank: 28 drugs), 44 drug target genes, 59 drug target pathways]

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. Fragile X syndrome [ 98 clinical trials, 73 drugs, (DrugBank: 27 drugs), 46 drug target genes, 57 drug target pathways]

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