DDrare: Database of Drug Development for Rare Diseases

Other: saline (DrugBank: -)

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
6	パーキンソン病	1
46	悪性関節リウマチ	1
97	潰瘍性大腸炎	1
98	好酸球性消化管疾患	1
256	筋型糖原病	1
274	骨形成不全症	1
301	黄斑ジストロフィー	1

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	NCT02286011 (ClinicalTrials.gov)	November 2014	24/10/2014	Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis	Phase I Clinical Trial on Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.	ALS (Amyotrophic Lateral Sclerosis)	Biological: MNC (Mononuclear cells);Other: Saline	Red de Terapia Celular	Spanish National Health System;Hospital Universitario Virgen de la Arrixaca;Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia;Public Health Service, Murcia;Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca	Active, not recruiting	18 Years	70 Years	All	20	Phase 1	Spain

6. パーキンソン病　［臨床試験数：2,123，薬物数：2,046（DrugBank：324），標的遺伝子数：183，標的パスウェイ数：198］

Searched query = "Parkinson disease"
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 / 2,123 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	NCT04202757 (ClinicalTrials.gov)	September 14, 2018	21/9/2018	Intravenous Plasma Treatment for Parkinson's Disease	Intravenous Young Fresh Frozen Plasma (yFFP) Investigational Treatment for Parkinson's Disease - Randomized Controlled Study	Idiopathic Parkinson Disease	Biological: [21CFR640.30] Plasma from 18 - 25 year old volunteer donors;Other: Saline	The Neurology Center	Carolina Longevity Institute	Completed	45 Years	90 Years	All	18	Phase 4	United States

46. 悪性関節リウマチ　［臨床試験数：4,183，薬物数：2,538（DrugBank：401），標的遺伝子数：183，標的パスウェイ数：219］

Searched query = "Malignant rheumatoid arthritis", "Rheumatoid arthritis", "Rheumatoid arthritis with vasculitis"
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 / 4,183 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	NCT03394131 (ClinicalTrials.gov)	July 1, 2017	25/12/2017	?Efficacy of Insulin Versus Hyalase Hydro-dissection of Median Nerve in Rheumatoid Arthritis Patients	Treatment of Carpal Tunnel in Rheumatoid Arthritis	Treatment Resistant Carpal Tunnel in RA Patients	Drug: Hylase;Drug: Insulin;Other: saline	Assiut University	NULL	Unknown status	20 Years	80 Years	All	90	N/A	Egypt

97. 潰瘍性大腸炎　［臨床試験数：2,269，薬物数：1,331（DrugBank：241），標的遺伝子数：114，標的パスウェイ数：181］

Searched query = "Ulcerative colitis"
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 / 2,269 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	NCT02084550 (ClinicalTrials.gov)	September 2015	5/3/2014	Amino Acids in Ileal Pouch-anal Anastomosis for Ulcerative Colitis	Amino Acids in Ileal Pouch-anal Anastomosis for Ulcerative Colitis: a Randomized, Assessor-blinded, Placebo-controlled Trial	Ulcerative Colitis	Dietary Supplement: Vaminolac;Other: Saline	Aarhus University Hospital	NULL	Active, not recruiting	18 Years	50 Years	All	8	N/A	Denmark

98. 好酸球性消化管疾患　［臨床試験数：128，薬物数：147（DrugBank：40），標的遺伝子数：34，標的パスウェイ数：132］

Searched query = "Eosinophilic gastrointestinal disease", "Eosinophilic gastroenteritis", "Eosinophilic esophagitis", "Eosinophilic colitis", "Eosinophilic gastro-intestinal disorder", "EGID", "Neonatal food-protein induced enterocolitis", "N-FPIES"
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 / 128 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	NCT00538434 (ClinicalTrials.gov)	March 2008	1/10/2007	Efficacy and Safety Study of Reslizumab to Treat Eosinophilic Esophagitis in Subjects Aged 5 to 18 Years	An Efficacy and Safety Study of Reslizumab (CTx55700) in the Treatment of Eosinophilic Esophagitis in Subjects Aged 5 to 18 Years	Eosinophilic Esophagitis	Biological: Reslizumab;Other: Saline	Ception Therapeutics	NULL	Completed	5 Years	18 Years	All	227	Phase 2;Phase 3	United States;Canada

256. 筋型糖原病　［臨床試験数：161，薬物数：114（DrugBank：23），標的遺伝子数：26，標的パスウェイ数：106］

Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
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 / 161 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	NCT02240407 (ClinicalTrials.gov)	October 17, 2017	11/9/2014	Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease	Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD)	Pompe Disease	Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase;Drug: Rapamycin;Other: saline;Drug: Rituxan;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: Lidocaine;Drug: LMX 4 Topical Cream	University of Florida	Lacerta Therapeutics, Inc	Recruiting	18 Years	50 Years	All	7	Phase 1	United States

274. 骨形成不全症　［臨床試験数：78，薬物数：87（DrugBank：19），標的遺伝子数：14，標的パスウェイ数：74］

Searched query = "Osteogenesis Imperfecta"
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 / 78 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	NCT00655681 (ClinicalTrials.gov)	September 2007	4/4/2008	Prevention of Post Operative Bone Loss in Children	Prevention of Post Operative Bone Loss in Children	Osteoporosis;Cerebral Palsy;Spina Bifida;Osteopenia;Osteogenesis Imperfecta	Drug: pamidronate;Other: saline	University of New Mexico	Thrasher Research Fund	Completed	4 Years	18 Years	Both	24	N/A	United States

301. 黄斑ジストロフィー　［臨床試験数：41，薬物数：47（DrugBank：12），標的遺伝子数：12，標的パスウェイ数：66］

Searched query = "Macular dystrophy", "Best disease", "Stargardt disease", "Cone dystrophy", "Cone rod dystrophy", "X-linked juvenile retinoschisis", "Central areolar choroidal dystrophy"
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 / 41 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	NCT00346853 (ClinicalTrials.gov)	November 2005	28/6/2006	Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy	Clinical Interventions Against Stargardt Macular Dystrophy: Phase 1 Pilot Study of 4-MP as an Inhibitor of Dark Adaptation	Macular Dystrophy, Corneal	Drug: 4-Methylpyrazole;Other: saline	University of Utah	NULL	Completed	18 Years	65 Years	All	10	Phase 1	United States

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