DDrare: Database of Drug Development for Rare Diseases

Other: sham (DrugBank: -)

2 diseases

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	1
301	Macular dystrophy	1

19. Lysosomal storage disease [ 784 clinical trials, 673 drugs, (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways]

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-ganglio ... show all
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 / 784 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	NCT02534844 (ClinicalTrials.gov)	October 2015	18/8/2015	VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease	A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease	Niemann-Pick Disease, Type C	Drug: VTS-270;Other: Sham	Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company	NULL	Active, not recruiting	4 Years	21 Years	All	51	Phase 2;Phase 3	United States;Australia;France;Germany;New Zealand;Singapore;Spain;Turkey;United Kingdom

301. Macular dystrophy [ 41 clinical trials, 47 drugs, (DrugBank: 12 drugs), 12 drug target genes, 66 drug target pathways]

Searched query = "Macular dystrophy", "Best disease", "Stargardt disease", "Cone dystrophy", "Cone rod dystrophy", "X-linked juvenile retinoschisis", "Central areolar ... show all
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	NCT03364153 (ClinicalTrials.gov)	January 12, 2018	1/12/2017	Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)	A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease	Stargardt Disease 1	Drug: Zimura;Other: Sham	IVERIC bio, Inc.	NULL	Recruiting	18 Years	50 Years	All	120	Phase 2	United States;Canada;France;Germany;Hungary;Israel;Italy;Spain;United Kingdom