DDrare: Database of Drug Development for Rare Diseases

( DrugBank: - / KEGG DRUG: - )

1 disease

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	899

19. Lysosomal storage disease

Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

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Showing 1 to 10 of 899 trials

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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	NCT05710367 (ClinicalTrials.gov)	August 2023	10/1/2023	Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients	Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients; ...	Fabry Disease	Drug: Dapagliflozin 10mg Tab;Drug: Placebo	Albina Nowak, MD	NULL	Not yet recruiting	18 Years	70 Years	All	46	Phase 2	NULL
2	NCT05698901 (ClinicalTrials.gov)	March 31, 2023	16/1/2023	Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease	Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease	Fabry Disease	Drug: Agalsidase beta	Mackay Memorial Hospital	NULL	Not yet recruiting	18 Years	N/A	All	150		NULL
3	NCT05238324 (ClinicalTrials.gov)	March 2023	7/12/2021	Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II	A Phase 1 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-203 in ERT-Tre ...	Mucopolysaccharidosis II	Biological: Genetic HMI-203	Homology Medicines, Inc	NULL	Recruiting	18 Years	45 Years	Male	9	Phase 1	United States;Canada
4	NCT05710692 (ClinicalTrials.gov)	March 2023	13/12/2022	Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Diseas ...	A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Effic ...	Fabry Disease	Drug: PRX-102 1 mg/kg every 2 weeks;Drug: PRX-102 2 mg/kg every 4 weeks	Chiesi Farmaceutici S.p.A.	NULL	Not yet recruiting	18 Years	60 Years	All	18	Phase 2/Phase 3	NULL
5	NCT05594992 (ClinicalTrials.gov)	February 22, 2023	6/10/2022	An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis ...	An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis ...	Mucopolysaccharidosis II	Drug: JR-141	JCR Pharmaceuticals Co., Ltd.	NULL	Enrolling by invitation	N/A	N/A	Male	80	Phase 3	United States
6	NCT05739643 (ClinicalTrials.gov)	February 3, 2023	13/2/2023	Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated in the Past Wit ...	A Phase 1b Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previ ...	Krabbe Disease	Biological: FBX-101	Forge Biologics, Inc	NULL	Recruiting	N/A	N/A	All	12	Phase 1/Phase 2	United States
7	NCT05665166 (ClinicalTrials.gov)	February 2023	7/12/2022	Autologous Ex-vivo Gene Modified HSCT in MPSII	A Phase I/II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With CD11B Lent ...	Mucopolysaccharidosis II	Genetic: Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEI ...	University of Manchester	Manchester University NHS Foundation Trust;AVROBIO;CTI Clinical Trial and Consulting Services;Great ...	Not yet recruiting	3 Months	12 Months	Male	5	Phase 1/Phase 2	NULL
8	NCT05682144 (ClinicalTrials.gov)	January 15, 2023	12/12/2022	ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I	A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With ...	Mucopolysaccharidosis IH/S;Mucopolysaccharidosis IS	Biological: Autologous Plasmablasts (B cells)	Immusoft Corporation	NULL	Not yet recruiting	18 Years	N/A	All	2	Phase 1	United States
9	NCT05529992 (ClinicalTrials.gov)	January 3, 2023	2/9/2022	A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher ...	A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucer ...	Gaucher Disease	Drug: Velaglucerase Alfa	Takeda	NULL	Recruiting	2 Years	N/A	All	20	Phase 3	China
10	EUCTR2021-002550-82-NL (EUCTR)	22/12/2022	30/07/2022	Effectiveness of ambroxol for treating children and adults with Gaucher disease type 3	Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series - ATTACK-GD3	Gaucher disease type 3 <br>MedDRA version: 20.0;Level: HLGT;Classification code 10027424;Term: Metab ...	Product Name: Ambroxol hydrochloride<br>INN or Proposed INN: AMBROXOL Hydrochloride<br>Other descrip ...	Amsterdam UMC	NULL	Authorised-recruitment may be ongoing or finished			Female: yes<br>Male: yes	4	Phase 3	Netherlands

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