DDrare: Database of Drug Development for Rare Diseases

Procedure: allogeneic stem cell transplantation (DrugBank: -)

3 diseases

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	1
20	Adrenoleukodystrophy	1
62	Paroxysmal nocturnal hemoglobinuria	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	NCT01043640 (ClinicalTrials.gov)	December 2009	5/1/2010	Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders	Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders	Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal Disorders	Drug: Campath-1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate Mofetil	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	21 Years	All	46	Phase 2	United States

20. Adrenoleukodystrophy [ 49 clinical trials, 86 drugs, (DrugBank: 29 drugs), 18 drug target genes, 112 drug target pathways]

Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
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 / 49 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	NCT01043640 (ClinicalTrials.gov)	December 2009	5/1/2010	Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders	Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders	Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal Disorders	Drug: Campath-1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate Mofetil	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	21 Years	All	46	Phase 2	United States

62. Paroxysmal nocturnal hemoglobinuria [ 202 clinical trials, 120 drugs, (DrugBank: 25 drugs), 15 drug target genes, 90 drug target pathways]

Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
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 / 202 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	NCT00143559 (ClinicalTrials.gov)	August 2005	1/9/2005	Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies	Haploidentical Hematopoietic Stem Cell Transplantation Utilizing Partial T-Cell Depletion as Immunotherapy for Hematologic Malignancies	Leukemia;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic Syndrome;Paroxysmal Nocturnal Hemoglobinuria;Hodgkin's Lymphoma;Non-Hodgkin Lymphoma	Drug: Systematic chemotherapy and antibodies;Procedure: Allogeneic stem cell transplantation;Device: Miltenyi CliniMACS	St. Jude Children's Research Hospital	NULL	Completed	2 Years	21 Years	Both	17	Phase 2	United States