DDrare: Database of Drug Development for Rare Diseases

Hematopoietic stem cell infusion (DrugBank: -)

2 diseases

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	1
20	Adrenoleukodystrophy	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	NCT01372228 (ClinicalTrials.gov)	April 2011	10/6/2011	Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders	Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders	Hurler Syndrome (MPS I);Hurler-Scheie Syndrome;Hunter Syndrome (MPS II);Sanfilippo Syndrome (MPS III);Krabbe Disease (Globoid Leukodystrophy);Metachromatic Leukodystrophy (MLD);Adrenoleukodystrophy (ALD and AMN);Sandhoff Disease;Tay Sachs Disease;Pelizaeus Merzbacher (PMD);Niemann-Pick Disease;Alpha-mannosidosis	Biological: hematopoietic stem cell infusion	Talaris Therapeutics Inc.	Duke University	Active, not recruiting	N/A	N/A	All	30	Phase 1;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	NCT01372228 (ClinicalTrials.gov)	April 2011	10/6/2011	Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders	Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders	Hurler Syndrome (MPS I);Hurler-Scheie Syndrome;Hunter Syndrome (MPS II);Sanfilippo Syndrome (MPS III);Krabbe Disease (Globoid Leukodystrophy);Metachromatic Leukodystrophy (MLD);Adrenoleukodystrophy (ALD and AMN);Sandhoff Disease;Tay Sachs Disease;Pelizaeus Merzbacher (PMD);Niemann-Pick Disease;Alpha-mannosidosis	Biological: hematopoietic stem cell infusion	Talaris Therapeutics Inc.	Duke University	Active, not recruiting	N/A	N/A	All	30	Phase 1;Phase 2	United States