Other: Palliative Care ( DrugBank: - )

---

5 diseases

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	1
65	Primary immunodeficiency	1
120	Hereditary dystonia	1
160	Congenital ichthyosis	1
326	Osteopetrosis	1

19. Lysosomal storage disease

---

Clinical trials : 854 /  Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

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	NCT03333200 (ClinicalTrials.gov)	January 11, 2012	24/4/2017	Longitudinal Study of Neurodegenerative Disorders	Longitudinal Study of Neurodegenerative Disorders	MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease	Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation	University of Pittsburgh	NULL	Recruiting	N/A	N/A	All	1500		United States

65. Primary immunodeficiency

---

Clinical trials : 482 /  Drugs : 653 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 212

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	NCT03333200 (ClinicalTrials.gov)	January 11, 2012	24/4/2017	Longitudinal Study of Neurodegenerative Disorders	Longitudinal Study of Neurodegenerative Disorders	MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease	Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation	University of Pittsburgh	NULL	Recruiting	N/A	N/A	All	1500		United States

120. Hereditary dystonia

---

Clinical trials : 25 /  Drugs : 21 - (DrugBank : 4) / Drug target genes : 2 - Drug target pathways : 2

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	NCT03333200 (ClinicalTrials.gov)	January 11, 2012	24/4/2017	Longitudinal Study of Neurodegenerative Disorders	Longitudinal Study of Neurodegenerative Disorders	MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease	Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation	University of Pittsburgh	NULL	Recruiting	N/A	N/A	All	1500		United States

160. Congenital ichthyosis

---

Clinical trials : 36 /  Drugs : 72 - (DrugBank : 21) / Drug target genes : 18 - Drug target pathways : 111

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	NCT03333200 (ClinicalTrials.gov)	January 11, 2012	24/4/2017	Longitudinal Study of Neurodegenerative Disorders	Longitudinal Study of Neurodegenerative Disorders	MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease	Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation	University of Pittsburgh	NULL	Recruiting	N/A	N/A	All	1500		United States

326. Osteopetrosis

---

Clinical trials : 20 /  Drugs : 51 - (DrugBank : 14) / Drug target genes : 17 - Drug target pathways : 81

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	NCT03333200 (ClinicalTrials.gov)	January 11, 2012	24/4/2017	Longitudinal Study of Neurodegenerative Disorders	Longitudinal Study of Neurodegenerative Disorders	MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease	Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation	University of Pittsburgh	NULL	Recruiting	N/A	N/A	All	1500		United States

Back to top