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-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
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.
| 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 | NCT03759639 (ClinicalTrials.gov) | September 4, 2019 | 27/11/2018 | N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) | Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study. | Niemann-Pick Disease, Type C | Drug: IB1001 | IntraBio Inc | NULL | Active, not recruiting | 6 Years | N/A | All | 34 | Phase 2 | United States;Germany;Slovakia;Spain;United Kingdom |
| 2 | EUCTR2018-004406-25-GB (EUCTR) | 20/06/2019 | 06/02/2020 | To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning and quality of life in patients with GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). | Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and SandhoffDisease): A multinational, multicenter, open-label, rater-blinded Phase II study - IB1001-202 | To demonstrate that N-Acetyl-L-Leucine is effective in improvingsymptoms, functioning, and quality of life in patients GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). MedDRA version: 20.0;Level: PT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.1;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: SUB195712 Other descriptive name: N-Acetyl-L-Leucine Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other descriptive name: N-Acetyl-L-Leucine | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 2 | United States;Spain;Germany;United Kingdom | ||
| 3 | EUCTR2018-004331-71-GB (EUCTR) | 20/06/2019 | 14/01/2019 | To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning and quality of life in patients with Niemann-Pick Type C disease (NPC). | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C: A multinational,multicenter, open-label, rater-blinded Phase II study. - IB1001-201Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease | To demonstrate that N-Acetyl-L-Leucine is effective in improvingsymptoms, functioning, and quality of life in patients withNiemann-Pick Type C disease (NPC). MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: SUB195712 Other descriptive name: N-Acetyl-L-Leucine Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other descriptive name: N-Acetyl-L-Leucine | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 2 | United States;Slovakia;Spain;Germany;United Kingdom | ||
| 4 | NCT03759665 (ClinicalTrials.gov) | June 7, 2019 | 27/11/2018 | N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease) | Effects of N-Acetyl-L-Leucine on GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study | GM2 Gangliosidosis;Tay-Sachs Disease;Sandhoff Disease | Drug: IB1001 | IntraBio Inc | NULL | Recruiting | 6 Years | N/A | All | 39 | Phase 2 | United States;Germany;Spain;United Kingdom |
| 5 | EUCTR2018-004406-25-DE (EUCTR) | 09/05/2019 | 23/01/2019 | A safety and efficacy study of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). | Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multicenter, open-label, rater-blinded Phase II study. | GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 2 | United States;Spain;Germany;United Kingdom | ||
| 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 |
| 6 | EUCTR2018-004331-71-DE (EUCTR) | 08/05/2019 | 02/01/2019 | A safety and efficacy study of N-Acetyl-L-Leucine on Niemann-Pick type C Disease. | Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II study | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 2 | United States;Slovakia;Spain;Germany;United Kingdom |