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 | EUCTR2017-003083-13-GB (EUCTR) | 02/03/2018 | 11/01/2018 | Phase 2 study to evaluate Long-Term Safety and Efficacy of AX 250 in Patients with MPS Type IIIB | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB) MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | Allievex Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 33 | Phase 2 | United States;Taiwan;Spain;Turkey;Germany;Colombia;United Kingdom | ||
| 2 | EUCTR2017-003083-13-ES (EUCTR) | 27/12/2017 | 27/11/2017 | Phase 2 study to evaluate Long-Term Safety and Efficacy of BMN 250 in Patients with MPS Type IIIB | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB) MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: BMN 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 2 | Germany;Colombia;United Kingdom;United States;Taiwan;Argentina;Spain;Turkey;Australia | ||
| 3 | EUCTR2015-000753-20-NL (EUCTR) | 16/08/2016 | 24/08/2015 | A Phase 1/2 open label study in MPS IIIB subjects to investigate the long term safety and effect of SBC-103 given by IV infusion. | A PHASE I/II OPEN LABEL STUDY IN MPS IIIB SUBJECTS TO INVESTIGATE THE SAFETY, BIODISTRIBUTION, PHARMACOKINETICS, AND PHARMACODYNAMICS/EFFICACY OF SBC-103 ADMINISTERED INTRAVENOUSLY | Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B MedDRA version: 19.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or Proposed INN: not available Other descriptive name: rhNAGLU Product Name: 89 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled) Product Code: 89Zr-SBC-103 INN or Proposed INN: not available Other descriptive name: rhNAGLU radiolabelled | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 4 | Phase 2 | Netherlands | ||
| 4 | EUCTR2013-003400-39-ES (EUCTR) | 14/04/2016 | 17/02/2016 | A Phase 1/2 open label study in MPS IIIB subjects to investigate the long-term safety and effect of SBC-103 given by IV infusion. | A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously | Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B MedDRA version: 18.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or Proposed INN: not available Other descriptive name: rhNAGLU | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 1 | United States;Spain;United Kingdom | ||
| 5 | EUCTR2015-001985-25-DE (EUCTR) | 22/03/2016 | 16/11/2015 | Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIB | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | Allievex Corporation | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 1;Phase 2 | Taiwan;Spain;Turkey;Australia;Colombia;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 | EUCTR2015-001985-25-ES (EUCTR) | 02/03/2016 | 13/01/2016 | Phase 1/2 Study to Evaluate the Safety and Efficacy of BMN 250 in Patients with MPS IIIB | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 18.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: BMN 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 1;Phase 2 | Taiwan;Brazil;Spain;Turkey;Australia;Germany;Colombia;United Kingdom | ||
| 7 | EUCTR2015-001985-25-GB (EUCTR) | 06/11/2015 | 30/09/2015 | Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIB | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | Allievex Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 33 | Phase 1;Phase 2 | Taiwan;Spain;Turkey;Australia;Germany;Colombia;United Kingdom |