19. Lysosomal storage disease Clinical trials / Disease details
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 | 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 | ||
| 2 | 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 | ||
| 3 | NCT02618512 (ClinicalTrials.gov) | October 15, 2015 | 21/10/2015 | A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously | A Phase I/II Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously | Mucopolysaccharidosis III, Type B (MPS IIIB);Sanfilippo B | Drug: SBC-103 | Alexion Pharmaceuticals | NULL | Terminated | 5 Years | N/A | All | 3 | Phase 1/Phase 2 | United Kingdom |
| 4 | EUCTR2015-001983-20-GB (EUCTR) | 06/08/2015 | 02/06/2015 | 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 Previously Studied, SBC-103 Treatment Naïve 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: 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 | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 5 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 5 | NCT02324049 (ClinicalTrials.gov) | January 22, 2015 | 15/12/2014 | Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) | 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 IIIB | Drug: SBC-103 | Alexion Pharmaceuticals | NULL | Completed | 2 Years | 12 Years | All | 11 | Phase 1/Phase 2 | United States;Spain;United Kingdom |
| 6 | EUCTR2013-003400-39-GB (EUCTR) | 15/07/2014 | 07/05/2014 | 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: 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 | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 9 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Spain;United Kingdom |