RhNAGLU
( DrugBank: - / KEGG DRUG: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 11 |
19. ライソゾーム病
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
Showing 1 to 10 of 11 trials
| 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 | EUCTR2022-002762-33-DE (EUCTR) | 12/12/202220221212 | 21/09/202220220921 | Phase3B/4 extension study to Further Evaluate Safety, Tolerability and Efficacy of AX 250 in Patients with MPS Type IIIB Phase3B/4 extension study to Further Evaluate Safety, Tolerability and Efficacy of AX 250 in Patient ... | A Phase 3B/4 Open-Label Multicenter Study Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients A Phase 3B/4 Open-Label Multicenter Study Extension Study to Further Evaluate Safety, Tolerability a ... | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) 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] Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 20.1;Level: P ... | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Tralesinidase alfa Other descriptive name: rhNAGLU-IGF2 Product Name: N/A Product Code: AX 250 INN or Proposed INN: Tralesinidase alfa Other descriptive name: ... | Allievex Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 3;Phase 4 | Turkey;Colombia;Germany;United Kingdom;United States | ||
| 2 | EUCTR2017-003083-13-GB (EUCTR) | 02/03/201820180302 | 11/01/201820180111 | 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) A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intra ... | 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] Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB) MedDRA version: 20.1;Level: PT ... | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAG ... | 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 | ||
| 3 | EUCTR2017-003083-13-ES (EUCTR) | 27/12/201720171227 | 27/11/201720171127 | 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) A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intra ... | 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] Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB) MedDRA version: 20.1;Level: PT ... | Product Name: N/A Product Code: BMN 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 Product Name: N/A Product Code: BMN 250 INN or Proposed INN: Not available Other descriptive name: RHNA ... | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 2 | Germany;Colombia;United Kingdom;United States;Taiwan;Argentina;Spain;Turkey;Australia | ||
| 4 | EUCTR2015-000753-20-NL (EUCTR) | 16/08/201620160816 | 24/08/201520150824 | 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 1/2 open label study in MPS IIIB subjects to investigate the long term safety and effect of ... | 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 A PHASE I/II OPEN LABEL STUDY IN MPS IIIB SUBJECTS TO INVESTIGATE THE SAFETY, BIODISTRIBUTION, PHARM ... | 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] Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B MedDRA version: 19.0;Level: LLT;Classific ... | 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 Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or P ... | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 4 | Phase 2 | Netherlands | ||
| 5 | EUCTR2013-003400-39-ES (EUCTR) | 14/04/201620160414 | 17/02/201620160217 | 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 1/2 open label study in MPS IIIB subjects to investigate the long-term safety and effect of ... | A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and ... | 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] Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B MedDRA version: 18.1;Level: LLT;Classific ... | Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or Proposed INN: not available Other descriptive name: rhNAGLU Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or P ... | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 1 | United States;Spain;United Kingdom | ||
| 6 | EUCTR2015-001985-25-DE (EUCTR) | 22/03/201620160322 | 16/11/201520151116 | 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) A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, ... | 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] Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 20.0;Level: L ... | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAG ... | Allievex Corporation | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 1;Phase 2 | Taiwan;Spain;Turkey;Australia;Colombia;Germany;United Kingdom | ||
| 7 | EUCTR2015-001985-25-ES (EUCTR) | 02/03/201620160302 | 13/01/201620160113 | 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) A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, ... | 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] Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 18.1;Level: L ... | Product Name: N/A Product Code: BMN 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 Product Name: N/A Product Code: BMN 250 INN or Proposed INN: Not available Other descriptive name: RHNA ... | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 1;Phase 2 | Taiwan;Brazil;Spain;Turkey;Australia;Germany;Colombia;United Kingdom | ||
| 8 | EUCTR2015-001985-25-GB (EUCTR) | 06/11/201520151106 | 30/09/201520150930 | 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) A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, ... | 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] Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 20.0;Level: L ... | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAG ... | 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 | ||
| 9 | EUCTR2015-001983-20-GB (EUCTR) | 06/08/201520150806 | 02/06/201520150602 | 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 1/2 open label study in MPS IIIB subjects to investigate the long term safety and effect of ... | 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 A Phase I/II Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to In ... | 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] Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B MedDRA version: 19.0;Level: LLT;Classific ... | Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or Proposed INN: not available Other descriptive name: rhNAGLU Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or P ... | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 2 | United Kingdom | ||
| 10 | EUCTR2013-003400-39-GB (EUCTR) | 15/07/201420140715 | 07/05/201420140507 | 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 1/2 open label study in MPS IIIB subjects to investigate the long-term safety and effect of ... | A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and ... | 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] Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B MedDRA version: 19.0;Level: LLT;Classific ... | Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or Proposed INN: not available Other descriptive name: rhNAGLU Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) Product Code: SBC-103 INN or P ... | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 1;Phase 2 | United States;Spain;United Kingdom |