19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


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

  
11 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2022-002762-33-DE
(EUCTR)
12/12/202221/09/2022Phase3B/4 extension study to Further Evaluate Safety, Tolerability and Efficacy of AX 250 in Patients with MPS Type IIIBA 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 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]
Product Name: N/A
Product Code: AX 250
INN or Proposed INN: Tralesinidase alfa
Other descriptive name: rhNAGLU-IGF2
Allievex CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 3;Phase 4Turkey;Colombia;Germany;United Kingdom;United States
2EUCTR2017-003083-13-GB
(EUCTR)
02/03/201811/01/2018Phase 2 study to evaluate Long-Term Safety and Efficacy of AX 250 in Patients with MPS Type IIIBA 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 CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
33Phase 2United States;Taiwan;Spain;Turkey;Germany;Colombia;United Kingdom
3EUCTR2017-003083-13-ES
(EUCTR)
27/12/201727/11/2017Phase 2 study to evaluate Long-Term Safety and Efficacy of BMN 250 in Patients with MPS Type IIIBA 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.NULLNot RecruitingFemale: yes
Male: yes
33Phase 2Germany;Colombia;United Kingdom;United States;Taiwan;Argentina;Spain;Turkey;Australia
4EUCTR2015-000753-20-NL
(EUCTR)
16/08/201624/08/2015A 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.NULLNot RecruitingFemale: yes
Male: yes
4Phase 2Netherlands
5EUCTR2013-003400-39-ES
(EUCTR)
14/04/201617/02/2016A 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.NULLNot RecruitingFemale: yes
Male: yes
9Phase 1United States;Spain;United Kingdom
6EUCTR2015-001985-25-DE
(EUCTR)
22/03/201616/11/2015Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIBA 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 CorporationNULLNot RecruitingFemale: yes
Male: yes
33Phase 1;Phase 2Taiwan;Spain;Turkey;Australia;Colombia;Germany;United Kingdom
7EUCTR2015-001985-25-ES
(EUCTR)
02/03/201613/01/2016Phase 1/2 Study to Evaluate the Safety and Efficacy of BMN 250 in Patients with MPS IIIBA 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.NULLNot RecruitingFemale: yes
Male: yes
33Phase 1;Phase 2Taiwan;Brazil;Spain;Turkey;Australia;Germany;Colombia;United Kingdom
8EUCTR2015-001985-25-GB
(EUCTR)
06/11/201530/09/2015Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIBA 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 CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
33Phase 1;Phase 2Taiwan;Spain;Turkey;Australia;Germany;Colombia;United Kingdom
9EUCTR2015-001983-20-GB
(EUCTR)
06/08/201502/06/2015A 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, IncNULLNot 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): noUnited Kingdom
10EUCTR2013-003400-39-GB
(EUCTR)
15/07/201407/05/2014A 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.NULLNot 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): noUnited States;Spain;United Kingdom
11EUCTR2017-003083-13-DE
(EUCTR)
24/11/2017Phase 2 study to evaluate Long-Term Safety and Efficacy of AX 250 in Patients with MPS Type IIIBA 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 CorporationNULLNAFemale: yes
Male: yes
33Phase 2United States;Taiwan;Spain;Turkey;Colombia;Germany;United Kingdom