19. ライソゾーム病
［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］
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 | EUCTR2018-001148-67-SI (EUCTR) | 09/01/2020 | 03/04/2019 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: PEGUNIGALSIDASE ALFA | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Slovenia;Spain;Turkey;Italy;Switzerland;United Kingdom;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands | ||
| 2 | EUCTR2018-001148-67-HU (EUCTR) | 07/11/2019 | 08/11/2019 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: PEGUNIGALSIDASE ALFA | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Slovenia;Spain;Turkey;Italy;Switzerland;United Kingdom;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands | ||
| 3 | EUCTR2018-001947-30-DK (EUCTR) | 22/10/2019 | 13/06/2019 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks in Patients with Fabry Disease | MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: PEGUNIGALSIDASE ALFA | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | United States;Taiwan;Spain;Turkey;United Kingdom;Italy;Czech Republic;Canada;Belgium;Denmark;Australia;Netherlands;Norway | ||
| 4 | EUCTR2018-001148-67-NL (EUCTR) | 21/06/2019 | 24/07/2019 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102)in Patients With Fabry Disease | MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: PEGUNIGALSIDASE ALFA | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | Switzerland;United Kingdom;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Italy;United States;Slovenia;Spain;Turkey | ||
| 5 | EUCTR2018-001947-30-GB (EUCTR) | 27/05/2019 | 24/10/2019 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks in Patients with Fabry Disease - PB-102-F51 | MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: PEGUNIGALSIDASE ALFA | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | United States;Taiwan;Spain;Turkey;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Denmark;Netherlands;Norway | ||
| 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 | EUCTR2016-000378-38-FR (EUCTR) | 13/05/2019 | 15/05/2019 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Denmark;Australia;Norway;Netherlands;Germany | ||
| 7 | EUCTR2018-001947-30-BE (EUCTR) | 29/04/2019 | 01/04/2019 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks in Patients with Fabry Disease | MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: PEGUNIGALSIDASE ALFA | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | United States;Taiwan;Spain;Turkey;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Denmark;Netherlands;Norway | ||
| 8 | EUCTR2016-000378-38-FI (EUCTR) | 12/03/2019 | 06/02/2019 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Finland;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Denmark;Australia;Norway;Netherlands;Germany;Sweden | ||
| 9 | EUCTR2018-001148-67-ES (EUCTR) | 08/02/2019 | 16/11/2018 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Slovenia;Spain;Turkey;Italy;Switzerland;United Kingdom;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Norway;Netherlands;Germany | |||
| 10 | NCT03614234 (ClinicalTrials.gov) | November 6, 2018 | 30/7/2018 | Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients | Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease | Fabry Disease | Biological: pegunigalsidase alfa | Protalix | NULL | Enrolling by invitation | 18 Years | N/A | All | 40 | Phase 3 | United States;Belgium;Czechia;Denmark;Italy;Norway;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 |
| 11 | EUCTR2018-001148-67-GB (EUCTR) | 26/09/2018 | 16/05/2018 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102)in Patients With Fabry Disease | MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: PEGUNIGALSIDASE ALFA | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Slovenia;Spain;Turkey;Italy;United Kingdom;Switzerland;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Norway;Netherlands;Germany | ||
| 12 | NCT03566017 (ClinicalTrials.gov) | September 20, 2018 | 24/5/2018 | Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | Fabry Disease | Biological: pegunigalsidase alfa | Protalix | NULL | Enrolling by invitation | 18 Years | 60 Years | All | 110 | Phase 3 | United States;Australia;Canada;Czechia;Hungary;Netherlands;Norway;Slovenia;Spain;United Kingdom |
| 13 | EUCTR2018-001148-67-CZ (EUCTR) | 02/08/2018 | 02/08/2018 | Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease | Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102)in Patients With Fabry Disease | MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: PEGUNIGALSIDASE ALFA | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Slovenia;Spain;Turkey;Italy;Switzerland;United Kingdom;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Norway;Netherlands;Germany | ||
| 14 | EUCTR2017-001528-23-IT (EUCTR) | 17/07/2018 | 10/11/2020 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme¿ (agalsidase beta) or Replagal¿ (agalsidase alfa) - Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | PROTALIX LTD | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | United States;Czechia;Taiwan;Spain;Turkey;Austria;Italy;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway | ||
| 15 | EUCTR2017-001528-23-DK (EUCTR) | 04/07/2018 | 10/04/2018 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway | ||
| 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 |
| 16 | EUCTR2017-001528-23-CZ (EUCTR) | 07/05/2018 | 06/03/2018 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway | ||
| 17 | EUCTR2016-001318-11-NL (EUCTR) | 27/03/2018 | 17/10/2017 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Germany;Netherlands;United Kingdom | ||
| 18 | EUCTR2016-000378-38-IT (EUCTR) | 20/11/2017 | 06/02/2018 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta - Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | PROTALIX LTD | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Spain;Turkey;Italy;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Australia;Germany;Netherlands;Norway | ||
| 19 | EUCTR2016-001318-11-SI (EUCTR) | 13/11/2017 | 05/10/2017 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 20 | EUCTR2017-001528-23-GB (EUCTR) | 18/10/2017 | 28/06/2017 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;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 |
| 21 | NCT03180840 (ClinicalTrials.gov) | September 27, 2017 | 29/5/2017 | Study of the Safety, Efficacy, & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients | Phase 3, Open-Label, Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa 2 mg/kg Administered Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy: Fabrazyme® (Agalsidase Beta) or Replagal™ (Agalsidase Alfa) | Fabry Disease | Biological: Pegunigalsidase alfa | Protalix | NULL | Active, not recruiting | 18 Years | 60 Years | All | 30 | Phase 3 | United States;Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom |
| 22 | EUCTR2017-001528-23-BE (EUCTR) | 21/08/2017 | 28/07/2017 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom | ||
| 23 | EUCTR2016-001318-11-GB (EUCTR) | 24/03/2017 | 04/10/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 24 | NCT03018730 (ClinicalTrials.gov) | February 23, 2017 | 9/1/2017 | Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) | Fabry Disease | Biological: PRX-102 (pegunigalsidase alfa) | Protalix | NULL | Active, not recruiting | 18 Years | 60 Years | All | 22 | Phase 3 | Australia;Canada;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;Czech Republic |
| 25 | EUCTR2016-001318-11-CZ (EUCTR) | 19/01/2017 | 12/12/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;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 |
| 26 | EUCTR2016-001318-11-ES (EUCTR) | 11/01/2017 | 24/10/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 22 | Phase 3 | Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 27 | EUCTR2016-000378-38-HU (EUCTR) | 15/09/2016 | 11/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Spain;Turkey;United Kingdom;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany | ||
| 28 | EUCTR2016-000378-38-GB (EUCTR) | 07/09/2016 | 08/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Spain;Turkey;United Kingdom;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany | ||
| 29 | EUCTR2016-000378-38-ES (EUCTR) | 22/08/2016 | 15/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Paraguay;Spain;Turkey;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Australia;Germany;Netherlands;Norway | ||
| 30 | NCT02795676 (ClinicalTrials.gov) | June 2016 | 2/6/2016 | Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function | A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta | Fabry Disease | Biological: PRX-102 (pegunigalsidase alfa);Biological: agalsidase beta | Protalix | NULL | Active, not recruiting | 18 Years | 60 Years | All | 78 | Phase 3 | United States;Argentina;Australia;Belgium;Brazil;Canada;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;Czech Republic |
| 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 |
| 31 | NCT04552691 (ClinicalTrials.gov) | April 2015 | 11/9/2020 | Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients | Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients | Fabry Disease | Drug: Pegunigalsidase Alfa | Protalix | Chiesi Farmaceutici S.p.A.;Covance | Available | 18 Years | N/A | All | NULL | ||
| 32 | NCT01981720 (ClinicalTrials.gov) | December 2013 | 5/11/2013 | Extension Study of PRX-102 for up to 60 Months | A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for up to 60 Months to Adult Fabry Patients | Fabry Disease | Biological: PRX-102 (pegunigalsidase alfa) | Protalix | NULL | Active, not recruiting | 18 Years | N/A | All | 15 | Phase 1;Phase 2 | United States;Paraguay;Spain;United Kingdom |