19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

  
39 trials found
No.TrialIDDate_
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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PhaseCountries
1NCT04552691
(ClinicalTrials.gov)
June 202211/9/2020Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease PatientsExpanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry PatientsFabry DiseaseDrug: Pegunigalsidase AlfaProtalixChiesi USA, Inc.Available18 YearsN/AAllUnited States
2EUCTR2018-001148-67-FI
(EUCTR)
16/04/202128/01/2021Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseOpen Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
MedDRA version: 24.1;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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Slovenia;Finland;Spain;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Australia;Netherlands;Norway
3EUCTR2018-001148-67-IT
(EUCTR)
11/11/202021/10/2020Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseOpen Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease - na 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 LTDNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Slovenia;Finland;Spain;Turkey;United Kingdom;Italy;Switzerland;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Netherlands;Norway
4EUCTR2018-001148-67-SI
(EUCTR)
09/01/202003/04/2019Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseOpen 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Slovenia;Spain;Turkey;Italy;Switzerland;United Kingdom;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands
5EUCTR2018-001148-67-HU
(EUCTR)
07/11/201908/11/2019Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseOpen 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Slovenia;Spain;Turkey;Italy;Switzerland;United Kingdom;Hungary;Netherlands;Norway;Australia;Brazil;Belgium;Argentina;Canada;Czech Republic
6EUCTR2018-001947-30-IT
(EUCTR)
31/10/201901/09/2021Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry DiseaseOpen 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 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 LTDNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Czechia;Taiwan;Finland;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Denmark;Netherlands;Norway;Sweden
7EUCTR2018-001947-30-DK
(EUCTR)
22/10/201913/06/2019Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry DiseaseOpen 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: 24.1;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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Czechia;Belgium;Denmark;Norway;United Kingdom;Italy
8EUCTR2018-001148-67-NL
(EUCTR)
21/06/201924/07/2019Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseOpen Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102)in Patients With Fabry Disease
MedDRA version: 24.1;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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Slovenia;Finland;Spain;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Australia;Netherlands;Norway
9EUCTR2018-001947-30-GB
(EUCTR)
27/05/201924/10/2019Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry DiseaseOpen 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Taiwan;Spain;Turkey;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Denmark;Netherlands;Norway
10EUCTR2016-000378-38-FR
(EUCTR)
13/05/201915/05/2019Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase BetaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
78Phase 3United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Denmark;Australia;Norway;Netherlands;Germany
11EUCTR2018-001947-30-BE
(EUCTR)
29/04/201901/04/2019Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry DiseaseOpen 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: 24.1;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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Czechia;Belgium;Denmark;Norway;United Kingdom;Italy
12EUCTR2016-000378-38-FI
(EUCTR)
12/03/201906/02/2019Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase BetaA 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.NULLNot RecruitingFemale: yes
Male: yes
78Phase 3United States;Czechia;Slovenia;Finland;Spain;Turkey;United Kingdom;Switzerland;Italy;France;Hungary;Canada;Argentina;Brazil;Belgium;Australia;Denmark;Norway;Germany;Netherlands;Sweden
13EUCTR2018-001148-67-ES
(EUCTR)
08/02/201916/11/2018Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseOpen 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]
Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: PEGUNIGALSIDASE ALFA
Protalix Ltd.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
110 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Slovenia;Spain;Turkey;Italy;Switzerland;United Kingdom;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Norway;Netherlands;Germany
14NCT03614234
(ClinicalTrials.gov)
November 6, 201830/7/2018Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease PatientsOpen 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 DiseaseFabry DiseaseBiological: pegunigalsidase alfaProtalixNULLEnrolling by invitation18 YearsN/AAll40Phase 3United States;Belgium;Czechia;Denmark;Italy;Norway;United Kingdom
15EUCTR2018-001148-67-GB
(EUCTR)
26/09/201816/05/2018Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseOpen 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Slovenia;Spain;Turkey;Italy;United Kingdom;Switzerland;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Norway;Netherlands;Germany
16NCT03566017
(ClinicalTrials.gov)
September 20, 201824/5/2018Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry DiseaseOpen Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseFabry DiseaseBiological: pegunigalsidase alfaProtalixNULLEnrolling by invitation18 Years60 YearsAll110Phase 3United States;Australia;Canada;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom
17EUCTR2018-001148-67-CZ
(EUCTR)
02/08/201802/08/2018Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry DiseaseOpen Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102)in Patients With Fabry Disease
MedDRA version: 24.1;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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Slovenia;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Australia;Netherlands;Norway
18EUCTR2017-001528-23-IT
(EUCTR)
17/07/201810/11/2020Phase 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 LTDNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Czechia;Taiwan;Spain;Turkey;Austria;Italy;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway
19EUCTR2017-001528-23-DK
(EUCTR)
04/07/201810/04/2018Phase 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.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway
20EUCTR2017-001528-23-CZ
(EUCTR)
07/05/201806/03/2018Phase 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.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway
21EUCTR2016-001318-11-NL
(EUCTR)
27/03/201817/10/2017Safety 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.NULLNot RecruitingFemale: yes
Male: yes
22Phase 3Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Germany;Netherlands;United Kingdom
22EUCTR2016-001318-11-DE
(EUCTR)
15/12/201710/01/2017Safety 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.NULLNot RecruitingFemale: yes
Male: yes
22Phase 3Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
23EUCTR2016-000378-38-IT
(EUCTR)
20/11/201706/02/2018Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase BetaA 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 LTDNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
78Phase 3United States;Slovenia;Paraguay;Spain;Turkey;Italy;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Australia;Germany;Netherlands;Norway
24EUCTR2016-001318-11-SI
(EUCTR)
13/11/201705/10/2017Safety 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.NULLNot RecruitingFemale: yes
Male: yes
22Phase 3Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
25EUCTR2017-001528-23-GB
(EUCTR)
18/10/201728/06/2017Phase 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.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom
26NCT03180840
(ClinicalTrials.gov)
September 27, 201729/5/2017Study of the Safety, Efficacy, & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease PatientsPhase 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 DiseaseBiological: Pegunigalsidase alfaProtalixNULLCompleted18 Years60 YearsAll30Phase 3United States;Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom
27EUCTR2017-001528-23-BE
(EUCTR)
21/08/201728/07/2017Phase 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.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom
28EUCTR2016-000378-38-SI
(EUCTR)
14/08/201730/06/2017Safety 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.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
78 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Norway;Netherlands;Germany
29EUCTR2016-000378-38-NL
(EUCTR)
24/05/201717/01/2017Safety 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.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
78 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany
30EUCTR2016-001318-11-GB
(EUCTR)
24/03/201704/10/2016Safety 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.NULLNot RecruitingFemale: yes
Male: yes
22Phase 3Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
31NCT03018730
(ClinicalTrials.gov)
February 23, 20179/1/2017Safety 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 DiseaseBiological: PRX-102 (pegunigalsidase alfa)ProtalixNULLCompleted18 Years60 YearsAll22Phase 3Australia;Canada;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;Czech Republic
32EUCTR2016-001318-11-CZ
(EUCTR)
19/01/201712/12/2016Safety 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.NULLNot RecruitingFemale: yes
Male: yes
22Phase 3Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
33EUCTR2016-001318-11-ES
(EUCTR)
11/01/201724/10/2016Safety 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
22Phase 3Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
34EUCTR2016-000378-38-BE
(EUCTR)
05/12/201630/09/2016Safety 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.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
78 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany
35EUCTR2016-000378-38-HU
(EUCTR)
15/09/201611/07/2016Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase BetaA 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.NULLNot RecruitingFemale: yes
Male: yes
78Phase 3United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Germany;Netherlands;Norway
36EUCTR2016-000378-38-GB
(EUCTR)
07/09/201608/07/2016Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase BetaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
78Phase 3United States;Slovenia;Paraguay;Spain;Turkey;United Kingdom;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany
37EUCTR2016-000378-38-ES
(EUCTR)
22/08/201615/07/2016Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase BetaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
78Phase 3United States;Paraguay;Spain;Turkey;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Australia;Germany;Netherlands;Norway
38NCT02795676
(ClinicalTrials.gov)
June 20162/6/2016Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal FunctionA 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 BetaFabry DiseaseBiological: PRX-102 (pegunigalsidase alfa);Biological: agalsidase betaProtalixNULLActive, not recruiting18 Years60 YearsAll78Phase 3United States;Argentina;Australia;Belgium;Brazil;Canada;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;Czech Republic
39NCT01981720
(ClinicalTrials.gov)
December 20135/11/2013Extension Study of PRX-102 for up to 60 MonthsA Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for up to 60 Months to Adult Fabry PatientsFabry DiseaseBiological: PRX-102 (pegunigalsidase alfa)ProtalixNULLCompleted18 YearsN/AAll15Phase 1/Phase 2United States;Paraguay;Spain;United Kingdom