AGALSIDASE ALFA ( DrugBank: Agalsidase alfa )


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56

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
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
Inclusion_
agemax
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gender
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size
PhaseCountries
1NCT05067868
(ClinicalTrials.gov)
November 1, 202224/9/2021A Study of Replagal in Children and Adults With Fabry Disease in IndiaA Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Agalsidase Alfa (r-DNA Origin) (Replagal™) in Indian Children and Adults With Fabry DiseaseFabry DiseaseBiological: ReplagalShireNULLRecruitingN/AN/AAll5Phase 4India
2EUCTR2018-004689-32-FI
(EUCTR)
12/10/202217/08/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease REPLAGAL is intended for use for 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLNot RecruitingFemale: yes
Male: yes
45Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Latvia;Germany;Sweden;Bosnia and Herzegovina
3JPRN-jRCT2071220022
14/07/202225/06/2022A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy Fabry's diseaseDrug: Venglustat (GZ402671)
Pharmaceutical form: Tablet, Route of administration: Oral
Drug: Agalsidase alfa
Pharmaceutical form: Concentrate for solution for infusion, Route of administration: IV infusion
Drug: Agalsidase beta (GZ419828)
Pharmaceutical form: Powder for concentrate for solution for infusion, Route of administration: IV infusion
Drug: Migalastat
Pharmaceutical form: Hard capsules, Route of administration: Oral
Tanaka TomoyukiNULLRecruiting>= 18age old<= 65age oldBoth90Phase 3China;Italy;Republic of Korea;Japan
4EUCTR2021-002320-20-NL
(EUCTR)
21/06/202216/03/2022A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
Other descriptive name: Galafold
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of
5EUCTR2021-002320-20-DK
(EUCTR)
05/05/202212/11/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Germany;Netherlands;Norway;China;Japan;Korea, Republic of
6NCT05280548
(ClinicalTrials.gov)
May 3, 20224/3/2022A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry DiseaseA Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular HypertrophyFabry's DiseaseDrug: Venglustat (GZ402671);Drug: Agalsidase alfa;Drug: Agalsidase beta (GZ419828);Drug: MigalastatGenzyme, a Sanofi CompanyNULLRecruiting18 Years65 YearsAll90Phase 3China;Czechia;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Spain;Taiwan;Turkey
7EUCTR2021-002320-20-PL
(EUCTR)
12/04/202211/01/2022A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of
8EUCTR2021-002320-20-NO
(EUCTR)
11/04/202212/10/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy Fabry’s 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Netherlands;Norway;Germany;China;Japan;Korea, Republic of
9EUCTR2018-004689-32-PL
(EUCTR)
08/10/202107/10/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Australia;Latvia;Germany;Sweden;Bosnia and Herzegovina
10EUCTR2018-004689-32-GR
(EUCTR)
06/10/202106/10/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease REPLAGAL is intended for use for 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLNot RecruitingFemale: yes
Male: yes
45Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Belgium;Brazil;Poland;Croatia;Romania;Australia;Latvia;Germany;Bosnia and Herzegovina;Sweden
11EUCTR2018-004689-32-PT
(EUCTR)
24/09/202111/08/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Belgium;Brazil;Poland;Croatia;Romania;Australia;Latvia;Germany;Bosnia and Herzegovina;Sweden
12EUCTR2018-004689-32-SE
(EUCTR)
24/08/202117/08/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina
13NCT04143958
(ClinicalTrials.gov)
September 202028/10/2019To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry DiseaseA Randomized, Open-label, Active Comparator, 2-arm, Prospective Study to Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry DiseaseFabry's DiseaseDrug: agalsidase beta (GZ419828);Drug: agalsidase alfaSanofiNULLWithdrawn16 Years45 YearsMale0Phase 4Czechia
14NCT04281537
(ClinicalTrials.gov)
March 1, 20207/2/2020A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.A Multi-country Time and Motion Study to Describe the Experience of Clinicians, Patients and Their Caregivers During the Treatment of Fabry Disease With Enzyme Replacement Therapy With Agalsidase Alfa and Agalsidase BetaFabry DiseaseDrug: Agalsidase Beta;Drug: Agalsidase AlphaAmicus TherapeuticsNULLCompleted18 YearsN/AAll76United States;Brazil;Japan;Taiwan;Turkey
15EUCTR2019-000064-21-GB
(EUCTR)
20/12/201911/02/2020To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfaA randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease. - Fabry study with Fabrazyme and Replagal Fabry's 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Fabrazyme
Product Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Sanofi Aventis Groupe (SAG)NULLNot RecruitingFemale: no
Male: yes
35Phase 4France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Norway;Germany;Italy;United Kingdom
16EUCTR2017-001528-23-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
17EUCTR2017-001528-23-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
18EUCTR2017-001528-23-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
19EUCTR2016-001318-11-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
20EUCTR2017-001528-23-ES
(EUCTR)
21/12/201716/10/2017Phase 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;Belgium;Spain;Turkey;Denmark;Netherlands;Germany;Italy;United Kingdom
21EUCTR2016-001318-11-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
22EUCTR2016-001318-11-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
23EUCTR2017-001528-23-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
24EUCTR2017-001528-23-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
25EUCTR2016-001318-11-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
26NCT03018730
(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
27EUCTR2016-001318-11-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
28EUCTR2016-001318-11-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.NULLNot RecruitingFemale: yes
Male: yes
22Phase 3Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
29EUCTR2010-022709-16-GB
(EUCTR)
23/08/201221/05/2012A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT).AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE Fabry disease
MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;United Kingdom
30EUCTR2010-022636-37-DE
(EUCTR)
09/05/201210/01/2012The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 15.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
50Phase 3Hungary;Germany;Turkey;Switzerland;France;Italy;Austria;Australia;Brazil;Israel;United Kingdom;Slovakia;Russian Federation;Taiwan;Mexico;Argentina;Belgium;Denmark;Japan;United States;Greece;Poland
31NCT01650779
(ClinicalTrials.gov)
April 201224/7/2012A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase BetaEvaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study)Fabry DiseaseBiological: Agalsidase betaGenzyme, a Sanofi CompanyNULLCompletedN/AN/AMale15Phase 4United States
32EUCTR2009-015985-75-SI
(EUCTR)
22/03/201205/03/2012Follow on study in adult Fabry Disease patientsAn Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease Fabry disease
MedDRA version: 14.1;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]
Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Shire Human Genetic Therapies (HGT), Inc.NULLNot RecruitingFemale: yes
Male: yes
43Phase 3;Phase 4United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
33EUCTR2010-022636-37-IT
(EUCTR)
16/12/201113/03/2012A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012 Fabry disease
MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrocloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrocloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Trade Name: REPLAGAL
INN or Proposed INN: Agalsidase alfa
Other descriptive name: NA
Trade Name: FABRAZYME
INN or Proposed INN: Agalsidase beta
Other descriptive name: NA
AMICUS THERAPEUTICS, INCNULLNot RecruitingFemale: yes
Male: yes
50United States;Taiwan;Greece;Turkey;Austria;Russian Federation;Italy;Switzerland;United Kingdom;Mexico;Argentina;Brazil;Belgium;Denmark;Australia;Germany;Japan
34NCT01304277
(ClinicalTrials.gov)
November 17, 201115/2/2011This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.A Phase II Comparability Study Between Replagal® Produced From Agalsidase Alfa Manufactured by 2 Different Processes in Adult Male Patients With Fabry DiseaseFabry DiseaseBiological: agalsidase alfaShireNULLCompleted18 Years65 YearsMale17Phase 2Canada
35EUCTR2009-015985-75-FI
(EUCTR)
15/11/201126/10/2011Follow on study in adault Fabry Disease patientsAn Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease Fabry disease
MedDRA version: 16.0;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]
Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Shire Human Genetic Therapies (HGT), Inc.NULLNot RecruitingFemale: yes
Male: yes
43United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
36EUCTR2010-022636-37-GR
(EUCTR)
02/11/201123/09/2011The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
50Phase 3United States;Taiwan;Slovakia;Greece;Turkey;Austria;Israel;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Argentina;Brazil;Poland;Belgium;Denmark;Australia;Germany;Japan
37EUCTR2009-015985-75-GB
(EUCTR)
18/10/201119/05/2011Follow on study in adault Fabry Disease patientsAn Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease Fabry disease
MedDRA version: 14.0;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]
Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Shire Human Genetic Therapies (HGT), Inc.NULLNot RecruitingFemale: yes
Male: yes
43Phase 3;Phase 4United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
38EUCTR2010-022709-16-BE
(EUCTR)
12/09/201110/08/2011A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT).AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE Fabry disease
MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
24Phase 2France;Canada;Belgium;Australia;Netherlands;United Kingdom;Switzerland
39NCT01298141
(ClinicalTrials.gov)
August 10, 201115/2/2011A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry DiseaseA Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal® (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry DiseaseFabry DiseaseBiological: agalsidase alfaShireNULLCompletedN/AN/AAll171Phase 3Canada
40EUCTR2010-022636-37-AT
(EUCTR)
03/08/201127/07/2011The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
68Phase 3France;United States;Greece;Brazil;Belgium;Denmark;Australia;Austria;Germany;Japan;Italy;United Kingdom
41EUCTR2010-022636-37-DK
(EUCTR)
03/05/201106/04/2011The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
68Phase 3United States;France;Greece;Belgium;Brazil;Austria;Australia;Denmark;Germany;United Kingdom;Japan;Italy
42NCT01363492
(ClinicalTrials.gov)
May 201131/3/2011Safety Study of Replagal® Therapy in Children With Fabry DiseaseAn Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement TherapyFabry DiseaseBiological: Replagal (agalsidase alfa)ShireNULLCompleted7 Years17 YearsAll15Phase 2United States
43EUCTR2010-022636-37-GB
(EUCTR)
15/04/201122/12/2010The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
68Phase 3United States;France;Greece;Belgium;Brazil;Denmark;Austria;Australia;Germany;Japan;Italy;United Kingdom
44NCT01196871
(ClinicalTrials.gov)
February 2, 20117/9/2010Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry DiseaseAn Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry DiseaseFabry DiseaseDrug: Migalastat HCl;Biological: Agalsidase Beta;Biological: Agalsidase AlfaAmicus TherapeuticsNULLCompleted18 Years65 YearsMale20Phase 2United States;Australia;Belgium;Canada;Netherlands;France;United Kingdom
45EUCTR2010-022636-37-BE
(EUCTR)
18/01/201116/12/2010The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
68Phase 3United States;France;Greece;Brazil;Belgium;Denmark;Austria;Australia;Germany;United Kingdom;Japan;Italy
46EUCTR2009-015985-75-CZ
(EUCTR)
08/03/201024/02/2010Follow on study in adult Fabry Disease patientsAn Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease Fabry disease
MedDRA version: 16.0;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]
Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Shire Human Genetic Therapies (HGT), Inc.NULLNot RecruitingFemale: yes
Male: yes
43United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
47NCT01031173
(ClinicalTrials.gov)
February 17, 20108/12/2009Treatment Protocol of Replagal for Patients With Fabry DiseaseAn Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.Fabry DiseaseBiological: agalsidase alfaShireNULLNo longer availableN/AN/AAllUnited States
48EUCTR2009-015985-75-PL
(EUCTR)
03/02/201030/11/2009Follow on study in adault Fabry Disease patientsAn Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease Fabry disease
MedDRA version: 14.0;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]
Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Shire Human Genetic Therapies (HGT), Inc.NULLNot RecruitingFemale: yes
Male: yes
43United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
49NCT00097890
(ClinicalTrials.gov)
November 200430/11/2004Replagal Enzyme Replacement Therapy for Adults With Fabry DiseaseAn Open Label Six-Month Maintenance Clinical Trial of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease Who Have Completed TKT027Fabry DiseaseDrug: Replagal (Agalsidase Alfa);Drug: ReplagalNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompletedN/AN/AMale25Phase 4United States
50EUCTR2004-000772-14-CZ
(EUCTR)
23/07/200428/06/2004A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027) - TKT027A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027) - TKT027 Fabry Disease
MedDRA version: 6.1;Level: PT;Classification code 10016016
Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: agalsidase alfa
TKT IncNULLNot RecruitingFemale: no
Male: yes
20Phase 1Czech Republic
51NCT00084084
(ClinicalTrials.gov)
June 20045/6/2004Replagal Enzyme Replacement Therapy for Children With Fabry DiseaseAn Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023 or Who Are Naive to Enzyme Replacement TherapyFabry DiseaseDrug: Agalsidase alfaShireNULLCompleted7 Years17 YearsAll17Phase 2United States;Canada
52NCT00357786
(ClinicalTrials.gov)
October 200326/7/2006An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry DiseaseAn Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal® (Registered Trademark) in Patients With Fabry DiseaseFabry DiseaseDrug: Replagal agalsidase alfa;Drug: ReplagalNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompleted39 Years45 YearsMale3Phase 1United States
53EUCTR2015-000697-35-Outside-EU/EEA
(EUCTR)
24/04/2015A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase BetaEvaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study) - The INFORM Study Fabry Disease
MedDRA version: 18.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Agalsidase beta
INN or Proposed INN: AGALSIDASE BETA
Other descriptive name: AGALSIDASE BETA
Genzyme, a Sanofi CompanyNULLNAFemale: no
Male: yes
15United States
54EUCTR2019-000064-21-CZ
(EUCTR)
29/08/2019To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfaA randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease Fabry's 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Sanofi Aventis Groupe (SAG)NULLNot RecruitingFemale: no
Male: yes
35Phase 4France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Norway;Germany;Italy;United Kingdom
55EUCTR2019-000064-21-NO
(EUCTR)
15/08/2019To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfaA randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical benefits of agalsidase beta (Fabrazyme®) in male patients with classic Fabry disease switching from agalsidase alfa (Replagal®) Fabry's 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Sanofi Aventis Groupe (SAG)NULLNot RecruitingFemale: no
Male: yes
35Phase 4France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Germany;Norway;Italy;United Kingdom
56EUCTR2021-002320-20-ES
(EUCTR)
19/11/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy Fabry’s 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
Other descriptive name: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of