Replagal    (DrugBank: -)

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50

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.
50 / 784 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04143958
(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
2EUCTR2019-000064-21-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
3EUCTR2017-001528-23-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
4EUCTR2017-001528-23-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
5EUCTR2017-001528-23-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2016-001318-11-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
7EUCTR2016-001318-11-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]
Protalix Ltd.NULLNot Recruiting Female: yes
Male: yes
22Phase 3Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
8EUCTR2016-001318-11-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
9EUCTR2017-001528-23-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
10NCT03180840
(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 alfaProtalixNULLActive, not recruiting18 Years60 YearsAll30Phase 3United States;Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2017-001528-23-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
12NCT02956954
(ClinicalTrials.gov)
March 25, 20173/11/2016Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry DiseaseFollow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®)Anderson-Fabry DiseaseDrug: Enzyme replacement therapy (Agalsidase alpha (Replagal®));Procedure: Magnetic Resonance ImagingUniversity Hospital, RouenNULLUnknown status18 YearsN/AAll25N/AFrance
13EUCTR2016-001318-11-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
14NCT03018730
(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)ProtalixNULLActive, not recruiting18 Years60 YearsAll22Phase 3Australia;Canada;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;Czech Republic
15EUCTR2016-001318-11-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2016-001318-11-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
17EUCTR2010-022709-16-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 2ABelgium;United Kingdom
18EUCTR2010-022636-37-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
19EUCTR2009-015985-75-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
20EUCTR2010-022636-37-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2009-015985-75-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
22EUCTR2010-022636-37-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
23EUCTR2009-015985-75-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
24EUCTR2010-022709-16-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
25NCT01298141
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2010-022636-37-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
27EUCTR2010-022636-37-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
28NCT01363492
(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
29EUCTR2010-022636-37-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
30EUCTR2007-005543-22-SI
(EUCTR)
18/03/201124/02/2011An investigation of 3 dosages of Replagal in adult patients with Fabry diseaseA Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in 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 alpha
Other descriptive name: Replagal
Shire Human Genetic Therapies, Inc. (Shire HGT)NULLNot RecruitingFemale: yes
Male: yes
43United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT01304277
(ClinicalTrials.gov)
March 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
32EUCTR2010-022636-37-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
33EUCTR2007-005543-22-FI
(EUCTR)
18/05/201023/03/2010An investigation of 3 dosages of Replagal in adult patients with Fabry diseaseA Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in 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 alpha
Other descriptive name: Replagal
Shire Human Genetic Therapies, Inc. (Shire HGT)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
43Finland;United Kingdom;Czech Republic;Paraguay;Slovenia;United States;Australia
34EUCTR2007-005543-22-GB
(EUCTR)
06/05/201017/08/2009An investigation of 3 dosages of Replagal in adult patients with Fabry disease A Multi-center, Open-Label, Randomized Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in 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]
Shire Human Genetic Therapies, Inc. (Shire HGT)NULLNot Recruiting Female: yes
Male: yes
43Phase 3;Phase 4United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom
35NCT01124643
(ClinicalTrials.gov)
April 13, 201023/4/2010Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry DiseaseAn Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry DiseaseFabry DiseaseBiological: ReplagalShireNULLCompleted18 YearsN/AAll35Phase 3United States;Australia;Czechia;Finland;Poland;Slovenia;United Kingdom;Czech Republic;Paraguay
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2009-015985-75-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
37EUCTR2009-015985-75-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
38NCT01031173
(ClinicalTrials.gov)
September 20098/12/2009Treatment 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/ABothN/AUnited States
39NCT00864851
(ClinicalTrials.gov)
December 29, 200818/3/2009Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry DiseaseA Multi-Center, Open-Label, Randomized Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in Adult Patients With Fabry DiseaseFabry DiseaseBiological: ReplagalShireNULLCompleted18 YearsN/AAll44Phase 3United States;Australia;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;Czech Republic
40EUCTR2007-005543-22-CZ
(EUCTR)
19/06/200802/01/2008An investigation of 3 dosages of Replagal in adult patients with Fabry diseaseA Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in 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 alpha
Other descriptive name: Replagal
Shire Human Genetic Therapies, Inc. (Shire HGT)NULLNot RecruitingFemale: yes
Male: yes
43United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41NCT00097890
(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
42EUCTR2004-000772-14-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 1;Phase 2Czech Republic
43NCT00084084
(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
44NCT00075244
(ClinicalTrials.gov)
January 20046/1/2004Alternative Dosing and Regimen of Replagal to Treat Fabry DiseaseA Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients With Fabry DiseaseFabry DiseaseDrug: ReplagalNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompletedN/AN/AMale25Phase 2United States
45NCT00071877
(ClinicalTrials.gov)
October 20033/11/2003An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry DiseaseA Clinical Trial of Replagal Enzyme Replacement Therapy in Children Ages 7 - 17 Years With Fabry DiseaseFabry DiseaseDrug: ReplagalNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompletedN/AN/ABoth25Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46NCT00357786
(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
47NCT00068107
(ClinicalTrials.gov)
September 20036/9/2003Dosing Study of Replagal in Patients With Fabry DiseaseStudy of Weekly Dosing Regimens of Replagal in Patients With Fabry Disease With Incomplete Clinical Response to Long-Term TherapyFabry DiseaseDrug: ReplagalBaylor Research InstituteNational Institute of Neurological Disorders and Stroke (NINDS)CompletedN/AN/AMale13Phase 2United States
48NCT00048906
(ClinicalTrials.gov)
November 20028/11/2002Alpha-Galactosidase A Replacement Therapy for Fabry DiseaseA Safety and Pharmacokinetic Study of Replagal Enzyme Replacement Therapy in Patients With Fabry DiseaseFabry DiseaseDrug: DRX005BNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompletedN/AN/ABoth3Phase 2United States
49EUCTR2019-000064-21-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
50EUCTR2019-000064-21-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