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

臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191

59 trials found

diseases per page:

Showing 1 to 10 of 59 diseases

Filter by

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04020055 (ClinicalTrials.gov)	October 1, 2022	24/6/2019	A Study to Evaluate Migalastatin Fabry Subjects With Amenable GLA Variants and Severe Renal Impairme ...	An Open-label Study to Evaluate the Safety and Pharmacokinetics of MigalastatHCl in Fabry Subjects W ...	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Not yet recruiting	16 Years	N/A	All	12	Phase 3	United States;Belgium;France;Italy;Spain;United Kingdom
2	EUCTR2021-002320-20-IT (EUCTR)	14/01/2022	22/10/2021	A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and fema ...	A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglusta ...	Fabry's disease MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;Sy ...	Trade Name: Fabrazyme Product Name: . Product Code: [.] INN or Proposed INN: AGALSIDASI BETA Trade Name: ...	SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110	Phase 3	United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;D ...
3	EUCTR2019-000222-21-GB (EUCTR)	25/08/2020	12/06/2020	Migalastat Pediatric Long Term Extension Study	A LONG-TERM, OPEN-LABEL STUDY TO EVALUATE THE SAFETY, PHARMACODYNAMICS, AND EFFICACY OF MIGALASTATIN ...	Fabry disease and with amenable GLA variants MedDRA version: 20.0;Level: PT;Classification code 1001 ...	Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: M ...	Amicus Therapeutics, UK Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 3	United States;United Kingdom
4	NCT04252066 (ClinicalTrials.gov)	April 17, 2020	18/12/2019	A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregna ...	A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregna ...	Fabry Disease	Drug: migalastat	Amicus Therapeutics	NULL	Recruiting	N/A	N/A	Female	20		United States
5	EUCTR2017-000146-21-IT (EUCTR)	30/01/2020	23/10/2020	AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREA ...	AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREA ...	Fabry disease and with amenable GLA variants MedDRA version: 20.0;Level: PT;Classification code 1001 ...	Trade Name: Galafold Product Name: Migalastat Product Code: [AT1001] INN or Proposed INN: migalastat Oth ...	Amicus Therapeutics UK, Ltd	NULL	NA			Female: yes Male: yes	20	Phase 3	United States;Spain;Australia;Germany;United Kingdom;Italy
6	NCT04049760 (ClinicalTrials.gov)	October 14, 2019	30/7/2019	Safety, Pharmacodynamics, and Efficacy of Migalastatin Pediatric Subjects (Aged >12 Years) With Fabr ...	A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastatin ...	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Active, not recruiting	12 Years	17 Years	All	15	Phase 3	United States;United Kingdom;Spain
7	EUCTR2017-000146-21-GB (EUCTR)	29/08/2019	22/03/2019	ASPIRE Paediatric Fabry Study	AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREA ...	Fabry disease and with amenable GLA variants MedDRA version: 20.0;Level: PT;Classification code 1001 ...	Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other ...	Amicus Therapeutics, UK Ltd	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 3	United States;Spain;Australia;Germany;Italy;United Kingdom
8	NCT03949920 (ClinicalTrials.gov)	May 16, 2019	18/4/2019	A Study of Migalastat in Fabry Disease	A Prospective Observational Study Investigating the Impact of Migalastaton Cardiovascular Structure ...	Fabry Disease	Drug: Migalastat	Manchester University NHS Foundation Trust	Salford Royal NHS Foundation Trust	Recruiting	16 Years	N/A	All	21		United Kingdom
9	EUCTR2017-000146-21-ES (EUCTR)	09/05/2019	11/04/2019	ASPIRE Pediatric Fabry Study	AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREA ...	Fabry disease and with amenable GLA variants MedDRA version: 20.0;Level: PT;Classification code 1001 ...	Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other ...	Amicus Therapeutics, UK Ltd	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United States;Spain;Australia;Germany;Italy;United Kingdom
10	NCT03500094 (ClinicalTrials.gov)	October 11, 2018	9/4/2018	Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastatin Pediatric Subjects (Aged 12 ...	An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Trea ...	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Completed	12 Years	17 Years	All	22	Phase 3	United States;Spain;United Kingdom

First Previous1 2 3 4 5 6Next Last