GLA ( DrugBank: - )
2 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 13 | 多発性硬化症／視神経脊髄炎 | 243 |
| 19 | ライソゾーム病 | 39 |
13. 多発性硬化症／視神経脊髄炎
臨床試験数 : 3,340 / 薬物数 : 2,163 - (DrugBank : 383) / 標的遺伝子数 : 241 - 標的パスウェイ数 : 238
Showing 1 to 10 of 243 diseases
| 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 | NCT05131971 (ClinicalTrials.gov) | November 1, 2021 | 27/10/2021 | A Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics (PD) of GSK3888130B in Healthy Participants A Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics (PD) of GSK3888130B ... | A Randomized, Double-Blind, Placebo Controlled, Single Dose Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of GSK3888130B in Healthy Participants Aged 18-55 Inclusive A Randomized, Double-Blind, Placebo Controlled, Single Dose Escalation Study to Evaluate Safety, Tol ... | Multiple Sclerosis | Drug: Placebo;Drug: GSK3888130B | GlaxoSmithKline | NULL | Recruiting | 18 Years | 55 Years | All | 70 | Phase 1 | United Kingdom |
| 2 | NCT04767698 (ClinicalTrials.gov) | October 1, 2021 | 18/2/2021 | Addition of Belimumab to B-cell Depletion in Relapsing-remitting Multiple Sclerosis | Addition of Belimumab to B-cell Depletion to Produce Prolonged Remission of Relapsing-remitting Multiple Sclerosis Disease Activity Addition of Belimumab to B-cell Depletion to Produce Prolonged Remission of Relapsing-remitting Mult ... | Multiple Sclerosis | Drug: Belimumab;Drug: Short-course Ocrelizumab;Drug: Continued Ocrelizumab | Johns Hopkins University | GlaxoSmithKline | Terminated | 18 Years | N/A | All | 3 | Phase 2 | United States |
| 3 | NCT05688436 (ClinicalTrials.gov) | September 24, 2021 | 9/1/2023 | A Study to Assess Pregnancy Outcomes in Women Exposed to Diroximel Fumarate | Pregnancy Outcomes in Women Exposed to Diroximel Fumarate | Multiple Sclerosis | Drug: Diroximel Fumarate;Biological: Alemtuzumab;Drug: Fingolimod;Drug: Glatiramer acetate;Biological: Interferon beta;Biological: Natalizumab;Biological: Ocrelizumab;Biological: Peginterferon beta-1a;Drug: Siponimod Drug: Diroximel Fumarate;Biological: Alemtuzumab;Drug: Fingolimod;Drug: Glatiramer acetate;Biologica ... | Biogen | NULL | Recruiting | 18 Years | 49 Years | Female | 825 | United States | |
| 4 | EUCTR2020-004505-32-IT (EUCTR) | 24/09/2021 | 12/10/2021 | Study of efficacy and tolerability of ofatumumab vs. First Line disease modifying treatment (DMT) - physician’s choice in the treatment of newly diagnosed relapsing multiple sclerosis (RMS) patients Study of efficacy and tolerability of ofatumumab vs. First Line disease modifying treatment (DMT) - ... | An Open-Label, Rater-Blind, Randomized, Multi-Center, Parallel-Arm, Active-Comparator Study to Assess the Efficacy and Tolerability of Ofatumumab 20mgSC monthly vs. First Line DMT - physician’s choice in the treatment of newly diagnosed RMS (STHENOS) - STHENOS An Open-Label, Rater-Blind, Randomized, Multi-Center, Parallel-Arm, Active-Comparator Study to Asses ... | Multiple Sclerosis MedDRA version: 20.0;Level: PT;Classification code 10048393;Term: Multiple sclerosis relapse;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Multiple Sclerosis MedDRA version: 20.0;Level: PT;Classification code 10048393;Term: Multiple sclero ... | Trade Name: COPAXONE - 20 MG/ML SOLUZIONE INIETTABILE 28 SIRINGHE PRERIEMPITE Product Name: COPAXONE Product Code: [-] INN or Proposed INN: glatiramer acetato Trade Name: Rebif Product Name: Rebif Product Code: [-] INN or Proposed INN: interferone beta-1a Trade Name: Rebif Product Name: rebif Product Code: [-] INN or Proposed INN: interferone beta-1a Trade Name: plegridy Product Name: plegridy Product Code: [-] INN or Proposed INN: peginterferone beta 1a Trade Name: Avonex Product Name: Avonex Product Code: [-] INN or Proposed INN: Interferone beta 1a Trade Name: Extavia Product Name: Extavia Product Code: [-] INN or Proposed INN: interferone beta-1b Trade Name: COPAXONE - 40 MG/ML SOLUZIONE INIETTABILE IN Trade Name: COPAXONE - 20 MG/ML SOLUZIONE INIETTABILE 28 SIRINGHE PRERIEMPITE Product Name: COPAXONE P ... | NOVARTIS PHARMA AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 236 | Phase 3 | France;Spain;Germany;Italy | ||
| 5 | EUCTR2020-004505-32-DE (EUCTR) | 06/07/2021 | 25/05/2021 | Study of efficacy and tolerability of ofatumumab vs. First Line disease modifying treatment (DMT) - physician’s choice in the treatment of newly diagnosed relapsing multiple sclerosis (RMS) patients Study of efficacy and tolerability of ofatumumab vs. First Line disease modifying treatment (DMT) - ... | An Open-Label, Rater-Blind, Randomized, Multi-Center, Parallel-Arm, Active-Comparator Study to Assess the Efficacy and Tolerability of Ofatumumab 20mg SC monthly vs. First Line DMT-physician’s choice in the treatment of newly diagnosed RMS - STHENOS An Open-Label, Rater-Blind, Randomized, Multi-Center, Parallel-Arm, Active-Comparator Study to Asses ... | Multiple Sclerosis MedDRA version: 20.0;Level: PT;Classification code 10048393;Term: Multiple sclerosis relapse;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Multiple Sclerosis MedDRA version: 20.0;Level: PT;Classification code 10048393;Term: Multiple sclero ... | Trade Name: Kesimpta Product Name: ofatumumab Product Code: OMB157 INN or Proposed INN: OFATUMUMAB Product Name: Glatiramer acetate INN or Proposed INN: GLATIRAMER ACETATE Other descriptive name: GLATIRAMER ACETATE Product Name: Glatiramer acetate INN or Proposed INN: GLATIRAMER ACETATE Other descriptive name: GLATIRAMER ACETATE Product Name: Interferon ß-1b INN or Proposed INN: INTERFERON BETA-1B Other descriptive name: INTERFERON BETA-1B Product Name: Interferon ß-1a INN or Proposed INN: Interferon beta-1a Product Name: Interferon ß-1a INN or Proposed INN: Interferon beta-1a Product Name: Interferon ß-1a Trade Name: Kesimpta Product Name: ofatumumab Product Code: OMB157 INN or Proposed INN: OFATUMUMAB Produ ... | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 236 | Phase 3 | France;Spain;Germany;United Kingdom;Italy | ||
| 6 | NCT04079088 (ClinicalTrials.gov) | June 30, 2021 | 3/9/2019 | Study to Evaluate Oral BIIB061 Added to Interferon-beta1 (IFN-ß1) or Glatiramer Acetate in Relapsing Multiple Sclerosis (RMS) Study to Evaluate Oral BIIB061 Added to Interferon-beta1 (IFN-ß1) or Glatiramer Acetate in Relapsing ... | A Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Phase 2 Study to Evaluate the Efficacy and Safety of Oral BIIB061 as Add-On Therapy to Interferon-Beta 1 or Glatiramer Acetate Therapies in Relapsing Multiple Sclerosis A Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Phase 2 Study to Evalu ... | Relapsing Multiple Sclerosis | Drug: Placebo;Drug: BIIB061;Biological: Interferon-beta1;Drug: Glatiramer acetate | Biogen | NULL | Not yet recruiting | 18 Years | 55 Years | All | 300 | Phase 2 | NULL |
| 7 | EUCTR2020-004505-32-FR (EUCTR) | 09/06/2021 | 26/04/2021 | Study of efficacy and tolerability of ofatumumab vs. First Line disease modifying treatment (DMT) - physician’s choice in the treatment of newly diagnosed relapsing multiple sclerosis (RMS) patients Study of efficacy and tolerability of ofatumumab vs. First Line disease modifying treatment (DMT) - ... | An Open-Label, Rater-Blind, Randomized, Multi-Center, Parallel-Arm, Active-Comparator Study to Assess the Efficacy and Tolerability of Ofatumumab 20mg SC monthly vs. First Line DMT-physician’s choice in the treatment of newly diagnosed RMS - STHENOS An Open-Label, Rater-Blind, Randomized, Multi-Center, Parallel-Arm, Active-Comparator Study to Asses ... | Multiple Sclerosis MedDRA version: 20.0;Level: PT;Classification code 10048393;Term: Multiple sclerosis relapse;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Multiple Sclerosis MedDRA version: 20.0;Level: PT;Classification code 10048393;Term: Multiple sclero ... | Trade Name: Kesimpta Product Name: ofatumumab Product Code: OMB157 INN or Proposed INN: OFATUMUMAB Product Name: Glatiramer acetate Other descriptive name: GLATIRAMER ACETATE Product Name: Glatiramer acetate Other descriptive name: GLATIRAMER ACETATE Product Name: Interferon ß-1b Other descriptive name: INTERFERON BETA-1B Product Name: Interferon ß-1a INN or Proposed INN: Interferon beta-1a Product Name: Interferon ß-1a INN or Proposed INN: Interferon beta-1a Product Name: Interferon ß-1a INN or Proposed INN: Interferon beta-1a Trade Name: Kesimpta Product Name: ofatumumab Product Code: OMB157 INN or Proposed INN: OFATUMUMAB Produ ... | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 236 | Phase 3 | France;Germany | ||
| 8 | EUCTR2020-005752-38-DE (EUCTR) | 18/03/2021 | 19/01/2021 | An open-label multicenter study to assess response to SARS-CoV-2 modRNA vaccines in participants with secondary progressive multiple sclerosis treated with Mayzent (siponimod) (AMA-VACC) An open-label multicenter study to assess response to SARS-CoV-2 modRNA vaccines in participants wit ... | An open-label multicenter study to assess response to SARS-CoV-2 modRNA vaccines in participants with secondary progressive multiple sclerosis treated with Mayzent (siponimod) (AMA-VACC) An open-label multicenter study to assess response to SARS-CoV-2 modRNA vaccines in participants wit ... | secondary progressive multiple sclerosis (SPMS);Therapeutic area: Diseases [C] - Nervous System Diseases [C10] secondary progressive multiple sclerosis (SPMS);Therapeutic area: Diseases [C] - Nervous System Dise ... | Trade Name: Mayzent 2 mg Filmtabletten Product Name: Mayzent 2 mg Filmtabletten Product Code: BAF312A INN or Proposed INN: SIPONIMOD Other descriptive name: Siponimod Trade Name: Mayzent 0,25 mg Filmtabletten Product Name: Mayzent 0,25 mg Filmtabletten Product Code: BAF312A INN or Proposed INN: SIPONIMOD Other descriptive name: Siponimod Trade Name: Spikevax INN or Proposed INN: COVID-19 mRNA Vaccine (nucleoside modified) Other descriptive name: COVID-19 mRNA vaccine Moderna (CX-024414) INN or Proposed INN: DIMETHYL FUMARATE Other descriptive name: DIMETHYL FUMARATE INN or Proposed INN: TERIFLUNOMIDE INN or Proposed INN: GLATIRAMER ACETATE Other descriptive name: GLATIRAMER ACETATE INN or Proposed INN: RECOMBINANT INTERFERON BE Trade Name: Mayzent 2 mg Filmtabletten Product Name: Mayzent 2 mg Filmtabletten Product Code: BAF312A I ... | Novartis Pharma GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 4 | Germany | ||
| 9 | EUCTR2018-000284-93-EE (EUCTR) | 10/12/2019 | 02/10/2019 | A multinational, multicenter, randomized, Phase III, double blind, parallel group, placebo controlled study in subjects with Relapsing forms of Multiple Sclerosis (RMS) to assess the efficacy, safety and tolerability of GA Depot, a long acting IM injection of glatiramer acetate, administered once every four weeks A multinational, multicenter, randomized, Phase III, double blind, parallel group, placebo controlle ... | A multinational, multicenter, randomized, Phase III, double blind, parallel group, placebo controlled study in subjects with Relapsing forms of Multiple Sclerosis (RMS) to assess the efficacy, safety and tolerability of GA Depot, a long acting IM injection of glatiramer acetate, administered once every four weeks A multinational, multicenter, randomized, Phase III, double blind, parallel group, placebo controlle ... | Relapsing forms of Multiple Sclerosis (RMS) MedDRA version: 20.1;Level: PT;Classification code 10028245;Term: Multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Relapsing forms of Multiple Sclerosis (RMS) MedDRA version: 20.1;Level: PT;Classification code 10028 ... | Product Name: GA Depot INN or Proposed INN: GLATIRAMER ACETATE | Mapi Pharma Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 960 | Phase 3 | United States;Serbia;Belarus;Estonia;Ukraine;Russian Federation;Israel;Montenegro;Czech Republic;Macedonia, the former Yugoslav Republic of;Poland;Croatia;Romania;Bulgaria;Georgia;Moldova, Republic of;Bosnia and Herzegovina United States;Serbia;Belarus;Estonia;Ukraine;Russian Federation;Israel;Montenegro;Czech Republic;Mac ... | ||
| 10 | EUCTR2018-000284-93-BG (EUCTR) | 15/11/2019 | 08/10/2019 | A multinational, multicenter, randomized, Phase III, double blind, parallel group, placebo controlled study in subjects with Relapsing forms of Multiple Sclerosis (RMS) to assess the efficacy, safety and tolerability of GA Depot, a long acting IM injection of glatiramer acetate, administered once every four weeks A multinational, multicenter, randomized, Phase III, double blind, parallel group, placebo controlle ... | A multinational, multicenter, randomized, Phase III, double blind, parallel group, placebo controlled study in subjects with Relapsing forms of Multiple Sclerosis (RMS) to assess the efficacy, safety and tolerability of GA Depot, a long acting IM injection of glatiramer acetate, administered once every four weeks A multinational, multicenter, randomized, Phase III, double blind, parallel group, placebo controlle ... | Relapsing forms of Multiple Sclerosis (RMS) MedDRA version: 20.1;Level: PT;Classification code 10028245;Term: Multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Relapsing forms of Multiple Sclerosis (RMS) MedDRA version: 20.1;Level: PT;Classification code 10028 ... | Product Name: GA Depot INN or Proposed INN: GLATIRAMER ACETATE | Mapi Pharma Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 1000 | Phase 3 | United States;Belarus;Estonia;Ukraine;Russian Federation;Israel;Georgia;Bulgaria;Montenegro;Moldova, Republic of;Bosnia and Herzegovina United States;Belarus;Estonia;Ukraine;Russian Federation;Israel;Georgia;Bulgaria;Montenegro;Moldova, ... |
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
Showing 1 to 10 of 39 diseases
| 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 31, 2022 | 24/6/2019 | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease | An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated With Hemodialysis An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With F ... | Fabry Disease | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Recruiting | 18 Years | N/A | All | 14 | Phase 3 | United States;Australia;France;Japan;Spain;Belgium;Italy;United Kingdom |
| 2 | 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 MIGALASTAT IN SUBJECTS > 12 YEARS OF AGE WITH FABRY DISEASE AND AMENABLE GLA VARIANTS A LONG-TERM, OPEN-LABEL STUDY TO EVALUATE THE SAFETY, PHARMACODYNAMICS, AND EFFICACY OF MIGALASTAT I ... | Fabry disease and with amenable GLA variants 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] Fabry disease and with amenable GLAvariants MedDRA version: 20.0;Level: PT;Classification code 10016 ... | Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE 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 | ||
| 3 | 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 TREATMENT WITH MIGALASTAT IN PEDIATRIC SUBJECTS (AGED 12 TO <18 YEARS) WITH FABRY DISEASE AND AMENABLE GLA VARIANTS 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 TREATMENT WITH MIGALASTAT IN PEDIATRIC SUBJECTS (AGED 12 TO <18 YEARS) WITH FABRY DISEASE AND AMENABLE GLA VARIANTS - ASPIRE 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 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] Fabry disease and with amenable GLAvariants MedDRA version: 20.0;Level: PT;Classification code 10016 ... | Trade Name: Galafold Product Name: Migalastat Product Code: [AT1001] INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE 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 | ||
| 4 | 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 TREATMENT WITH MIGALASTAT IN PEDIATRIC SUBJECTS (AGED 12 TO <18 YEARS) WITH FABRY DISEASE AND AMENABLE GLA VARIANTS 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 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] Fabry disease and with amenable GLAvariants MedDRA version: 20.0;Level: PT;Classification code 10016 ... | Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE 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 | ||
| 5 | 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 TREATMENT WITH MIGALASTAT IN PEDIATRIC SUBJECTS (AGED 12 TO <18 YEARS) WITH FABRY DISEASE AND AMENABLE GLA VARIANTS 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 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] Fabry disease and with amenable GLAvariants MedDRA version: 20.0;Level: PT;Classification code 10016 ... | Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other ... | Amicus Therapeutics, UK Ltd | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | United States;Spain;Australia;Germany;United Kingdom;Italy | ||
| 6 | NCT03500094 (ClinicalTrials.gov) | October 11, 2018 | 9/4/2018 | Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 1 ... | An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants 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 |
| 7 | NCT05056636 (ClinicalTrials.gov) | June 1, 2018 | 31/8/2021 | Epidemiological Study of Fabry Disease Screening in Chronic Kidney Disease Patients | Epidemiological Study of Fabry Disease Screening in Chronic Kidney Disease Patients | Fabry Disease | Diagnostic Test: Plasma a-Gal A activity; Plasma Lyso-GB3; GLA genetic sequencing. | Chang Gung Memorial Hospital | NULL | Recruiting | 18 Years | N/A | All | 2000 | Taiwan | |
| 8 | NCT03596398 (ClinicalTrials.gov) | January 1, 2016 | 15/6/2018 | Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients | Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients | Fabry Disease | Genetic: GLA gene | Chiayi Christian Hospital | Sanofi | Enrolling by invitation | 20 Years | 55 Years | All | 1000 | NULL | |
| 9 | EUCTR2014-002701-38-DK (EUCTR) | 15/05/2015 | 20/01/2015 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry Disease A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in ... | An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlo ... | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 19.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: NA Other descriptive name: Inactive Reminder Capsules Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descr ... | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Denmark;Australia;Japan United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 10 | EUCTR2014-002701-38-ES (EUCTR) | 12/03/2015 | 16/01/2015 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry Disease A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in ... | An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlo ... | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descr ... | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Japan United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil ... |