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
Showing 1 to 10 of 854 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 1, 2022 | 24/6/2019 | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairm ... | An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects ... | 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 | NCT04552691 (ClinicalTrials.gov) | June 2022 | 11/9/2020 | Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients | Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients | Fabry Disease | Drug: Pegunigalsidase Alfa | Protalix | Chiesi USA, Inc. | Available | 18 Years | N/A | All | United States | ||
| 3 | NCT05058391 (ClinicalTrials.gov) | April 30, 2022 | 24/9/2021 | A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India | A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II) A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Sa ... | Hunter Syndrome | Biological: Elaprase | Shire | NULL | Not yet recruiting | N/A | N/A | All | 5 | Phase 4 | India |
| 4 | NCT05067868 (ClinicalTrials.gov) | April 30, 2022 | 24/9/2021 | A Study of Replagal in Children and Adults With Fabry Disease in India | A 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 Disease A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the S ... | Fabry Disease | Biological: Replagal | Shire | NULL | Not yet recruiting | N/A | N/A | All | 5 | Phase 4 | India |
| 5 | NCT05222906 (ClinicalTrials.gov) | March 30, 2022 | 20/1/2022 | Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher ... | A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT) A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Stu ... | Gaucher's Disease Type III | Drug: Venglustat;Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Not yet recruiting | 12 Years | N/A | All | 40 | Phase 3 | NULL |
| 6 | EUCTR2021-005356-10-SK (EUCTR) | 15/03/2022 | 28/12/2021 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C(NPC): A Phase III, randomized, placebo- ... | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Niemann-Pick Disease type C(NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ... | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other des ... | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Slovakia;Australia;Netherlands;Germany;United Kingdom;Switzerland | ||
| 7 | NCT05206773 (ClinicalTrials.gov) | March 11, 2022 | 20/10/2021 | A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and F ... | A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Adults With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Ven ... | Fabry Disease | Drug: Venglustat (GZ402671);Drug: Placebo | Genzyme, a Sanofi Company | NULL | Recruiting | 18 Years | N/A | All | 114 | Phase 3 | United States |
| 8 | NCT04316637 (ClinicalTrials.gov) | March 9, 2022 | 18/3/2020 | Early Access Program With Arimoclomol in US Patients With NPC | Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US | Niemann-Pick Disease, Type C | Drug: Arimoclomol | Orphazyme | NULL | Available | 2 Years | N/A | All | United States | ||
| 9 | EUCTR2020-003136-25-DE (EUCTR) | 07/03/2022 | 17/06/2021 | Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1 Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ and Stand ... | A Phase 3, Double blind, Randomized, Placebo controlled, Parallel group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ (Hydroxypropyl ß cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1 A Phase 3, Double blind, Randomized, Placebo controlled, Parallel group, Multicenter Study to Evalua ... | Niemann Pick Disease Type C1 MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] Niemann Pick Disease Type C1 MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niema ... | Product Name: Trappsol® Cyclo™ INN or Proposed INN: NA Other descriptive name: HYDROXYPROPYLBETADEX | Cyclo Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | France;United States;Poland;Australia;Israel;Germany;Italy;United Kingdom | ||
| 10 | EUCTR2021-005356-10-DE (EUCTR) | 04/03/2022 | 29/12/2021 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C(NPC): A Phase III, randomized, placebo- ... | Niemann-Pick Disease type C (NPC);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Niemann-Pick Disease type C(NPC);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonat ... | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other des ... | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Slovakia;Spain;Australia;Netherlands;Germany;United Kingdom;Switzerland |