19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | 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 | 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 | 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] | 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 | ||
| 2 | EUCTR2020-003136-25-PL (EUCTR) | 02/02/2022 | 15/07/2021 | 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 | 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 | 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] | 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 | ||
| 3 | EUCTR2020-003136-25-FR (EUCTR) | 25/10/2021 | 10/06/2021 | 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 | 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 | 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] | 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 | United States;France;Australia;Israel;United Kingdom | ||
| 4 | EUCTR2020-003136-25-IT (EUCTR) | 23/08/2021 | 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 | 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 - Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ and Stand | 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] | Product Name: Trappsol® Cyclo™ Product Code: [N/A] INN or Proposed INN: N/A Other descriptive name: HYDROXYPROPYLBETADEX | Cyclo Therapeutics, Inc. | NULL | NA | Female: yes Male: yes | 105 | Phase 3 | France;United States;Spain;Poland;Australia;Israel;Germany;United Kingdom;Italy |