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
| 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 | NCT04860960 (ClinicalTrials.gov) | July 20, 2021 | 15/4/2021 | Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 | Phase 3, Double-blind, Randomized, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of 2000mg/kg of Trappsol®Cyclo™ (Hydroxypropyl-B-cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients With Niemann-Pick Disease Type C1 (TransportNPC) | Niemann-Pick Disease, Type C1 | Drug: Hydroxypropyl-beta-cyclodextrin;Drug: Placebo | Cyclo Therapeutics, Inc. | NULL | Recruiting | 3 Years | N/A | All | 93 | Phase 3 | United States |
| 2 | NCT03471143 (ClinicalTrials.gov) | February 22, 2019 | 7/3/2018 | Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C | Phase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C | Niemann-Pick Disease, Type C | Drug: 2-Hydroxypropyl-Beta-Cyclodextrin | Washington University School of Medicine | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Active, not recruiting | N/A | 6 Months | All | 3 | Phase 1/Phase 2 | United States |
| 3 | NCT02939547 (ClinicalTrials.gov) | October 11, 2017 | 21/9/2016 | Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) | A Phase I Study to Evaluate the Single and Multiple-dose Pharmacokinetics of Intravenous Trappsol Cyclo (HP-Beta-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) and the Effects of Dosing Upon Biomarkers of NPC Disease | Niemann-Pick Disease, Type C1 | Drug: Hydroxypropyl-beta-cyclodextrin | Cyclo Therapeutics, Inc. | NULL | Completed | 18 Years | N/A | All | 13 | Phase 1 | United States |
| 4 | NCT02912793 (ClinicalTrials.gov) | March 20, 2017 | 19/8/2016 | Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients | A Phase I/II Study to Evaluate the Safety and PK of iv Trappsol Cyclo (HP-ß-CD) in Patients With Niemann-Pick Disease Type C NPC-1 and the Pharmacodynamic Effects of Treatment Upon Markers of Cholesterol Metabolism and Clinical Outcomes | Niemann-Pick Disease, Type C1 | Drug: Hydroxypropyl-beta-cyclodextrin | Cyclo Therapeutics, Inc. | NULL | Completed | 2 Years | N/A | All | 12 | Phase 1/Phase 2 | Israel;Sweden;United Kingdom |
| 5 | EUCTR2015-005761-23-SE (EUCTR) | 19/01/2017 | 09/09/2016 | A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Niemann-Pick disease type C (NPC-1) to assess what the drug does to the body, and what the body does to the drug, and the side effects and benefits experienced by patients | A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes | Niemann-Pick disease type C MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Trappsol Cyclo INN or Proposed INN: hydroxypropyl-beta-cyclodextrin Other descriptive name: hydroxypropyl-beta-cyclodextrin | Cyclo Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 11 | Phase 1;Phase 2 | United Kingdom;Italy;Sweden | ||
| 6 | JPRN-UMIN000023517 | 2016/10/01 | 01/10/2016 | A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C | Niemann-Pick disease type C | 2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three years | Nara Medical University | NULL | Pending | 4years-old | 6years-old | Male and Female | 1 | Not selected | Japan | |
| 7 | EUCTR2015-005761-23-GB (EUCTR) | 26/09/2016 | 03/08/2016 | A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Niemann-Pick disease type C (NPC-1) to assess what the drug does to the body, and what the body does to the drug, and the side effects and benefits experienced by patients | A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes | Niemann-Pick disease type C MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Trappsol Cyclo INN or Proposed INN: hydroxypropyl-beta-cyclodextrin Other descriptive name: hydroxypropyl-beta-cyclodextrin | CTD Holdings, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Italy;United Kingdom;Sweden | ||
| 8 | JPRN-UMIN000020032 | 2014/12/19 | 02/12/2015 | Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC) | Niemann-Pick disease type C | 2-hydroxypropyl-beta-cyclodextrin (HPBCD) 100-400mg, every 4 week, two years | Jichi Medical University, Saitama Medical Center | Saga UniversityKumamoto UniversityTohoku UniversityInstitute for Neurological Disorders | Complete: follow-up continuing | 20years-old | Not applicable | Male and Female | 2 | Not applicable | Japan | |
| 9 | JPRN-UMIN000023094 | 2014/08/01 | 10/07/2016 | A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick disease type C | Niemann-Pick disease type C | 2-hydroxypropyl-beta-cyclodextrin (HPBCD) 5-20mg/kg, every 4 weeks, for two years | Nara Medical University | Saga UniversityKumamoto University | Complete: follow-up continuing | 1years-old | 25years-old | Male and Female | 1 | Not selected | Japan |