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

  
9 trials found
No.TrialIDDate_
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PhaseCountries
1NCT04860960
(ClinicalTrials.gov)
July 20, 202115/4/2021Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1Phase 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 C1Drug: Hydroxypropyl-beta-cyclodextrin;Drug: PlaceboCyclo Therapeutics, Inc.NULLRecruiting3 YearsN/AAll93Phase 3United States
2NCT03471143
(ClinicalTrials.gov)
February 22, 20197/3/2018Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type CPhase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type CNiemann-Pick Disease, Type CDrug: 2-Hydroxypropyl-Beta-CyclodextrinWashington University School of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Active, not recruitingN/A6 MonthsAll3Phase 1/Phase 2United States
3NCT02939547
(ClinicalTrials.gov)
October 11, 201721/9/2016Study 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 DiseaseNiemann-Pick Disease, Type C1Drug: Hydroxypropyl-beta-cyclodextrinCyclo Therapeutics, Inc.NULLCompleted18 YearsN/AAll13Phase 1United States
4NCT02912793
(ClinicalTrials.gov)
March 20, 201719/8/2016Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C PatientsA 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 OutcomesNiemann-Pick Disease, Type C1Drug: Hydroxypropyl-beta-cyclodextrinCyclo Therapeutics, Inc.NULLCompleted2 YearsN/AAll12Phase 1/Phase 2Israel;Sweden;United Kingdom
5EUCTR2015-005761-23-SE
(EUCTR)
19/01/201709/09/2016A 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 patientsA 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.NULLNot RecruitingFemale: yes
Male: yes
11Phase 1;Phase 2United Kingdom;Italy;Sweden
6JPRN-UMIN000023517
2016/10/0101/10/2016A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C Niemann-Pick disease type C2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three yearsNara Medical UniversityNULLPending4years-old6years-oldMale and Female1Not selectedJapan
7EUCTR2015-005761-23-GB
(EUCTR)
26/09/201603/08/2016A 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 patientsA 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.NULLAuthorised-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): noItaly;United Kingdom;Sweden
8JPRN-UMIN000020032
2014/12/1902/12/2015Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC) Niemann-Pick disease type C2-hydroxypropyl-beta-cyclodextrin (HPBCD) 100-400mg, every 4 week, two yearsJichi Medical University, Saitama Medical CenterSaga UniversityKumamoto UniversityTohoku UniversityInstitute for Neurological DisordersComplete: follow-up continuing20years-oldNot applicableMale and Female2Not applicableJapan
9JPRN-UMIN000023094
2014/08/0110/07/2016A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick disease type C Niemann-Pick disease type C2-hydroxypropyl-beta-cyclodextrin (HPBCD) 5-20mg/kg, every 4 weeks, for two yearsNara Medical UniversitySaga UniversityKumamoto UniversityComplete: follow-up continuing1years-old25years-oldMale and Female1Not selectedJapan