19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


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

  
3 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2018-002984-24-GB
(EUCTR)
25/10/201915/07/2019Open-label, Single-arm, Multicenter Study to Assess the Safety of, Cystadrops® in Pediatric Cystinosis Patients,from 6 Months to Less Than 2 Years Old [SCOB2 Study]Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old [SCOB2 Study] - SCOB2 Corneal cystine crystal deposits in pediatric patients withnephropathic cystinosis from 6 months to less than 2 years old;Therapeutic area: Diseases [C] - Eye Diseases [C11]Trade Name: Cystadrops
Product Name: Cystadrops
INN or Proposed INN: Mercaptamine hydrochloride
Other descriptive name: Cysteamine hydrochloride
RECORDATI Rare Diseases SARLNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 3France;United Kingdom
2EUCTR2013-003228-35-ES
(EUCTR)
16/07/201412/11/2013Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patientsStudy of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients - Viscou solution cysteamina Cystinosis
MedDRA version: 16.1;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: cysteamine (mercaptamine) viscous solution
INN or Proposed INN: Mercaptamine
Other descriptive name: MERCAPTAMINE HYDROCHLORIDE
Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)NULLNot RecruitingFemale: yes
Male: yes
Spain
3EUCTR2009-012564-13-FR
(EUCTR)
03/01/201319/06/2013Cysteamine Hydrochloride for nephrOpathic Cystinosis, open-label Phase III pivotal studyCysteamine Hydrochloride for nephropathic Cystinosis, open-label Phase III pivotal study - CYSTADROPS CHOC study Nephropatic cystinosis patients with cystine corneal deposits
MedDRA version: 16.0;Level: LLT;Classification code 10071112;Term: Nephropathic cystinosis;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: CYSTADROPS 0.55% eye drops, solution
INN or Proposed INN: CYSTEAMINE HYDROCHLORIDE
Other descriptive name: Mercaptamine hydrochloride
Orphan Europe SARLNULLNot RecruitingFemale: yes
Male: yes
Phase 3France