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

  
6 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-002984-24-BE
(EUCTR)
11/02/202102/10/2020Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years OldStudy to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old - SCOB2 (Study Cystadrops® Ophthalmic for Patients Below 2 years) Nephropatic Cystinosis patients with corneal cystine crystal deposits
MedDRA version: 20.0;Level: LLT;Classification code 10071112;Term: Nephropathic cystinosis;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: Cystadrops
Product Name: Cystadrops
INN or Proposed INN: Cysteamine hydrochloride
Other descriptive name: ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride
Recordati Rare DiseasesNULLNot RecruitingFemale: yes
Male: yes
5Phase 3France;Belgium;Germany;Italy;United Kingdom
2EUCTR2018-002984-24-DE
(EUCTR)
30/06/202010/01/2020Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years OldStudy to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old - SCOB2 (Study Cystadrops® Ophthalmic for Patients Below 2 years) Nephropatic Cystinosis patients with corneal cystine crystal deposits
MedDRA version: 20.0;Level: LLT;Classification code 10071112;Term: Nephropathic cystinosis;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: Cystadrops
Product Name: Cystadrops
INN or Proposed INN: Cysteamine hydrochloride
Other descriptive name: ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride
Recordati Rare DiseasesNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 3France;Germany;United Kingdom
3EUCTR2018-002984-24-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
4EUCTR2009-012564-13-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
5EUCTR2007-006024-35-FR
(EUCTR)
04/02/200812/12/2007Adaptive dose regimen of Cystadrops for cOrneal Crystal deposiTs and ocular manifestations in nephropathic cystinosis : an open label, dose-response pilot study - CYSTADROPS OCT-1 pilot studyAdaptive dose regimen of Cystadrops for cOrneal Crystal deposiTs and ocular manifestations in nephropathic cystinosis : an open label, dose-response pilot study - CYSTADROPS OCT-1 pilot study Nephropathic cystinotic patients with cystine crystals corneal deposits
MedDRA version: 9.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders
Product Name: CYSTADROPS
INN or Proposed INN: cysteamine hydrochloride
Other descriptive name: mercaptamine, mercamine, 2-aminoethanethiol
Orphan Europe SARLNULLNot RecruitingFemale: yes
Male: yes
France
6NCT00010426
(ClinicalTrials.gov)
December 19992/2/2001Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With CystinosisCystinosisDrug: cysteamine hydrochlorideFDA Office of Orphan Products DevelopmentSigma Tau Pharmaceuticals, Inc.Completed1 Year50 YearsBoth30N/ANULL