19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
9 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2012-002773-64-BE
(EUCTR)
21/01/201419/04/2013A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with CystinosisA Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis - RP103-07 Cystinosis
MedDRA version: 14.1;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate )
Product Code: RP103
INN or Proposed INN: mercaptamine bitartrate
Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate )
Product Code: RP103
INN or Proposed INN: mercaptamine bitartrate
Trade Name: Cystagon 50mg
Product Name: Cystagon
INN or Proposed INN: MERCAPTAMINE BITARTRATE
Trade Name: Cystagon 150mg
Product Name: Cystagon
INN or Proposed INN: MERCAPTAMINE BITARTRATE
Raptor Therapeutics Inc.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;France;Belgium;Netherlands;Germany;United Kingdom;Italy
2EUCTR2012-002773-64-NL
(EUCTR)
15/08/201301/11/2013Study comparing the effectiveness of cysteamine bitartrate delayed-release capsules (RP103) in Patients with Cystinosis (Adult and Children 12 years and older)A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis - Superiority, Safety and Efficacy Study of RP103 in Cystinosis Cystinosis
MedDRA version: 20.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: PROCYSBI
Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate)
Product Code: RP103
INN or Proposed INN: mercaptamine bitartrate
Trade Name: PROCYSBI
Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate)
Product Code: RP103
INN or Proposed INN: mercaptamine bitartrate
Trade Name: Cystagon 50mg
Product Name: Cystagon
INN or Proposed INN: mercaptamine bitartrate
Trade Name: Cystagon 150mg
Product Name: Cystagon
INN or Proposed INN: mercaptamine bitartrate
Horizon Pharma USA, Inc.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;United States;Belgium;Germany;Netherlands;Italy;United Kingdom
3NCT01733316
(ClinicalTrials.gov)
January 31, 201316/11/2012Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in CystinosisA Long-Term, Open-Label, Safety, Tolerability and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With CystinosisCystinosisDrug: RP103;Drug: Cystagon®Horizon Pharma USA, Inc.NULLCompleted12 YearsN/AAll41Phase 3United States;Belgium;France;Italy;Netherlands;United Kingdom
4NCT01744782
(ClinicalTrials.gov)
December 20, 20125/12/2012Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With CystinosisAn Open-Label, Safety and Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naïve Patients With CystinosisCystinosisDrug: RP103Horizon Pharma USA, Inc.NULLCompletedN/A6 YearsAll17Phase 3United States;Brazil
5EUCTR2010-018365-34-NL
(EUCTR)
14/12/201006/07/2010A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic CystinosisA Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04 Cystinosis
MedDRA version: 20.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: PROCYSBI
Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate )
Product Code: RP103
INN or Proposed INN: mercaptamine bitartrate
Trade Name: PROCYSBI
Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate )
Product Code: RP103
INN or Proposed INN: mercaptamine bitartrate
Horizon Pharma USA, Inc.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;United States;Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT01197378
(ClinicalTrials.gov)
August 27, 20103/9/2010Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With CystinosisCystinosisDrug: Cysteamine Bitartrate Delayed-release CapsulesHorizon Pharma USA, Inc.NULLCompleted1 YearN/AAll60Phase 3United States;France;Netherlands
7NCT01000961
(ClinicalTrials.gov)
June 201022/10/2009Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With CystinosisA Randomized, Crossover Pharmacokinetic and Pharmacodynamic Study to Determine the Safety and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cystagon® in Patients With Nephropathic CystinosisCystinosisDrug: Cystagon® (Cysteamine Bitartrate);Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)Horizon Pharma USA, Inc.NULLCompleted6 YearsN/AAll43Phase 3United States;France;Netherlands
8NCT00872729
(ClinicalTrials.gov)
May 200927/3/2009Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With CystinosisA Pilot Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cysteamine Bitartrate (Cystagon®) in Patients With Nephropathic CystinosisCystinosisDrug: Cystagon®;Drug: RP103Horizon Pharma USA, Inc.NULLCompletedN/AN/AAll9Phase 1;Phase 2United States
9EUCTR2010-018365-34-FR
(EUCTR)
18/06/2010A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04 Cystinosis
MedDRA version: 12.1;Level: LLT;Classification code 10011777;Term: Cystinosis
Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate )
Product Code: RP103
INN or Proposed INN: mercaptamine bitartrate
Raptor Therapeutics Inc.NULLNAFemale: yes
Male: yes
20Phase 3France;Netherlands