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.
| 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 | NCT04125927 (ClinicalTrials.gov) | January 2021 | 3/10/2019 | Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) | 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 | Cystinosis | Drug: Mercaptamine | Recordati Rare Diseases | NULL | Not yet recruiting | 6 Months | 2 Years | All | 5 | Phase 3 | NULL |
| 2 | NCT04246060 (ClinicalTrials.gov) | July 31, 2020 | 27/1/2020 | Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients | Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectiveness of Extended Release (ER) Oral Cysteamine Therapy (Procysbi) in Belgian Patients Suffering From Nephropathic Cystinosis | Nephropathic Cystinosis | Drug: Cysteamine Bitartrate | Chiesi SA/NV | NULL | Enrolling by invitation | N/A | N/A | All | 31 | Belgium | |
| 3 | EUCTR2018-002984-24-DE (EUCTR) | 30/06/2020 | 10/01/2020 | 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 | Study 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 Diseases | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 3 | France;Germany;United Kingdom | ||
| 4 | EUCTR2018-002984-24-GB (EUCTR) | 25/10/2019 | 15/07/2019 | 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] | 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 SARL | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 3 | France;United Kingdom | ||
| 5 | EUCTR2013-003228-35-ES (EUCTR) | 16/07/2014 | 12/11/2013 | Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients | Study 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) | NULL | Not Recruiting | Female: yes Male: yes | Spain | ||||
| 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 |
| 6 | EUCTR2012-002773-64-IT (EUCTR) | 12/02/2014 | 21/11/2013 | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis - RP103-07 | 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] - 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 Product Name: Cystagon INN or Proposed INN: Cystagon Other descriptive name: Cysteamine bitartrate Trade Name: CYSTAGON Product Name: Cystagon INN or Proposed INN: Cystagon Other descriptive name: Cysteamine bitartrate | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 7 | EUCTR2012-002773-64-GB (EUCTR) | 22/01/2014 | 26/02/2013 | Study comparing the effectiveness of cysteamine bitartrate delayed release capsules (RP103) to Cystagon in Patients with Cystinosis(Adults and Children 12 years and older). | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine 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. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 8 | EUCTR2012-002773-64-BE (EUCTR) | 21/01/2014 | 19/04/2013 | A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;France;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 9 | EUCTR2012-002773-64-NL (EUCTR) | 15/08/2013 | 01/11/2013 | Study 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. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Belgium;Germany;Netherlands;Italy;United Kingdom | ||
| 10 | EUCTR2012-002773-64-FR (EUCTR) | 03/06/2013 | 28/09/2015 | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis - RP103-07 | Cystinosis MedDRA version: 18.0;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] | Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate Trade Name: Cystagon Product Name: Cystagon INN or Proposed INN: MERCAPTAMINE bitartrate | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;France;Belgium;Netherlands;Italy;United Kingdom | ||
| 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 |
| 11 | NCT01733316 (ClinicalTrials.gov) | January 31, 2013 | 16/11/2012 | Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis | A Long-Term, Open-Label, Safety, Tolerability and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis | Cystinosis | Drug: RP103;Drug: Cystagon® | Horizon Pharma USA, Inc. | NULL | Completed | 12 Years | N/A | All | 41 | Phase 3 | United States;Belgium;France;Italy;Netherlands;United Kingdom |
| 12 | EUCTR2009-012564-13-FR (EUCTR) | 03/01/2013 | 19/06/2013 | Cysteamine Hydrochloride for nephrOpathic Cystinosis, open-label Phase III pivotal study | Cysteamine 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 SARL | NULL | Not Recruiting | Female: yes Male: yes | Phase 3 | France | |||
| 13 | NCT01744782 (ClinicalTrials.gov) | December 20, 2012 | 5/12/2012 | Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis | An Open-Label, Safety and Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naïve Patients With Cystinosis | Cystinosis | Drug: RP103 | Horizon Pharma USA, Inc. | NULL | Completed | N/A | 6 Years | All | 17 | Phase 3 | United States;Brazil |
| 14 | NCT02012114 (ClinicalTrials.gov) | December 2011 | 11/12/2012 | A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications | A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications | Cystinosis | Other: Cysteamine bitartrate | Hospices Civils de Lyon | NULL | Recruiting | 4 Years | N/A | Both | 90 | N/A | France |
| 15 | NCT01432561 (ClinicalTrials.gov) | September 2011 | 9/9/2011 | Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™ | Food-Effect on Bioavailability of Cystagon™ in Normal, Healthy Adults | Cystinosis;Nephropathic Cystinosis | Drug: Cysteamine bitartrate | University of California, San Diego | Raptor Pharmaceuticals Corp. | Completed | 18 Years | N/A | All | 8 | N/A | United States |
| 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 |
| 16 | EUCTR2010-018365-34-NL (EUCTR) | 14/12/2010 | 06/07/2010 | A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Netherlands | ||
| 17 | EUCTR2009-017882-42-NL (EUCTR) | 19/10/2010 | 25/06/2010 | A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis | A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis | 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 Trade Name: Cystagon | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Netherlands;France | |||
| 18 | EUCTR2010-020098-18-FR (EUCTR) | 09/09/2010 | 12/07/2010 | CrYSTobs A cohort of patients with cystinosis : compliance to cysteamine and neurological complicationsAn auxiliary study to Raptor RP103 03 and RP103 04 - CrYSTobs | CrYSTobs A cohort of patients with cystinosis : compliance to cysteamine and neurological complicationsAn auxiliary study to Raptor RP103 03 and RP103 04 - CrYSTobs | nephropatic cystinosis MedDRA version: 12.1;Level: LLT;Classification code 10011777;Term: Cystinosis | Trade Name: CYSTAGON Product Name: CYSTAGON INN or Proposed INN: MERCAPTAMINE BITARTRATE Other descriptive name: CYSTEAMINE Product Name: RP 103 INN or Proposed INN: MERCAPTAMINE BITARTRATE Other descriptive name: CYSTEAMINE Product Name: RP 103 INN or Proposed INN: MERCAPTAMINE BITARTRATE Other descriptive name: CYSTEAMINE | HOSPICES CIVILS DE LYON | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | France | ||||
| 19 | NCT01197378 (ClinicalTrials.gov) | August 27, 2010 | 3/9/2010 | Long-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 Cystinosis | Cystinosis | Drug: Cysteamine Bitartrate Delayed-release Capsules | Horizon Pharma USA, Inc. | NULL | Completed | 1 Year | N/A | All | 60 | Phase 3 | United States;France;Netherlands |
| 20 | EUCTR2009-017882-42-FR (EUCTR) | 12/08/2010 | 14/06/2010 | A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis | A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis | 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 INN or Proposed INN: mercaptamine bitartrate Trade Name: Cystagon | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | Phase 3 | France;Netherlands | |||
| 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 |
| 21 | NCT01000961 (ClinicalTrials.gov) | June 2010 | 22/10/2009 | Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis | A 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 Cystinosis | Cystinosis | Drug: Cystagon® (Cysteamine Bitartrate);Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103) | Horizon Pharma USA, Inc. | NULL | Completed | 6 Years | N/A | All | 43 | Phase 3 | United States;France;Netherlands |
| 22 | NCT00872729 (ClinicalTrials.gov) | May 2009 | 27/3/2009 | Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis | A 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 Cystinosis | Cystinosis | Drug: Cystagon®;Drug: RP103 | Horizon Pharma USA, Inc. | NULL | Completed | N/A | N/A | All | 9 | Phase 1;Phase 2 | United States |
| 23 | EUCTR2007-006024-35-FR (EUCTR) | 04/02/2008 | 12/12/2007 | Adaptive 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 | Adaptive 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 SARL | NULL | Not Recruiting | Female: yes Male: yes | France | ||||
| 24 | NCT00010426 (ClinicalTrials.gov) | December 1999 | 2/2/2001 | Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis | Cystinosis | Drug: cysteamine hydrochloride | FDA Office of Orphan Products Development | Sigma Tau Pharmaceuticals, Inc. | Completed | 1 Year | 50 Years | Both | 30 | N/A | NULL | |
| 25 | NCT00001736 (ClinicalTrials.gov) | May 1998 | 3/11/1999 | New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis | Safety and Efficacy Trial of a Proposed NDA Formulation of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis | Cystinosis | Drug: Cysteamine | National Eye Institute (NEI) | NULL | Completed | N/A | N/A | Both | 51 | Phase 1 | United States |
| 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 |
| 26 | NCT00001213 (ClinicalTrials.gov) | April 1986 | 3/11/1999 | Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis | Trial of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis | Cystinosis | Drug: Cysteamine | National Eye Institute (NEI) | NULL | Completed | 2 Years | N/A | All | 328 | Phase 2 | United States |
| 27 | EUCTR2018-002984-24-BE (EUCTR) | 02/10/2020 | 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 | Study 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 Diseases | NULL | NA | Female: yes Male: yes | 5 | Phase 3 | France;Belgium;Germany;Italy;United Kingdom | |||
| 28 | EUCTR2018-002984-24-IT (EUCTR) | 21/10/2020 | Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old | 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 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 Product Code: [045251016] INN or Proposed INN: Mercaptamina Other descriptive name: ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride | Recordati Rare Diseases | NULL | NA | Female: yes Male: yes | 5 | Phase 3 | France;Belgium;Germany;United Kingdom;Italy | |||
| 29 | EUCTR2010-018365-34-FR (EUCTR) | 18/06/2010 | A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04 | A 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. | NULL | NA | Female: yes Male: yes | 20 | Phase 3 | France;Netherlands |