19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | NCT00813865 (ClinicalTrials.gov) | May 11, 2009 | 22/12/2008 | A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients | An Open-Label, Multicenter, Long-Term Extension Study To Assess The Safety, Efficacy, And Pharmacodynamics Of AT2101 In Adult Patients With Type 1 Gaucher Disease | Gaucher Disease;Type 1 Gaucher Disease;Gaucher Disease, Type 1 | Drug: afegostat tartrate | Amicus Therapeutics | NULL | Completed | 18 Years | N/A | All | 8 | Phase 2 | United States;United Kingdom;Paraguay |
| 2 | NCT00875160 (ClinicalTrials.gov) | April 2009 | 2/4/2009 | A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101 | An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of Repeated Doses of Orally Administered AT2101 in Adult Patients With Type 1 Gaucher Disease | Type 1 Gaucher Disease | Drug: AT2101 | Amicus Therapeutics | NULL | Terminated | 18 Years | 65 Years | Both | 1 | Phase 1 | United States;United Kingdom |
| 3 | EUCTR2008-007158-36-GB (EUCTR) | 25/02/2009 | 29/04/2009 | An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease. | An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease. | Type I Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: afegostat tartrate Product Code: AT2101 INN or Proposed INN: afegostat tartrate | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 16 | United Kingdom | |||
| 4 | NCT00446550 (ClinicalTrials.gov) | June 11, 2008 | 9/3/2007 | A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease | A Randomized, Open-label Study To Assess the Safety and Tolerability of AT2101 in Treatment-naive Adult Patients With Type 1 Gaucher Disease | Gaucher Disease;Type 1 Gaucher Disease;Gaucher Disease, Type 1 | Drug: afegostat tartrate | Amicus Therapeutics | NULL | Completed | 18 Years | 74 Years | All | 19 | Phase 2 | United States;Israel;South Africa;United Kingdom;Germany |
| 5 | EUCTR2007-003359-35-DE (EUCTR) | 28/12/2007 | 22/12/2008 | A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE | A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE | Type 1 Gaucher Disease | Product Name: Isofagamine tartrate Product Code: AT2101 | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 16 | United Kingdom;Germany | |||
| 6 | EUCTR2007-003359-35-GB (EUCTR) | 19/12/2007 | 14/09/2007 | A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE | A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE | Type 1 Gaucher Disease | Product Name: Isofagamine tartrate Product Code: AT2101 | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 16 | Germany;United Kingdom | |||
| 7 | NCT00433147 (ClinicalTrials.gov) | March 23, 2007 | 7/2/2007 | A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy | A Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy | Gaucher Disease, Type 1;Type 1 Gaucher Disease;Gaucher Disease | Drug: Afegostat tartrate | Amicus Therapeutics | NULL | Completed | 18 Years | 74 Years | All | 30 | Phase 2 | United States |