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 | EUCTR2016-002328-10-NL (EUCTR) | 29/10/2018 | 07/11/2018 | Gene Therapy in patients with Mucopolysaccharidosis disease | A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects withMucopolysaccharidosis type VI (MPS VI) Using Adeno-Associated ViralVector 8 to Deliver the human ARSB gene to Liver. | The clinical trial will be conducted on patients withMucopolysaccharidosis Type VI. MPS VI is characterized by growthretardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: AAV2/8.TBG.hARSB Product Code: not applicable INN or Proposed INN: NA Other descriptive name: ADENO-ASSOCIATED VIRAL (AAV) SEROTYPE 8 (AAV2/8) VECTOR WITH LIVER-SPECIFIC THYROXINE-BINDING GLOBULIN (TBG) PROMOTER, DRIVING THE EXPRESSION OF THE HUMAN ARSB GENE | FONDAZIONE TELETHON | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 1 | Turkey;Netherlands;Italy | ||
| 2 | NCT03173521 (ClinicalTrials.gov) | July 17, 2017 | 12/4/2017 | Gene Therapy in Patients With Mucopolysaccharidosis Disease | A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects With Mucopolysaccharidosis Type VI (MPS VI) Using Adeno-Associated Viral Vector 8 to Deliver the Human ARSB Gene to Liver | Mucopolysaccharidosis Type VI | Biological: AAV2/8.TBG.hARSB | Fondazione Telethon | NULL | Active, not recruiting | 4 Years | 65 Years | All | 9 | Phase 1/Phase 2 | Italy;Turkey;Netherlands |
| 3 | EUCTR2016-002328-10-IT (EUCTR) | 22/02/2017 | 17/06/2021 | Gene Therapy in patients with Mucopolysaccharidosis disease | A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects with Mucopolysaccharidosis type VI (MPS VI) Using Adeno-Associated Viral Vector 8 to Deliver the human ARSB gene to Liver. - TIGEM1-MPSVI | The clinical trial will be conducted on patients with Mucopolysaccharidosis Type VI. MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement MedDRA version: 21.1;Level: PT;Classification code 10059318;Term: Hepatic cancer stage I;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 20.1;Classification code 10054889;Term: Transaminases increased;System Organ Class: 10022891 - Investigations MedDRA version: 20.0;Level: LLT;Classification code 10037933;Term: Reaction anaphylactic anaphylactoid;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10019717;Term: Hepatitis;System Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: AAV2/8.TBG.hARSB Product Code: [AAV2/8.TBG.hARSB] | FONDAZIONE TELETHON | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 1 | Turkey;Netherlands;Italy |