19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 854 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191

  
3 trials found
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1EUCTR2016-002328-10-NL
(EUCTR)
29/10/201807/11/2018Gene Therapy in patients with Mucopolysaccharidosis diseaseA 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 TELETHONNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 1Turkey;Netherlands;Italy
2NCT03173521
(ClinicalTrials.gov)
July 17, 201712/4/2017Gene Therapy in Patients With Mucopolysaccharidosis DiseaseA 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 LiverMucopolysaccharidosis Type VIBiological: AAV2/8.TBG.hARSBFondazione TelethonNULLActive, not recruiting4 Years65 YearsAll9Phase 1/Phase 2Italy;Turkey;Netherlands
3EUCTR2016-002328-10-IT
(EUCTR)
22/02/201717/06/2021Gene Therapy in patients with Mucopolysaccharidosis diseaseA 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 TELETHONNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 1Turkey;Netherlands;Italy