19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

  
3 trials found
No.TrialIDDate_
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PhaseCountries
1NCT04628871
(ClinicalTrials.gov)
November 3, 20209/11/2020Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIXLong-Term Follow-up of Subjects Who Were Treated With SB-318, SB-913, or SB-FIX, for Targeted Genome Editing Into the Albumin Gene in the LiverHemophilia B;Mucopolysaccharidosis I;Mucopolysaccharidosis IIBiological: SB-318;Biological: SB-913;Biological: SB-FIXSangamo TherapeuticsNULLEnrolling by invitation18 YearsN/AAll13United States
2EUCTR2018-000192-33-GB
(EUCTR)
17/08/201809/04/2018A phase 1/2 study to evaluate the safety and tolerability of SB-913, a rAAV2/6-based gene therapy in patients with Mucopolysaccharidosis II (MPS II)A Phase 1/2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPS II) Mucopolysaccharidosis type II (MPS II)
MedDRA version: 20.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: rAAV2/6 Left ZFN Vector
Product Code: SB-47171
INN or Proposed INN: Not yet assigned
Other descriptive name: Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector
Product Name: rAAV2/6 Right ZFN Vector
Product Code: SB-47898
INN or Proposed INN: Not yet assigned
Other descriptive name: Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector
Product Name: rAAV 2/6 hIDS DONOR
Product Code: hIDS DONOR
INN or Proposed INN: Not yet assigned
Other descriptive name: Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector
Sangamo Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
32 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
3NCT03041324
(ClinicalTrials.gov)
May 11, 201713/1/2017Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS IIA Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis II (MPS II)Mucopolysaccharidosis II;MPS IIBiological: SB-913Sangamo TherapeuticsNULLTerminated5 YearsN/AAll9Phase 1/Phase 2United States