19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 899 Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

  
3 trials found
No.TrialIDDate_
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agemin
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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 TherapeuticsNULLActive, not recruiting18 YearsN/AAll13United States
2EUCTR2018-000206-28-GB
(EUCTR)
14/08/201827/03/2018A phase 1/2 study to evaluate the safety and tolerability of SB-318, a rAAV2/6-based Gene Therapy, in patients with Mucopolysaccharidosis I (MPS I)A Phase 1/2, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis I (MPS I) Mucopolysaccharidosis type I (MPS I)
MedDRA version: 20.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;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: SB-A6P-HRL Donor Vector
Product Code: SB-IDUA
INN or Proposed INN: Not yet assigned
Other descriptive name: Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector
Sangamo Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
27 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
3NCT02702115
(ClinicalTrials.gov)
May 24, 201729/2/2016Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS IA Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)MPS IBiological: SB-318Sangamo TherapeuticsNULLActive, not recruiting5 YearsN/AAll3Phase 1/Phase 2United States