19. Lysosomal storage disease Clinical trials / Disease details


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

  
1 trial found
No.TrialIDDate_
enrollment
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registration
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agemin
Inclusion_
agemax
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gender
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PhaseCountries
1NCT03580083
(ClinicalTrials.gov)
April 3, 201918/6/2018RGX-111 Gene Therapy in Patients With MPS IA Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 in Subjects With Mucopolysaccharidosis Type IMucopolysaccharidosis Type I (MPS I);Hurler Syndrome;Hurler-Scheie SyndromeGenetic: RGX-111REGENXBIO Inc.NULLActive, not recruiting4 MonthsN/AAll8Phase 1/Phase 2United States;Brazil;Israel