Other: no it treatment    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
19Lysosomal storage disease1

19. Lysosomal storage disease    [ 784 clinical trials,   673 drugs,   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways]
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-ganglio ... show all
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 784 trial found
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PhaseCountries
1NCT02055118
(ClinicalTrials.gov)
March 24, 201417/1/2014Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive ImpairmentA Controlled, Randomized, Two-arm, Open-label, Assessor-blinded, Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive ImpairmentHunter SyndromeBiological: idursulfase-IT;Other: No IT treatmentShireNULLCompletedN/A18 YearsMale58Phase 2;Phase 3United States;Australia;Canada;France;Mexico;Spain;United Kingdom;Argentina;Colombia