Aavrh.10-hmpsiiia    (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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agemin
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PhaseCountries
1EUCTR2010-019962-10-FR
(EUCTR)
19/11/2010AN OPEN-LABEL, SINGLE ARM, MONOCENTRIC, PHASE I/II CLINICAL STUDY OF INTRACEREBRAL ADMINISTRATION OF ADENO-ASSOCIATED VIRAL VECTORS SEROTYPE 10 CARRYING THE HUMAN SGSH AND SUMF1 cDNAS FOR THE TREATMENT OF SANFILIPPO TYPE A SYNDROME - Intracerebral Gene therapy for Sanfilippo type A syndromeAN OPEN-LABEL, SINGLE ARM, MONOCENTRIC, PHASE I/II CLINICAL STUDY OF INTRACEREBRAL ADMINISTRATION OF ADENO-ASSOCIATED VIRAL VECTORS SEROTYPE 10 CARRYING THE HUMAN SGSH AND SUMF1 cDNAS FOR THE TREATMENT OF SANFILIPPO TYPE A SYNDROME - Intracerebral Gene therapy for Sanfilippo type A syndrome Sanfilippo type A syndrome (also named Mucopolysaccharidosis Type A)
MedDRA version: 12.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome
Product Name: AAVrh.10-hMPSIIIA
Product Code: SAF-301
SANFILIPPO Therapeutics SASNULLNot RecruitingFemale: yes
Male: yes
4Phase 1;Phase 2France