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IV trehalose for lysosomal storage disorders
IRCT20130829014521N23Phase 2Completed
- Sponsor
- Deputy for Research and Technology, Ministry of Health and Medical Education
- Start
- Apr 2024
- Modality
- DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules, Drug Delivery Systems
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of trehalose in acid sphingomyelinase deficiency (ASMD) and Sanfilippo syndrome, sponsored by Deputy for Research and Technology, Ministry of Health and Medical Education. Primary endpoint: Quality of life. Started Apr 2024; status: completed.
02
Drugs
trehalose
03
Conditions
acid sphingomyelinase deficiency (ASMD)Sanfilippo syndrome
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