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Trehalose in lysosomal storage disorders
IRCT20201129049534N10Phase 3Completed
- Sponsor
- Isfahan University of Medical Sciences
- Start
- Feb 2025
- 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 3, open-label, single-arm trial of trehalose in Sanfilippo syndrome and acid sphingomyelinase deficiency (ASMD), sponsored by Isfahan University of Medical Sciences. Primary endpoints: Quality of life assessment, Biochemical markers and 1 more. Started Feb 2025; status: completed.
02
Drugs
trehalose
03
Conditions
Sanfilippo syndromeacid sphingomyelinase deficiency (ASMD)
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