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Adrabetadex for Infantile Liver Disease in Niemann-Pick Disease, Type C
NCT03471143Phase 2Completed
- Sponsor
- Washington University School of Medicine
- Start
- Feb 2019
- Primary completion
- Oct 2024
- Modality
- Nanoparticles, Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of Trappsol Cyclo in Niemann-Pick disease type C (NPC), sponsored by Washington University School of Medicine. Primary endpoint: Plasma Levels of a Conjugated Bile Acid (5α-cholanic Acid-3β, 5α, 6β-triol N-(Carboxymethyl)-Amide) at 6 months. Started Feb 2019; status: completed.
02
Drugs
Trappsol Cyclo
03
Conditions
Niemann-Pick disease type C (NPC)
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