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Intrathecal cyclodextrin study for Niemann-Pick disease type C
jRCTs071190008Recruiting
- Sponsor
- Kaken Pharmaceutical Co., Ltd.
- Start
- Jul 2019
- Modality
- Small Molecules, Nanoparticles → Drug Delivery Systems
- Countries
- 1
01
Overview
Open-label, single-arm trial of Trappsol Cyclo in Niemann-Pick disease type C (NPC), sponsored by Kaken Pharmaceutical Co., Ltd. Primary endpoint: Change ratio of modified disability scale for Niemann-Pick disease type C between before and after treatment. Started Jul 2019; status: recruiting.
02
Drugs
Trappsol Cyclo
03
Conditions
Niemann-Pick disease type C (NPC)
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