Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC)

Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC) - IT-HPBCD therapy in adult-onset NPC

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000020032

Enrollment

Registered

2015-12-02

Start date

2014-12-19

Completion date

Unknown

Last updated

2026-09-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Niemann-Pick disease type C

Interventions

2-hydroxypropyl-beta-cyclodextrin (HPBCD) 100-400mg, every 4 week, two years

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) Genetically proven NPC 2) Neurological involvement 3) Miglustat resistance

Exclusion criteria

Exclusion criteria: 1) Infantile and child NPC 2) CNS or systemic infection 3) Without neurological involvement

Design outcomes

Primary

Measure	Time frame
NPC-ADL score one year after starting intervention	—

Secondary

Measure	Time frame
hearing loss, pulmonary lesion, serum oxysterol, urinary bile acids	—

Countries

Japan

Contacts

Public ContactYuka Hayashi

Jichi Medical University, Saitama Medical Center Department of Neurology

yukahayashi@jichi.ac.jp048-647-2111

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Sep 19, 2026