A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick disease type C

A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick disease type C - IT-HPBCD therapy for NPC

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000023094

Enrollment

Registered

2016-07-10

Start date

2014-08-01

Completion date

Unknown

Last updated

2026-06-29

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) 5-20mg/kg, every 4 weeks, for 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) CNS or systemic infection 2) Without neurological involvement

Design outcomes

Primary

Measure	Time frame
NPC-ADL score after 4th administration of HPBCD	—

Countries

Japan

Contacts

Public ContactTakafumi Sakakibara

Nara Medical University Department of Pediatrics

kumasan@naramed-u.ac.jp0744-22-3051

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026