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

A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C - IT-HPBCD therapy for NPC (Long-term)

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000023517

Enrollment

Registered

2016-10-01

Start date

2016-10-01

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) 20mg/kg/dose, every 3-4 weeks, for three 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 12th 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