UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C
Title of the study (Brief title)	IT-HPBCD therapy for NPC (Long-term)
Region	Japan

Condition
Condition	Niemann-Pick disease type C
Classification by specialty	Endocrinology and Metabolism Neurology Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	We use IT-HPBCD therapy for NPC patients with Miglustat resistance.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	NPC-ADL score after 12th administration of HPBCD
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three years
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	4 years-old <=
Age-upper limit	6 years-old >
Gender	Male and Female
Key inclusion criteria	1)Genetically proven NPC 2)Neurological involvement 3)Miglustat resistance
Key exclusion criteria	1)CNS or systemic infection 2)Without neurological involvement
Target sample size	1

Research contact person
Name of lead principal investigator	Takafumi Sakakibara
Organization	Nara Medical University
Division name	Department of Pediatrics
Address	840 Shijo-Cho, Kashihara, Nara, Japan
TEL	0744-22-3051
Email	kumasan@naramed-u.ac.jp

Public contact
Name of contact person	Takafumi Sakakibara
Organization	Nara Medical University
Division name	Department of Pediatrics
Address	840 Shijo-Cho, Kashihara, Nara, Japan
TEL	0744-22-3051
Homepage URL
Email	kumasan@naramed-u.ac.jp

Sponsor
Institute	Nara Medical University
Institute
Department

Funding Source
Organization	Nara Medical University
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2016 Year 10 Month 01 Day

Progress
Recruitment status	Preinitiation
Date of protocol fixation	2016 Year 10 Month 01 Day
Anticipated trial start date	2016 Year 10 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2016 Year 08 Month 06 Day
Last modified on	2016 Year 08 Month 06 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027090