UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000020032 |
|---|---|
| Receipt number | R000023134 |
| Scientific Title | Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC) |
| Date of disclosure of the study information | 2015/12/02 |
| Last modified on | 2022/06/08 18:34:48 |
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Basic information
Public title
Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC)
Acronym
IT-HPBCD therapy in adult-onset NPC
Scientific Title
Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC)
Scientific Title:Acronym
IT-HPBCD therapy in adult-onset NPC
Region
| Japan |
Condition
Condition
Niemann-Pick disease type C
Classification by specialty
| Endocrinology and Metabolism | Neurology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
We used IT-HPBCD therapy for the adult-onset NPC patients with Miglustat resistance.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Pragmatic
Developmental phase
Not applicable
Assessment
Primary outcomes
NPC-ADL score one year after starting intervention
Key secondary outcomes
hearing loss, pulmonary lesion, serum oxysterol, urinary bile acids
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) 100-400mg, every 4 week, two 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
| 20 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) Genetically proven NPC
2) Neurological involvement
3) Miglustat resistance
Key exclusion criteria
1) Infantile and child NPC
2) CNS or systemic infection
3) Without neurological involvement
Target sample size
2
Research contact person
Name of lead principal investigator
| 1st name | Yoshio |
| Middle name | |
| Last name | Sakiyama |
Organization
Jichi Medical University, Saitama Medical Center
Division name
Department of Neurology
Zip code
330-8503
Address
Amanuma-cho 1-847, Omiya-ku, Saitama City 330-8503 Japan
TEL
048-647-2111
sakiyamay@jichi.ac.jp
Public contact
Name of contact person
| 1st name | Yoshio |
| Middle name | |
| Last name | Sakiyama |
Organization
Jichi Medical University, Saitama Medical Center
Division name
Department of Neurology
Zip code
330-8503
Address
Amanuma-cho 1-847, Omiya-ku, Saitama City 330-8503 Japan
TEL
048-647-2111
Homepage URL
sakiyamay@jichi.ac.jp
Sponsor or person
Institute
Jichi Medical University, Saitama Medical Center
Institute
Department
Personal name
Funding Source
Organization
none
Organization
Division
Category of Funding Organization
Other
Nationality of Funding Organization
Other related organizations
Co-sponsor
Saga University
Kumamoto University
Tohoku University
Institute for Neurological Disorders
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Jichi Medical University, Saitama Medical Center, General Affairs Division
Address
Amanuma-cho 1-847, Omiya-ku, Saitama City 330-8503 Japan
Tel
048-647-2111(ext.2341)
inahara@jichi.ac.jp
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
自治医科大学附属さいたま医療センター (埼玉県）
Other administrative information
Date of disclosure of the study information
| 2015 | Year | 12 | Month | 02 | Day |
Related information
URL releasing protocol
Publication of results
Partially published
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
No longer recruiting
Date of protocol fixation
| 2014 | Year | 12 | Month | 19 | Day |
Date of IRB
Anticipated trial start date
| 2014 | Year | 12 | Month | 19 | Day |
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2015 | Year | 12 | Month | 02 | Day |
Last modified on
| 2022 | Year | 06 | Month | 08 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000023134
