UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000023517 |
|---|---|
| Receipt number | R000027090 |
| Scientific Title | A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C |
| Date of disclosure of the study information | 2016/10/01 |
| Last modified on | 2016/08/06 10:45:04 |
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Basic information
Public title
A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C
Acronym
IT-HPBCD therapy for NPC (Long-term)
Scientific Title
A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C
Scientific Title:Acronym
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
| 1st name | |
| Middle name | |
| Last name | Takafumi Sakakibara |
Organization
Nara Medical University
Division name
Department of Pediatrics
Zip code
Address
840 Shijo-Cho, Kashihara, Nara, Japan
TEL
0744-22-3051
kumasan@naramed-u.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Takafumi Sakakibara |
Organization
Nara Medical University
Division name
Department of Pediatrics
Zip code
Address
840 Shijo-Cho, Kashihara, Nara, Japan
TEL
0744-22-3051
Homepage URL
kumasan@naramed-u.ac.jp
Sponsor or person
Institute
Nara Medical University
Institute
Department
Personal name
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)
IRB Contact (For public release)
Organization
Address
Tel
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 |
Related information
URL releasing protocol
Publication of results
Unpublished
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
Preinitiation
Date of protocol fixation
| 2016 | Year | 10 | Month | 01 | Day |
Date of IRB
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
Other
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027090
