Unique ID issued by UMIN | UMIN000023517 |
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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 |
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)
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)
Japan |
Niemann-Pick disease type C
Endocrinology and Metabolism | Neurology | Pediatrics |
Others
NO
We use IT-HPBCD therapy for NPC patients with Miglustat resistance.
Safety,Efficacy
NPC-ADL score after 12th administration of HPBCD
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
1
Treatment
Medicine |
2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three years
4 | years-old | <= |
6 | years-old | > |
Male and Female
1)Genetically proven NPC
2)Neurological involvement
3)Miglustat resistance
1)CNS or systemic infection
2)Without neurological involvement
1
1st name | |
Middle name | |
Last name | Takafumi Sakakibara |
Nara Medical University
Department of Pediatrics
840 Shijo-Cho, Kashihara, Nara, Japan
0744-22-3051
kumasan@naramed-u.ac.jp
1st name | |
Middle name | |
Last name | Takafumi Sakakibara |
Nara Medical University
Department of Pediatrics
840 Shijo-Cho, Kashihara, Nara, Japan
0744-22-3051
kumasan@naramed-u.ac.jp
Nara Medical University
Nara Medical University
Other
NO
2016 | Year | 10 | Month | 01 | Day |
Unpublished
Preinitiation
2016 | Year | 10 | Month | 01 | Day |
2016 | Year | 10 | Month | 01 | Day |
2016 | Year | 08 | Month | 06 | Day |
2016 | Year | 08 | Month | 06 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027090
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