JRCT ID: jRCT2031220251
Registered date:07/08/2022
Post-marketing surveillance for Hunterase ICV injection 15 mg
Basic Information
Recruitment status | Recruiting |
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Health condition(s) or Problem(s) studied | Mucopolysaccharidosis type II |
Date of first enrollment | 06/05/2021 |
Target sample size | 30 |
Countries of recruitment | |
Study type | Observational |
Intervention(s) |
Outcome(s)
Primary Outcome | 1) Patient background 2) Presence or absence of anaphylaxis 3) Efficacy primary endpoint (1) Heparan sulfate (HS) concentration in cerebrospinal fluid (CSF) (2) Developmental age (new edition K-type developmental age test) Collect information on the effectiveness of the same evaluation items used in the BHP001 test used in the manufacturing and marketing approval application. 4) Antibody test 5) Clinical examination 6) Concomitant therapy 7) Clinical findings (including vital signs and physical examination) 8) Adverse events (1) Adverse events associated with implantable cerebrospinal fluid reservoir mounting procedure (medical device-related complications) (2) Adverse events associated with this medicine 9) Observation frequency (1) Developmental age is measured at the start, at 52 weeks, and every 48 weeks thereafter. If possible, take measurements every 24 weeks. (2) Patient background is measured at the start, and other items are measured at the start, 12 weeks, 24 weeks, 36 weeks, 52 weeks, and every 12 weeks thereafter. |
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Secondary Outcome |
Key inclusion & exclusion criteria
Age minimum | Not applicable |
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Age maximum | Not applicable |
Gender | Male |
Include criteria | All the patients with mucopolysaccharidosis type II who administered Hunterase ICV injection 15 mg |
Exclude criteria | none |
Related Information
Primary Sponsor | Yanagi Komei |
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Secondary Sponsor | |
Source(s) of Monetary Support | |
Secondary ID(s) |
Contact
Public contact | |
Name | Hirotoshi Nakashima |
Address | 6F, Nihonbashi Kimura Bldg., 1-16-3, Nihonbashi, Chuo-ku, Tokyo Tokyo Japan 103-0027 |
Telephone | +81-3-6869-0663 |
hirotoshi.nakashima@clinigen.co.jp | |
Affiliation | Clinigen K.K. |
Scientific contact | |
Name | Komei Yanagi |
Address | 6F, Nihonbashi Kimura Bldg., 1-16-3, Nihonbashi, Chuo-ku, Tokyo Tokyo Japan 103-0027 |
Telephone | +81-3-6869-0663 |
komei.yanagi@clinigen.co.jp | |
Affiliation | Clinigen K.K. |