臨床研究等提出・公開システム

Post-marketing surveillance for Hunterase ICV injection 15 mg

Post-marketing surveillance for Hunterase ICV injection 15 mg

Contact for PMS Information

Clinigen K.K.

Muromachi Chibagin Mitsui Bldg 10F 1-5-5 Nihonbashi Muromachi Chuo-ku, Tokyo

Takashi.Namba@clinigen.co.jp

Contact for PMS Information

Clinigen K.K.

Muromachi Chibagin Mitsui Bldg 10F 1-5-5 Nihonbashi Muromachi Chuo-ku, Tokyo

+81-3-6869-0663

Takashi.Namba@clinigen.co.jp

Recruiting

May. 06, 2021

Observational

All the patients with mucopolysaccharidosis type II who administered Hunterase ICV injection 15 mg

none

Male

Mucopolysaccharidosis type II

D016532

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.

Yes

Mainly individual efficacy will be evaluated by Hunterase Study Group (Chairperson: Dr. Torayuki Okuyama (Saitama Medical University)), and effectiveness of Hunterase ICV injection 15 mg will be assessed (one of the items for approval condition).

none