Post-marketing surveillance for Hunterase ICV injection 15 mg

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-jRCT2031220251

Enrollment

Registered

2022-08-07

Start date

2021-05-06

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mucopolysaccharidosis type II

Interventions

None listed

Eligibility

Sex/Gender

Male

Inclusion criteria

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

Exclusion criteria

Exclusion criteria: none

Design outcomes

Primary

Measure	Time frame
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.	—

Measure

Time frame

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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Contacts

Public ContactPMS Information Contact for

Clinigen K.K.

Takashi.Namba@clinigen.co.jp+81-3-6869-0663

Outcome results