Phase I/II clinical trial of idursulfase beta for mucopolysaccharidosis type II

Phase I/II clinical trial of idursulfase beta for mucopolysaccharidosis type II - Brain Hunter Project 001

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT1091220350

Enrollment

Registered

2018-06-04

Start date

2016-07-29

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

Patients at the age between 6 months and 15 years with severe type mucopolysaccharidosis type II intravenously administered isursulfase at least for 24 weeks

Interventions

Intervention type:DRUG Name of intervention:BHP001 Dose form / Japanese Medical Device Nomenclature:INJECTION Route of administration / Site of application:OTHER Dose per administration:30 mg Dosing f

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: Patients diagnosed as mucopolysacchridosis type II based on biochemical or genetic anlysis

Exclusion criteria

Exclusion criteria: Patients received hematopoietic stem cell ransplantation previously

Design outcomes

Primary

Measure	Time frame
Heparan sulfate concentration in cerebrospinal fluid	—

Secondary

Measure	Time frame
Developmental age	—

Countries

Japan

Contacts

Public ContactTorayuki Okuyama

National Center for Child Health and Developmen

okuyama-t@ncchd.go.jp+81-3-3416-0181

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026