Lon-term study of idursulfase beta in patients with mucopolysaccharidosis type II

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-jRCT2031220250
• Date of registration: 07/08/2022
• Prospective Registration: No
• Primary sponsor: Yanagi Komei
• Public title: Lon-term study of idursulfase beta in patients with mucopolysaccharidosis type II
• Scientific title: Lon-term study of idursulfase beta in patients with mucopolysaccharidosis type II - CKK001
• Date of first enrolment: 11/03/2020
• Target sample size: 6
• Recruitment status: Complete
• URL: https://jrct.niph.go.jp/latest-detail/jRCT2031220250
• Study type: Interventional
• Study design: single arm study, open(masking not used), uncontrolled control, single assignment, treatment purpose
• Phase: 3

Contacts

• Name: Komei Yanagi
• Affiliation: Clinigen K.K.

• Name: Komei Yanagi
• Affiliation: Clinigen K.K.

Key inclusion & exclusion criteria

Inclusion criteria: Patients who met all of the following criteria 1) to 4) or who met 5) were included in this study.
1) Patients with confirmed diagnosis of severe mucopolysaccharidosis type II (type C or D)
The diagnosis of severe mucopolysaccharidosis type II is confirmed when both of the following are satisfied.
- The activity of isulonate 2-sulfatase in leukocytes is low or below the limit of quantification, and the amount of uronic acid excreted in urine exceeds the standard value.
- Has a severe type of gene mutation that is thought to reduce the activity of isulonate 2-sulfatase.
2) Male patients aged 6 months to under 15 years
3) Patients who have never received hematopoietic stem cell transplantation in the past
4) Patients who received idursulfase intravenously for 12 weeks or more before the start of the study (the total number of doses for the 12 weeks before the start of the study was 10 or more, and it was confirmed that there was no problem with tolerability)
5) atients who have been treated with idursulfase beta in the BHP001 study and wish to continue treatment after approval (patients who are judged by the principal investigator or co-investigator to be beneficial to continue treatment)
Exclusion criteria: Patients who met any of the following, even if they met the selection criteria, were not included in this study.
1) Patients whose urinary uronic acid level is 50 times or more the upper limit of the age-specific standard
2) Patients who underwent ventricular/intraperitoneal shunt
3) Patients with end-stage organ dysfunction and other serious illnesses
4) Patients with malignant neoplasms
5) Patients with a history of anaphylactic shock in the components of the investigational drug
6) Other patients who are judged to be ineligible by the principal investigator or co-investigator

Age minimum: >= 6month old
Age maximum: < 15age old
Gender: Male

Health Condition(s) or Problem(s) studied

D016532

Mucopolysaccharidosis type II

Intervention(s)

D007276

[Investigative drug, dose, and administration method]
- Investigative drug: Vial formulation filled with idursulfase beta for intracerebroventricular administration
(Concentration 15 +/- 1.5 mg/mL, liquid volume 1.25 mL)
- Dose and administration method:
An implantable cerebrospinal fluid reservoir is attached to the subject's head, and idursulfase beta 30 mg is administered to the ventricles once every 4 weeks.

Primary Outcome(s)

[Exploratory effectiveness evaluation items]
1) Heparan sulfate concentration in cerebrospinal fluid
2) Developmental age
[Safety evaluation items]
1) Adverse events
2) Clinical tests and vital signs

Ethics review

Status: Approval
Approval date: 23/01/2020
Contact:

[e-mail]

National Center for Child Health and Development Institutional Review Board

[e-mail]