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Lon-term study of idursulfase beta in patients with mucopolysaccharidosis type II (CKK001)

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

Yanagi Komei

Clinigen K.K.

6F, Nihonbashi Kimura Bldg., 1-16-3, Nihonbashi, Chuo-ku, Tokyo

komei.yanagi@clinigen.co.jp

Yanagi Komei

Clinigen K.K.

6F, Nihonbashi Kimura Bldg., 1-16-3, Nihonbashi, Chuo-ku, Tokyo

+81-3-6869-0663

komei.yanagi@clinigen.co.jp

Complete

Mar. 11, 2020

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

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)

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

Male

Mucopolysaccharidosis type II

[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.

D016532

D007276

[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

+81-3-3416-0181

Jan. 23, 2020

No

none