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Post-marketing Clinical Study of IZCARGO in Patients with Mucopolysaccharidosis Type II

Post-marketing Clinical Study of IZCARGO in Patients with Mucopolysaccharidosis Type II

Ibaraki Ryo

JCR Pharmaceuticals Co., Ltd.

11-18 Kusunoki-cho Ashiya-City, Hyogo

clinical_development@jp.jcrpharm.com

Ibaraki Ryo

JCR Pharmaceuticals Co., Ltd.

11-18 Kusunoki-cho Ashiya-City, Hyogo

+81-797-32-8582

clinical_development@jp.jcrpharm.com

Recruiting

May. 31, 2021

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1) A patient from whom written informed consent can be obtained. Or, a patient from whom written informed consent can be obtained from the patient's legally acceptable representative if the patient is a minor at the time of informed consent or whose willingness to participate in the study cannot be confirmed intellectual disability (however, written informed consent should be obtained from the patient, wherever possible)
2) A patient diagnosed with MPS II comprehensively based on decreased activity of iduronate-2-sulfatase (IDS) in leucocytes, plasma, or cultured skin fibroblasts, genetic analysis, etc.
3) Male patient whose partner is of child-bearing potential or female patient who agrees to use a medically accepted, highly effective method of contraception.

1) A patient who has received IZCARGO treatment
2) A patient with a history of hematopoietic stem cell transplantation, unless enzyme replacement therapy is required after hematopoietic stem cell transplantation
3) A patient who has received gene therapy
4) A patient who has received intracerebroventricular injection of IDS
5) A patient who has developed serious drug allergy or hypersensitivity that, in the opinion of the post-marketing clinical study investigator or post-marketing clinical study subinvestigator, is inappropriate for participation in the study
6) A patient who has received investigational product within 4 months before enrollment in the study
7) A patient who, in the opinion of the post-marketing clinical study investigator or post-marketing clinical study subinvestigator, is ineligible to participate in the study

Both

mucopolysaccharidosis type II (MPS II)

[Post-marketing clinical study drug]
Name
- Post-marketing clinical study drug (code): IZCARGO for I.V. infusion 10 mg (test drug code: JR-141)
- Nonproprietary name
International nonproprietary name (INN): pabinafusp alfa (r-INN List 82, WHO Drug Information, Vol. 33. No. 3, 2019)
Japanese accepted names for pharmaceuticals (JAN)
Registered number: 301-4-B3
Japanese name: pabinafusp alfa (genetical recombination)
English name: Pabinafusp Alfa (Genetical Recombination)
Dose and regimen: Subjects will receive 2.0 mg of pabinafusp alfa (genetical recombination) per kilogram of body weight once a week as an intravenous infusion.

1) Time course of developmental assessment (KSPD, Vineland-II, and Bayley-III/KABC-II)
2) Time course of T.O.V.A.
3) Time course of HS and DS concentrations in CSF
4) Time course of serum HS and DS concentrations
5) Time course of urinary HS and DS concentrations
6) Time course of liver and spleen volumes (CT or MRI)
7) Time course of walk distance at the 6-minute walk test

+81-3-5494-7297

May. 20, 2021

No

none