mucopolysaccharidosis type II (MPS II)
Conditions
Interventions
[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
Sponsors
Ibaraki Ryo
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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.
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 | — |
Contacts
Public ContactRyo Ibaraki
JCR Pharmaceuticals Co., Ltd.
Outcome results
None listed