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

JRCT ID: jRCT2031210126

Registered date:31/05/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: mucopolysaccharidosis type II (MPS II)
• Date of first enrollment: 27/07/2021
• Target sample size: 20
• Study type: Interventional

Intervention(s)

[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.

Outcome(s)

Primary Outcome

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

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both

Include 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.

Exclude 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

Related Information

• Primary Sponsor: Yamamoto Tatsuyoshi