An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Phase 3Active Not RecruitingNCT04348136

JCR Pharmaceuticals Co., Ltd.27 enrolled

Overview

Multicenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Mucopolysaccharidosis II

Interventions

JR-141DRUG

IV infusion, 2.0 mg/kg/week

Eligibility

Sex: MALE

Medical Language ↔ Plain English

Inclusion Criteria: 1. A patient who participated in the preceding Study JR-141-301 and completed the Week 52 visit, and has no safety concerns to enter this study in the opinion of the investigator or subinvestigator. 2. A patient capable of providing written informed consent in person (However, this is not required for patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified.) 3. For patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified, written consent must be obtained from the legally acceptable representative. (Wherever possible, written consent of the patient should be obtained.) 4. Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception. Exclusion Criteria: 1. A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator. 2. A patient with a history of serious drug allergy or hypersensitivity that precludes participation in this study in the opinion of the investigator or subinvestigator. 3. A patient judged to be ineligible by the investigator or subinvestigator for other reasons.

Locations (20)

Fukui Clinical site

Fukui, Japan

Fukuoka Clinical site 2

Fukuoka, Japan

Outcomes

Primary Outcomes

Occurrence of adverse events

Time frame: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years

Occurrence of adverse reactions

Time frame: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years

Incidence of abnormal vital signs

Laboratory tests (hematology)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Incidence of abnormal vital signs

Laboratory tests (biochemistry)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Incidence of abnormal vital signs

Laboratory tests (iron-related tests)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Laboratory tests (urinalysis)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Vital signs (pulse rate)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Vital signs (body temperature)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Vital signs (blood pressure)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Data from ClinicalTrials.gov

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Fukuoka Clinical site

Fukuoka, Japan

Gifu Clinical site

Gifu, Japan

Hiroshima Prefectural Hospital

Hiroshima, Japan

Hokkaido Clinical site

Hokkaido, Japan

Kananagawa Ckinical site

Kanagawa, Japan

Kumamoto Clinical site

Kumamoto, Japan

Okayama Clinical site

Okayama, Japan

Okayama Clinical site 2

Okayama, Japan

...and 10 more locations

12-lead electrocardiogram

The presence or absence of abnormal findings (if present, specific findings and whether or not they are reported as adverse events)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Antibody tests (anti-JR-141 antibodies)

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

IAR

Time frame: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years

Time course of developmental assessment data (Kyoto Scale of Psychological Development 2001) from initial dosing in the preceding study

Time frame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of developmental assessment data (Vineland-II) from initial dosing in the preceding study

Time frame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of developmental assessment data (Bayley-III or KABC-II) from initial dosing in the preceding study

Time frame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of CSF substrate (HS and DS) concentrations from initial dosing in the preceding study

Time frame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of serum HS and DS concentrations from initial dosing in the preceding study

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of urinary HS concentration from initial dosing in the preceding study

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of urinary DS concentration from initial dosing in the preceding study

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of uronic acid concentration from initial dosing in the preceding study

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of liver volume (assessed by CT or MRI) from initial dosing in the preceding study

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of spleen volume (assessed by CT or MRI) from initial dosing in the preceding study

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of cardiac function (assessed by echocardiography) from initial dosing in the preceding study

Time frame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of 6-minute walk test distance from initial dosing in the preceding study

Time frame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Time course of joint range of motion from initial dosing in the preceding study

Time frame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years