Clinical trial NCT04348136: An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Official title: An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Mucopolysaccharidosis II
Lead sponsor: JCR Pharmaceuticals Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2020-04-16
Enrollment: 27
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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.

Primary outcomes:
measure: Occurrence of adverse events
timeFrame: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
measure: Occurrence of adverse reactions
timeFrame: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
measure: Incidence of abnormal vital signs
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Incidence of abnormal vital signs
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Incidence of abnormal vital signs
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Laboratory tests (urinalysis)
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Vital signs (pulse rate)
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Vital signs (body temperature)
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Vital signs (blood pressure)
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: 12-lead electrocardiogram
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Antibody tests (anti-JR-141 antibodies)
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: IAR
timeFrame: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
measure: Time course of developmental assessment data (Kyoto Scale of Psychological Development 2001) from initial dosing in the preceding study
timeFrame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of developmental assessment data (Vineland-II) from initial dosing in the preceding study
timeFrame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of developmental assessment data (Bayley-III or KABC-II) from initial dosing in the preceding study
timeFrame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of CSF substrate (HS and DS) concentrations from initial dosing in the preceding study
timeFrame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of serum HS and DS concentrations from initial dosing in the preceding study
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of urinary HS concentration from initial dosing in the preceding study
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of urinary DS concentration from initial dosing in the preceding study
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of uronic acid concentration from initial dosing in the preceding study
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of liver volume (assessed by CT or MRI) from initial dosing in the preceding study
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of spleen volume (assessed by CT or MRI) from initial dosing in the preceding study
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of cardiac function (assessed by echocardiography) from initial dosing in the preceding study
timeFrame: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of 6-minute walk test distance from initial dosing in the preceding study
timeFrame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
measure: Time course of joint range of motion from initial dosing in the preceding study
timeFrame: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Secondary outcomes:
Other outcomes:
Locations:
city: Fukui
country: Japan
facility: Fukui Clinical site
city: Fukuoka
country: Japan
facility: Fukuoka Clinical site 2
city: Fukuoka
country: Japan
facility: Fukuoka Clinical site
city: Gifu
country: Japan
facility: Gifu Clinical site
city: Hiroshima
country: Japan
facility: Hiroshima Prefectural Hospital
city: Hokkaido
country: Japan
facility: Hokkaido Clinical site
city: Kanagawa
country: Japan
facility: Kananagawa Ckinical site
city: Kumamoto
country: Japan
facility: Kumamoto Clinical site
city: Okayama
country: Japan
facility: Okayama Clinical site
city: Okayama
country: Japan
facility: Okayama Clinical site 2
city: Okinawa
country: Japan
facility: Okinawa Clinical site
city: Osaka
country: Japan
facility: Osaka Clinical site 3
city: Osaka
country: Japan
facility: Osaka Clinical site 2
city: Osaka
country: Japan
facility: Osaka Clinical site
city: Saitama
country: Japan
facility: Saitama Clinical site
city: Shizuoka
country: Japan
facility: Shizuoka Clinical site
city: Shizuoka
country: Japan
facility: Shizuoka Clinical site 2
city: Tochigi
country: Japan
facility: Tochigi Clinical site
city: Tokyo
country: Japan
facility: Tokyo Clinical site
city: Tottori
country: Japan
facility: Tottori Clinical site
Study officials:
References: