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

Mucopolysaccharidosis II Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04348136
• Other study ID #: JR-141-302
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: September 1, 2019
• Est. completion date: March 31, 2030

Study information

• Verified date: March 2024
• Source: JCR Pharmaceuticals Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 27
• Est. completion date: March 31, 2030
• Est. primary completion date: March 31, 2030
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis II

Intervention

Drug:
• JR-141
IV infusion, 2.0 mg/kg/week

Locations

Country	Name	City	State
Japan	Fukui Clinical site	Fukui
Japan	Fukuoka Clinical site	Fukuoka
Japan	Fukuoka Clinical site 2	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	Okinawa Clinical site	Okinawa
Japan	Osaka Clinical site	Osaka
Japan	Osaka Clinical site 2	Osaka
Japan	Osaka Clinical site 3	Osaka
Japan	Saitama Clinical site	Saitama
Japan	Shizuoka Clinical site	Shizuoka
Japan	Shizuoka Clinical site 2	Shizuoka
Japan	Tochigi Clinical site	Tochigi
Japan	Tokyo Clinical site	Tokyo
Japan	Tottori Clinical site	Tottori

Sponsors (1)

Lead Sponsor	Collaborator
JCR Pharmaceuticals Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Occurrence of adverse events		From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Primary	Occurrence of adverse reactions		From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Primary	Incidence of abnormal vital signs	Laboratory tests (hematology)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Incidence of abnormal vital signs	Laboratory tests (biochemistry)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Incidence of abnormal vital signs	Laboratory tests (iron-related tests)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Laboratory tests (urinalysis)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Vital signs (pulse rate)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Vital signs (body temperature)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Vital signs (blood pressure)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	12-lead electrocardiogram	The presence or absence of abnormal findings (if present, specific findings and whether or not they are reported as adverse events)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Antibody tests (anti-JR-141 antibodies)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	IAR		From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Primary	Time course of developmental assessment data (Kyoto Scale of Psychological Development 2001) from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of developmental assessment data (Vineland-II) from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of developmental assessment data (Bayley-III or KABC-II) from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of CSF substrate (HS and DS) concentrations from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of serum HS and DS concentrations from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of urinary HS concentration from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of urinary DS concentration from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of uronic acid concentration from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of liver volume (assessed by CT or MRI) from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of spleen volume (assessed by CT or MRI) from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of cardiac function (assessed by echocardiography) from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of 6-minute walk test distance from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Primary	Time course of joint range of motion from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years