Clinical trial

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

Name

JR-141-302

Description

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

Trial arms

Trial start

2019-09-01

Estimated PCD

2030-03-31

Trial end

2030-03-31

Status

Active (not recruiting)

Phase

Early phase I

Treatment

JR-141

IV infusion, 2.0 mg/kg/week

Arms:

JR-141

Size

Primary endpoint

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

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

Incidence of abnormal vital signs

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

Incidence of abnormal vital signs

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

Incidence of abnormal vital signs

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

Laboratory tests (urinalysis)

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

Vital signs (pulse rate)

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

Vital signs (body temperature)

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

Vital signs (blood pressure)

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

12-lead electrocardiogram

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

Antibody tests (anti-JR-141 antibodies)

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

IAR

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2', 'PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 27, 'type': 'ACTUAL'}}

Updated at

2024-03-15

1 organization

1 product

1 indication

Organization

JCR Pharmaceuticals

Product

JR-141

Indication

MPS II