A study to extend treatment with JR-141 after completion of Study JR-141-GS31
- Conditions
- Mucopolysaccharidosis II, Hunter SyndromeMedDRA version: 20.1Level: PTClassification code: 10056889Term: Mucopolysaccharidosis II Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2022-503142-41-00
- Lead Sponsor
- Jcr Pharmaceuticals Co. Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 71
A subject who participated in the Parent Study (JR-141-GS31) and completed the assessments at Week 105 in Cohort A or Week 53 in Cohort B, and in the opinion of the principal investigator has no safety concerns to enter this study., A subject from whom an IRB or IEC-approved written informed consent form (ICF) can be obtained, which is voluntarily signed. If the subject is aged under 18 years (aged under 16 years in the United Kingdom [UK]) at the time of enrollment or willingness to participate in the study cannot be confirmed due to MPS II related intellectual disability, the subject’s legally acceptable representative (e.g., his parents or guardians) may sign the ICF on behalf of the subject. Written informed assent should be obtained from the subject, wherever possible. The principal investigator or his/her designee will retain the original copy of each subject’s signed consent and assent (if applicable) document., Female subject of child bearing potential or male subject whose female partner is of child-bearing potential , i.e., fertile, following menarche and until becoming post- menopausal unless permanently sterile, agrees to use a medically accepted, highly effective method of contraception, from the time of signing the ICF., For subjects with hearing impairment requiring hearing aid(s), every effort will be made to encourage compliance with the use of functioning hearing aid(s). Subject or/and parent/legally acceptable representative agrees to wearing them during the study and on neurocognitive testing days.
A subject who has received gene therapy treatment at any point., Refusal to sign the ICF., A subject who is judged by the principal investigator or sub-investigator as being unable to undergo lumbar puncture, including those who have difficulties in attaining the required position for lumbar puncture due to joint contracture or those who are likely to experience breathing difficulties during the lumbar puncture process., A subject who changed treatment from JR-141 to idursulfase during the treatment period in the Parent Study (JR-141-GS31)., A subject who is unable to comply with the protocol (e.g., is unable to return for safety evaluations or is otherwise unlikely to complete the study) as determined by the principal investigator or sub-investigator., A subject who is judged by the principal investigator or sub-investigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity including to anesthesia or hypersensitivity to any component of JR-141., A subject who has a known or suspected local or general chronic infection or is at risk of abnormal bleeding due to medical conditions* or therapies the investigator classifies as causing the patient to be ineligible to participate in the study. * Medical Conditions: - Clinically significant multiple or severe drug allergies, intolerance to topical corticosteroids, or severe post-treatment hypersensitivity reactions (including, but not limited to, erythema multiforme major, linear immunoglobulin A [IgA], toxic epidermal necrolysis, and exfoliative dermatitis) - Evidence or history of significant active bleeding or coagulation disorder or use of non-steroidal anti-inflammatory drugs or other drugs that affect coagulation or platelet function within 14 days prior to lumbar catheter insertion - Allergy to lidocaine (Xylocaine®) or its derivatives, A subject who otherwise is judged by the principal investigator to be ineligible to participate in the study., [Only in France] (9) Persons deprived of their liberty by a judicial or administrative decision, according to article L. 1121-6 of the Public Health Code (Code dela santé publique, CSP) adults who are the subject of a measure of legal protection or unable to express their consent according to article L. 1121-8 of the CSP.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the long-term efficacy of JR-141 on CNS symptoms in subjects with MPS II;Secondary Objective: To evaluate the long-term efficacy of JR-141 on somatic symptoms in subjects with MPS II, To evaluate the long-term safety of JR-141 in subjects with MPS II;Primary end point(s): CSF HS and DS concentration and opening pressure, Neuropsychological assessment, Others: Time course of QoL assessment, sleep disturbance, and global impression at each time point in Cohort A and Cohort B, Time course of TAS at each time point in Cohort A and Cohort B, Continuous assessment of narrative subject/caregiver reports in Cohort A and Cohort B
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Liver and spleen volume;Secondary end point(s):Shoulder range of motion;Secondary end point(s):Six-minute walk test;Secondary end point(s):Serum HS and DS concentrations;Secondary end point(s):Urinary HS and DS concentrations;Secondary end point(s):Pulmonary function (Cohort B only, if deemed feasible by principal investigator);Secondary end point(s):Others: Time course of cardiac function (LVMI, IVST, and PWT) at each time point in Cohort A and Cohort B, Time course of T.O.V.A.® at each time point in Cohort B;Secondary end point(s):Safety endpoints (Cohort A and Cohort B): Continuous assessment of AEs, Periodical assessment of laboratory tests (hematology, blood biochemistry, iron-related tests, and urinalysis); vital signs (pulse rate, body temperature, respiratory rate, blood pressure); 12-lead ECG; anti-JR-141 antibodies, physical and neurological examination, weight, and height, and IAR