An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects

Conditions

Hunter Syndrome, Mucopolysaccharidosis type II

Brief summary

CSF HS (Total HS and HS disaccharide), dermatan sulfate (DS), and neurofilament light chain (NfL) concentrations, and opening pressure, Neuropsychological assessment, Others: Time course of QoL assessment, sleep disturbance, and global impression in Cohort A and Cohort B, Time course of Toileting Abilities Survey (TAS) in Cohort A and Cohort B

Detailed description

Liver and spleen volume, Shoulder range of motion, Six-minute walk test, Serum HS (Total), DS, and NfL concentrations, Urinary HS (Total) and DS concentrations, Pulmonary function (Cohort B only, if deemed feasible by principal investigator), Others: Time course of cardiac function (LVMI, IVST, and PWT) in Cohort A and Cohort B, Time course of T.O.V.A.® in Cohort B, Safety endpoints (Cohort A and Cohort B): Continuous assessment of AEs, including IARs, 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

Related papers

No related papers found yet.

Interventions

DRUGJR-141

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
CSF HS (Total HS and HS disaccharide), dermatan sulfate (DS), and neurofilament light chain (NfL) concentrations, and opening pressure, Neuropsychological assessment, Others: Time course of QoL assessment, sleep disturbance, and global impression in Cohort A and Cohort B, Time course of Toileting Abilities Survey (TAS) in Cohort A and Cohort B	—

Secondary

Measure	Time frame
Liver and spleen volume, Shoulder range of motion, Six-minute walk test, Serum HS (Total), DS, and NfL concentrations, Urinary HS (Total) and DS concentrations, Pulmonary function (Cohort B only, if deemed feasible by principal investigator), Others: Time course of cardiac function (LVMI, IVST, and PWT) in Cohort A and Cohort B, Time course of T.O.V.A.® in Cohort B, Safety endpoints (Cohort A and Cohort B): Continuous assessment of AEs, including IARs, 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	—

Measure

Time frame

Liver and spleen volume, Shoulder range of motion, Six-minute walk test, Serum HS (Total), DS, and NfL concentrations, Urinary HS (Total) and DS concentrations, Pulmonary function (Cohort B only, if deemed feasible by principal investigator), Others: Time course of cardiac function (LVMI, IVST, and PWT) in Cohort A and Cohort B, Time course of T.O.V.A.® in Cohort B, Safety endpoints (Cohort A and Cohort B): Continuous assessment of AEs, including IARs, 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

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Countries

France, Germany, Italy, Poland, Spain

Outcome results

None listed