Hunter syndrome (Mucopolysaccharidosis II, [MPS II])
Conditions
Interventions
Trade Name: Elaprase
Pharmaceutical Form: Concentrate for solution for injection/infusion
Sponsors
Takeda Biopharmaceuticals India Pvt. Ltd.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Male or female Elaprase naïve subjects (and who are not part of any other program at the time of study enrollment and during the study period) of any age with confirmed diagnosis of Hunter syndrome based on the following documented biochemical and genetic criteria: - Documented deficiency in iduronate 2-sulfatase (IDS [12S]) enzyme activity of less than or equal to 10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory). - A normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory). - The patient has a documented mutation in the IDS gene. Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Subject has received hematopoietic stem cell transplantation or a bone marrow transplant at any time. - Subject is suffering from any comorbid conditions (including hepatic impairment, acute or chronic) or having any other clinical observation or history during the screening examination, which would interfere with the objectives of the study as per investigators judgement. - Subject has a chronic kidney disease with estimated Glomerular Filtration rate less than 15 mL/min/1.73 m2 and/or is on dialysis. - Subject has participated in any other clinical study or received any investigational compound or non-investigational idursulfase beta within the past 30 days before informed consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of Elaprase in pediatric and adult subjects with Hunter syndrome during 53 weeks of study duration.;Secondary Objective: To evaluate the efficacy of Elaprase in pediatric and adult subjects with Hunter syndrome during 53 weeks of study duration.;Primary end point(s): Incidence of AEs, serious AEs (SAEs), treatment emergent AEs, treatment emergent SAEs, adverse drug reactions (ADRs), discontinuation due to AEs, infusion-related reactions, and death.;Timepoint(s) of evaluation of this end point: From start of the study drug administration up to EOS (Week 53) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Change from baseline in percentage Forced Vital Capacity (%FVC) as a measure of respiratory function at Weeks 27 and 53. - Change from baseline in 6 Minute Walk Test (6MWT) as a measure of physical functional capacity at Weeks 27 and 53. - Change from baseline in Cardiac Left Ventricular Mass Index (LVMI) and Ejection Fraction at Weeks 27 and 53. - Change from baseline in liver volume at Weeks 27 and 53. - Change from baseline in spleen volume at Weeks 27 and 53. - Change from baseline in uGAG levels at Weeks 14, 27, 40, and 53. - Change from baseline in Global Joint Range of Motion (JROM) score at Weeks 27 and 53 - Changes from baseline in anthropometric parameters including height in subjects <18 years at baseline and weight in all subjects at Weeks 27 and 53. - Changes from baseline in the health-related quality of life based on Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) (shortened version) and Childhood Health Assessment Questionnaire (CHAQ) in subjects between age of =1 year to =18 years at Weeks 27 and 53.;Timepoint(s) of evaluation of this end point: Baseline, Weeks 27 and 53 | — |
Countries
India
Contacts
Public ContactStudy Director
Takeda
Outcome results
None listed