Health Condition 1: E761- Mucopolysaccharidosis, type II
Conditions
Interventions
Intervention1: Idursulfase (r-DNA origin)-2 mg/ml (Elaprase ™ ) and Water for Injection: 1 vial contains Active Ingredient: Each vial contains 6 mg of Idursulfase. Each ml contains 2 mg of Idursulfa
Sponsors
Shire Biotech India Pvt Ltd
Eligibility
Inclusion criteria
Inclusion criteria: 1. 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. 2. In the opinion of the investigator, the subject or the subject’s parents/guardians are capable of understanding and complying with protocol requirements. 3. The subject or, when applicable, the subject’s parents/guardians/LAR signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures. If the subject participating in this study is =7 and 4. A male subject who is nonsterilized and sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (eg, condom with or without spermicide) from signing of informed consent throughout the duration of the study. The female partner of a male subject should also be advised to use a highly effective/effective method of contraception. 5. A female subject of childbearing potential who is sexually active with a nonsterilized male partner agrees to use a highly effective method of contraception from signing of informed consent throughout the duration of the study.
Exclusion criteria
Exclusion criteria: 1. Subject has received hematopoietic stem cell transplantation or a bone marrow transplant at any time. 2. Subject is unable to comply with the protocol, eg, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator. 3. 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. 4. The subject has a chronic kidney disease with estimated Glomerular Filtration rate less than 15 mL/min/1.73 m2 and/or is on dialysis. 5. The subject is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in conduct of this study (eg, spouse, parent, child, sibling) or may consent under duress. 6. The subject has a history of hypersensitivity or allergies to related compounds including any associated excipients. 7. If female, the subject is pregnant or lactating or intending to become pregnant before participating in this study, during the study; or intending to donate ova during such time period. 8. If male, the subject intends to donate sperm during the course of this study. 9. 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 |
|---|---|
| Incidence of adverse events (AEs), serious AEs (SAEs), treatment emergent AEs, treatment emergent SAEs, adverse drug reactions (ADRs), discontinuations due to AEs, infusion-related reactions, and death.Timepoint: Baseline to 53 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in 6 Minute Walk Test (6MWT) as a measure of physical functional capacityTimepoint: Weeks 27 and 53;Change from baseline in Cardiac Left Ventricular Mass Index (LVMI) and Ejection FractionTimepoint: Weeks 27 and 53;Change from baseline in Global Joint Range of Motion (JROM) scoreTimepoint: Weeks 27 and 53;Change from baseline in liver volumeTimepoint: Weeks 27 and 53;Change from baseline in normalized urine GAG (uGAG) levelsTimepoint: Weeks 14, 27, 40, and 53;Change from baseline in percentage Forced Vital Capacity (%FVC) as a measure of respiratory function.Timepoint: Weeks 27 and 53;Change from baseline in spleen volumeTimepoint: Weeks 27 and 53;Change from baseline in the health-related quality of life (HRQoL) based on Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) (shortened version) in all subjects and Childhood Health Assessment Questionnaire (CHAQ) in subjects between age of =1 year to =18 yearsTimepoint: Weeks 27 and 53;Changes from baseline in anthropometric parameters including height in subjects less than 18 years at baseline and weight in all subjectsTimepoint: Weeks 27 and 53 | — |
Countries
India
Contacts
Public ContactRuchi Sogarwal
Shire Biotech India Private Limited
Outcome results
None listed