Clinical trial NCT05058391: A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
Official title: A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hunter Syndrome
Keywords: Mucopolysaccharidosis II
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2021-09-27
Enrollment: 5
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The main aim of this study is to learn more about the safety profile of Elaprase in Indian children and adults with hunter syndrome.

Participants will receive Elaprase once per week over a 3-hour period which can be reduced to 1 hour as determined by the study doctor. Participants will need to visit the clinic weekly during the duration of the study.


Eligibility criteria: Inclusion Criteria:

* Male or female Elaprase naive participants (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 percent (%) 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 participant has a documented mutation in the IDS gene.
* In the opinion of the investigator, the participant or the participant's parents/guardians are capable of understanding and complying with protocol requirements.
* The participant or, when applicable, the participant's parents/guardians/legal authorized representative (LAR) signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures. If the participant participating in this study is greater than or equal to (\>=) 7 years and less than (\<) 18 years of age signs and dates an assent form.
* A male participant who is nonsterilized and sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (example, condom with or without spermicide) from signing of informed consent throughout the duration of the study. The female partner of a male participant should also be advised to use a highly effective/effective method of contraception.
* A female participant 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:

* Participant has received hematopoietic stem cell transplant (HSCT) or a bone marrow transplant at any time.
* Participant is unable to comply with the protocol, example, 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.
* Participant 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.
* The participant has a chronic kidney disease with estimated Glomerular Filtration rate less than 15 milliliter per minute per 1.73 meter square (mL/min/1.73 m\^2) and/or is on dialysis.
* The participant 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 (example, spouse, parent, child, sibling) or may consent under duress.
* The participant has a history of hypersensitivity or allergies to related compounds including any associated excipients.
* If female, the participant 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.
* If male, the participant intends to donate sperm during the course of this study.
* The participant has participated in another clinical study or received any investigational compound or non-investigational idursulfase beta within the past 30 days before informed consent.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death
timeFrame: From start of the study drug administration up to Week 53
measure: Number of Participants With Adverse Drug Reactions (ADRs)
timeFrame: From start of the study drug administration up to Week 53
measure: Number of Participants With Infusion-related Reactions (IRRs)
timeFrame: From start of the study drug administration up to Week 53
Secondary outcomes:
measure: Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in Liver Volume at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in Spleen Volume at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53
timeFrame: Baseline, Weeks 14, 27, 40, and 53
measure: Change From Baseline in Global Joint Range of Motion (JROM) Score at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in Anthropometric Parameter: Height at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in Anthropometric Parameter: Weight at Weeks 27 and 53
timeFrame: Baseline, Weeks 27 and 53
measure: Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores
timeFrame: Baseline, Weeks 27 and 53
measure: Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores
timeFrame: Baseline, Weeks 27 and 53
Other outcomes:
Locations:
city: Thiruvananthapuram
country: India
facility: SAT Hospital - Govt Medical College
state: Kerala
city: Jaipur
country: India
facility: JK Lone Hospital
state: Rajasthan
city: Kolkata
country: India
facility: Institute of Child Health
city: New Delhi
country: India
facility: All India Institute of Medical Sciences (AIIMS)
city: New Delhi
country: India
facility: Sir Gangaram Hospital
Study officials:
affiliation: Takeda
name: Medical Director
role: STUDY_DIRECTOR
References: