A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

• Register: ClinicalTrials.gov
• Last refreshed on: 2 December 2024
• Main ID: NCT05058391
• Date of registration: 24/09/2021
• Prospective Registration: Yes
• Primary sponsor: Takeda
• Public title: A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
• Scientific 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)
• Date of first enrolment: April 21, 2022
• Target sample size: 5
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT05058391
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

India

Contacts

• Name: Study Director
• Affiliation: Takeda

Key inclusion & exclusion 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.


Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Hunter Syndrome

Intervention(s)

Biological: Elaprase

Primary Outcome(s)

Number of Participants With Adverse Events (AEs) [Time Frame: From start of the study drug administration up to end of study (EOS) (Week 53)]

Number of Participants With Infusion-related Reactions (IRRs)) [Time Frame: From start of the study drug administration up to EOS (Week 53)]

Number of Participants With Adverse Drug Reactions (ADRs) [Time Frame: From start of the study drug administration up to EOS (Week 53)]

Secondary Outcome(s)

Change From Baseline in Anthropometric Parameter (Height) [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in Anthropometric Parameter (Weight) at Weeks 27 and 53 [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in Global Joint Range of Motion (JROM) Score at Week 27 and 53 [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in Liver Volume at Week 27 and 53 [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Week 27 and 53 [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in 6 Minute Walk Test (6MWT) at Week 27 and 53 [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in Spleen Volume at Week 27 and 53 [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in Ejection Fraction at Week 27 and 53 [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53 [Time Frame: Baseline, Weeks 14, 27, 40, 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) [Time Frame: Baseline, Weeks 27 and 53]

Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Week 27 and 53 [Time Frame: Baseline, Weeks 27 and 53]

Secondary ID(s)

TAK-665-4001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors