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

NCT05058391 | Completed Phase 4 Results

• 2 other identifiers: TAK-665-40012022-004193-39
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 5

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.

Conditions

Hunter Syndrome

Keywords

Mucopolysaccharidosis II

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death

  An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A serious TEAE was defined as any untoward medical occurrence that at any dose results in: death; is life-threatening: requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; leads to a congenital anomaly/birth defect or is an important medical event. Number of participants with TEAEs, serious TEAEs, discontinuation due to TEAEs, and death are reported.

  From start of the study drug administration up to Week 53

• Number of Participants With Adverse Drug Reactions (ADRs)

  An ADR was defined as a response to a drug which was noxious and unintended, and which occurred at doses normally used in humans for prophylaxis, diagnosis, or therapy of disease, or for the modification of physiological function.

  From start of the study drug administration up to Week 53

• Number of Participants With Infusion-related Reactions (IRRs)

  An IRR was defined as an AE that had been assessed as at least possibly related to treatment with elaprase and occurred during an infusion or up to 24 hours post-infusion.

  From start of the study drug administration up to Week 53

Secondary Outcomes (12)

• Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53

  Baseline, Weeks 27 and 53

• Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53

  Baseline, Weeks 27 and 53

• Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Weeks 27 and 53

  Baseline, Weeks 27 and 53

• Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 27 and 53

  Baseline, Weeks 27 and 53

• Change From Baseline in Liver Volume at Weeks 27 and 53

  Baseline, Weeks 27 and 53

Study Arms (1)

Elaprase 0.5 mg/kg

EXPERIMENTAL

Participants received a single dose of elaprase 0.5 milligrams per kilogram (mg/kg) IV infusion every week from Week 1 (Day 1) up to end of treatment (EOT) at Week 52.

Biological: Elaprase

Interventions

Elaprase BIOLOGICAL

Elaprase IV infusion.

Also known as: Idursulfase

Elaprase 0.5 mg/kg

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (5)

SAT Hospital - Govt Medical College

Thiruvananthapuram, Kerala, 69501, India

Location

JK Lone Hospital

Jaipur, Rajasthan, 302004, India

Location

Institute of Child Health

Kolkata, 700017, India

Location

All India Institute of Medical Sciences (AIIMS)

New Delhi, 110 029, India

Location

Sir Gangaram Hospital

New Delhi, 110060, India

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Mucopolysaccharidosis II

Interventions

idursulfase

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Medical Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 24, 2021
• First Posted: September 27, 2021
• Study Start: April 21, 2022
• Primary Completion: April 6, 2024
• Study Completion: April 18, 2024
• Last Updated: January 23, 2025
• Results First Posted: January 23, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

IN (5)