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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-04-21

Study Completion Date

2024-04-18

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

NCT05795361

Hunter Syndrome

AVAILABLE

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

NCT02412787

Hunter Syndrome

COMPLETED PHASE2/PHASE3

To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

NCT01301898

Mucopolysaccharidosis II

COMPLETED PHASE1/PHASE2

A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723

NCT01602601

Mucopolysaccharidosis II

COMPLETED

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

NCT00607386

Hunter Syndrome Mucopolysaccharidosis II MPS II

COMPLETED PHASE4

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hunter Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Elaprase 0.5 mg/kg

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.

Group Type EXPERIMENTAL

Elaprase

Intervention Type BIOLOGICAL

Elaprase IV infusion.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Elaprase

Elaprase IV infusion.

Intervention Type BIOLOGICAL

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Idursulfase

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Eligible Sex

ALL

Accepts Healthy Volunteers

No