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Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

NCT ID: NCT06031259

Last Updated: 2025-11-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-05

Study Completion Date

2029-01-01

Brief Summary

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The study is an extension of two previous studies (HGT-HIT-046 \[NCT01506141\] and SHP609-302 \[NCT02412787\]). Participants must have completed one of the previous studies. The main aim of this study is to collect more information about the safety of the treatments, idursulfase-IT and elaprase, in children and adults with Hunter syndrome and cognitive impairment. Participants will receive the same treatment as in the previous studies.

Detailed Description

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The drug being tested in this study is called idursulfase. Idursulfase is being tested for long term safety in participants with Hunter syndrome and cognitive impairment.

The study will enroll up to approximately 8 patients. Participants will receive idursulfase-IT via intrathecal drug delivery device (IDDD) once monthly along with elaprase intravenous (IV) infusion, weekly.

This multi-center trial will be conducted in France and Canada. The overall time to participate in this study is approximately 4.8 years.

Conditions

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Hunter Syndrome

Keywords

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Drug Therapy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Idursulfase-IT

Participants will receive idursulfase-IT once monthly and weekly IV infusions of elaprase at the dose used in previous studies (HGT-HIT-045/SHP609-302) via IDDD until benefit is no longer derived from the treatment, or treatment is no longer tolerable, or up to approximately 4.8 years.

Group Type EXPERIMENTAL

Idursulfase-IT

Intervention Type DRUG

Idursulfase-IT intrathecally via IDDD.

Elaprase

Intervention Type DRUG

Elaprase IV infusion.

Interventions

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Idursulfase-IT

Idursulfase-IT intrathecally via IDDD.

Intervention Type DRUG

Elaprase

Elaprase IV infusion.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. The participant must have completed end of study assessments in studies HGT-HIT-046 \[NCT01506141\] or SHP609-302 \[NCT02412787\] and received a clinical benefit from idursulfase-IT in the opinion of the investigator.
2. The participant, or participant's legally designated representative, must have been informed of the nature of this open-label extension and must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed. Written consent of the participant's legally designated representative (if applicable) and the participant's consent/assent, as relevant, must be obtained.
3. The participant has continued to receive elaprase on a regular basis in studies HGT-HIT-046 or SHP609-302.

Exclusion Criteria

1. The participant has experienced, in the opinion of the investigator, a safety or medical issue that contraindicates treatment with idursulfase-IT, including, but not limited to, uncontrolled seizure disorder, bleeding disorder, and clinically relevant hypertension.
2. The participant has clinically relevant intracranial hypertension.
3. The participant is enrolled in another clinical study, other than studies HGT-HIT-046 or SHP609-302, that involves clinical investigations or use of any investigational product (drug or \[intrathecal/spinal\] device) within 30 days prior to study enrolment or at any time during the study.
Minimum Eligible Age

3 Years

Maximum Eligible Age

19 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Takeda

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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M.A.G.I.C. Clinic

Calgary, Alberta, Canada

Site Status

University of British Columbia

Vancouver, British Columbia, Canada

Site Status

Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Hopital Femme Mere Enfant

Bron, Auvergne-Rhône-Alpes, France

Site Status

Countries

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Canada France

Related Links

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https://clinicaltrials.takeda.com/study-detail/5684362136074625?idFilter=%5B%22TAK-609-3001%22%5D

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

Other Identifiers

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2023-504127-90-00

Identifier Type: CTIS

Identifier Source: secondary_id

TAK-609-3001

Identifier Type: -

Identifier Source: org_study_id