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

Phase 3Active Not RecruitingNCT06031259

Takeda6 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hunter Syndrome

Interventions

Idursulfase-ITDRUG

Idursulfase-IT intrathecally via IDDD.

ElapraseDRUG

Elaprase IV infusion.

Eligibility

Sex: MALEMin age: 3 YearsMax age: 19 Years

Medical Language ↔ Plain English

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.

Locations (4)

M.A.G.I.C. Clinic

Calgary, Alberta, Canada

University of British Columbia

Vancouver, British Columbia, Canada

Hospital for Sick Children

Toronto, Ontario, Canada

Hopital Femme Mere Enfant

Bron, Auvergne-Rhône-Alpes, France

Outcomes

Primary Outcomes

Number of Participants with Adverse Events (AEs) by Type and Severity

An adverse event (AE) means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. The severity of AEs will be determined per the investigator's assessment.

Time frame: Up to approximately 4.8 years

Number of Participants with AEs Related to Idursulfase-IT

An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to idursulfase-IT will be reported in this outcome measure.

Time frame: Up to approximately 4.8 years

Number of Participants with AEs Related to the IDDD

Time frame: Up to approximately 4.8 years

Number of Participants with AEs Related to Device Surgical Procedure

An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to device surgical procedure will be reported in this outcome measure.

Data from ClinicalTrials.gov

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