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

Hunter Syndrome Clinical Trial

Official title:

An Open-Label Extension for Subjects in Studies HGT-HIT-046 and SHP609-302 Evaluating Long-Term Safety of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Subjects With Hunter Syndrome and Cognitive Impairment

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06031259
• Other study ID #: TAK-609-3001
• Secondary ID: 2023-504127-90
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: March 5, 2024
• Est. completion date: June 1, 2034

Study information

• Verified date: April 2024
• Source: Takeda
• Contact: Takeda Contact
• Phone: +1-877-825-3327
• Email: medinfoUS[@]takeda.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

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 10.4 years.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 8
• Est. completion date: June 1, 2034
• Est. primary completion date: January 1, 2034
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 3 Years to 19 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cognitive Dysfunction
• Hunter Syndrome
• Mucopolysaccharidosis II
• Syndrome

Intervention

Drug:
• Idursulfase-IT
Idursulfase-IT intrathecally via IDDD.
• Elaprase
Elaprase IV infusion.

Locations

Country	Name	City	State
Canada	M.A.G.I.C. Clinic	Calgary	Alberta
Canada	Hospital for Sick Children	Toronto	Ontario
Canada	University of British Columbia	Vancouver	British Columbia
France	Hopital Femme Mere Enfant	Bron	Auvergne-Rhone-Alpes

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Countries where clinical trial is conducted

Canada,  France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Up to approximately 10.4 years
Primary	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.	Up to approximately 10.4 years
Primary	Number of Participants with AEs Related to the IDDD	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 the IDDD will be reported in this outcome measure.	Up to approximately 10.4 years
Primary	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.	Up to approximately 10.4 years
Primary	Number of Participants with AEs Related to IT Administration Process	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 IT administration process will be reported in this outcome measure.	Up to approximately 10.4 years
Primary	Number of Participants with AEs Related to IV Elaprase Infusion	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 IV elaprase infusion will be reported in this outcome measure.	Up to approximately 10.4 years

External Links

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