Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome… (NCT06031259) | Clinical Trial Compass
Active — Not RecruitingPhase 2/3
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
Canada, France6 participantsStarted 2024-03-05
Plain-language 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.
Who can participate
Age range
3 Years – 19 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
. 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.
. The participant has continued to receive elaprase on a regular basis in studies HGT-HIT-046 or SHP609-302.
Exclusion criteria
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants with Adverse Events (AEs) by Type and Severity
Timeframe: Up to approximately 4.8 years
2
Number of Participants with AEs Related to Idursulfase-IT
Timeframe: Up to approximately 4.8 years
3
Number of Participants with AEs Related to the IDDD
Timeframe: Up to approximately 4.8 years
4
Number of Participants with AEs Related to Device Surgical Procedure
Timeframe: Up to approximately 4.8 years
5
Number of Participants with AEs Related to IT Administration Process
Timeframe: Up to approximately 4.8 years
6
Number of Participants with AEs Related to IV Elaprase Infusion
. The participant has clinically relevant intracranial hypertension.
. 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.