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 range3 Years – 19 Years

SexMALE

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.
✕. 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.

What they're measuring

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

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to Idursulfase-IT

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to the IDDD

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to Device Surgical Procedure

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to IT Administration Process

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to IV Elaprase Infusion

Timeframe: Up to approximately 4.8 years

Trial details

NCT IDNCT06031259

SponsorTakeda

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-01-01

View on ClinicalTrials.gov More Hunter Syndrome trials

Plain-language summary

Who can participate

Age range3 Years – 19 Years

SexMALE

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.
✕. 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.

What they're measuring

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

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to Idursulfase-IT

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to the IDDD

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to Device Surgical Procedure

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to IT Administration Process

Timeframe: Up to approximately 4.8 years

Number of Participants with AEs Related to IV Elaprase Infusion

Timeframe: Up to approximately 4.8 years