Clinical trial • Not applicable • Rare Disease

IDURSULFASE for Hunter syndrome | Cognitive impairment

Not applicable trial of IDURSULFASE for Hunter syndrome | Cognitive impairment. open-label, none/not specified-controlled. 4 participants.

Overview

open-label, none/not specified-controlled Not applicable trial across 1 site in France.

Recruits 4 paediatric patients.

Vulnerable Population: Vulnerable population selected. The study enrols subjects with Hunter syndrome and cognitive impairment, including minors (documents include ICFs for 7 to 12 years and 13 to 18 years) and adults lacking capacity (ICF for Adult Lacking Capacity). Consent must be obtained from the subject or the subject's legally designated representative as applicable; subject consent/assent must be obtained as relevant. Age-specific subject information and informed consent forms are provided (7-12 yrs, 13-18 yrs, Parent, Adult, Adult Lacking Capacity).

{"criterion_text":"- 01. The subject must have completed end of study assessments in studies HGT-HIT-046 or SHP609-302 and received a clinical benefit from idursulfase-IT in the opinion of the investigator."}
{"criterion_text":"- 02. The subject, or subject'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. Consent of the subject’s legally designated representative (as applicable) and the subject’s consent/assent, as relevant, must be obtained"}
{"criterion_text":"- 03. The subject has continued to receive Elaprase on a regular basis in studies HGT-HIT-046 or SHP609-302"}

{"criterion_text":"- 01. The subject 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"}
{"criterion_text":"- 02. The subject has clinically relevant intracranial hypertension"}
{"criterion_text":"- 03. The subject 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"}

{"endpoint_text":"- 01. The primary endpoint of this study is the safety of intrathecal idursulfase-IT administration. Safety will be assessed by AEs (by type, severity, and relationship to treatment [idursulfase-IT, the IDDD, device surgical procedure, or IT administration process] and IV Elaprase infusion).","definition_or_measurement_approach":"Safety will be assessed by adverse events (AEs) classified by type, severity, and relationship to treatment (idursulfase-IT, the IDDD/device, device surgical procedure, IT administration process) and by IV Elaprase infusion."}

Planned Sample Size: 4
Recruitment Window Months: 120
Consent Approach: Informed consent must be voluntarily signed by the subject or the subject's legally designated representative after explanation of all relevant aspects. Consent of the legally designated representative (as applicable) and the subject’s consent/assent (as relevant) must be obtained. Age-specific information sheets and ICFs are provided (7 to 12 years; 13 to 18 years; Parent information/consent; Adult; Adult Lacking Capacity).

Only enrolling subjects who completed and derived clinical benefit from the parent clinical trials HGT-HIT-046 and SHP609-302 (i.e., recruitment is restricted to subjects from those parent trials).

Name: Bionical Limited
Responsibilities: sponsorDuties codes: 1, 10, 11, 12, 5, 6, 7, 8, 9; regulatory and clinical development contact regulatoryclindev@bionicalemas.com

{"country":"United Kingdom","full_name":"Bionical Limited","duties_or_roles":"sponsorDuties codes: 1, 10, 11, 12, 5, 6, 7, 8, 9; contact email regulatoryclindev@bionicalemas.com; phone +441462424400","organisation_type":"Pharmaceutical company"}

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