Clinical trial NCT06031259: Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Hunter Syndrome
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2023-09-11
Enrollment: 6
Sex: MALE
Minimum age (years): 3
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed 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 4.8 years.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants with Adverse Events (AEs) by Type and Severity
timeFrame: Up to approximately 4.8 years
measure: Number of Participants with AEs Related to Idursulfase-IT
timeFrame: Up to approximately 4.8 years
measure: Number of Participants with AEs Related to the IDDD
timeFrame: Up to approximately 4.8 years
measure: Number of Participants with AEs Related to Device Surgical Procedure
timeFrame: Up to approximately 4.8 years
measure: Number of Participants with AEs Related to IT Administration Process
timeFrame: Up to approximately 4.8 years
measure: Number of Participants with AEs Related to IV Elaprase Infusion
timeFrame: Up to approximately 4.8 years
Secondary outcomes:
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: M.A.G.I.C. Clinic
state: Alberta
city: Vancouver
country: Canada
facility: University of British Columbia
state: British Columbia
city: Toronto
country: Canada
facility: Hospital for Sick Children
state: Ontario
city: Bron
country: France
facility: Hopital Femme Mere Enfant
state: Auvergne-Rhone-Alpes
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: