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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT06031259




Registration number
NCT06031259
Ethics application status
Date submitted
4/09/2023
Date registered
11/09/2023
Date last updated
29/09/2023

Titles & IDs
Public title
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
Scientific 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
Secondary ID [1] 0 0
2023-504127-90
Secondary ID [2] 0 0
TAK-609-3001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hunter Syndrome 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
Mental Health 0 0 0 0
Other mental health disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Idursulfase-IT
Treatment: Drugs - Elaprase

Experimental: Idursulfase-IT - Participants will receive idursulfase-IT once monthly and weekly IV infusions of elaprase at the dose used in previous studies (HGT-HIT-045/SHP609-302) via IDDD until benefit is no longer derived from the treatment, or treatment is no longer tolerable, or up to approximately 10.4 years.


Treatment: Drugs: Idursulfase-IT
Idursulfase-IT intrathecally via IDDD.

Treatment: Drugs: Elaprase
Elaprase IV infusion.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Adverse Events (AEs) by Type and Severity
Timepoint [1] 0 0
Up to approximately 10.4 years
Primary outcome [2] 0 0
Number of Participants with AEs Related to Idursulfase-IT
Timepoint [2] 0 0
Up to approximately 10.4 years
Primary outcome [3] 0 0
Number of Participants with AEs Related to the IDDD
Timepoint [3] 0 0
Up to approximately 10.4 years
Primary outcome [4] 0 0
Number of Participants with AEs Related to Device Surgical Procedure
Timepoint [4] 0 0
Up to approximately 10.4 years
Primary outcome [5] 0 0
Number of Participants with AEs Related to IT Administration Process
Timepoint [5] 0 0
Up to approximately 10.4 years
Primary outcome [6] 0 0
Number of Participants with AEs Related to IV Elaprase Infusion
Timepoint [6] 0 0
Up to approximately 10.4 years

Eligibility
Key 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.
Minimum age
3 Years
Maximum age
19 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2/Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/01/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2034
Actual
Sample size
Target
8
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Alberta
Country [2] 0 0
Canada
State/province [2] 0 0
British Columbia
Country [3] 0 0
Canada
State/province [3] 0 0
Ontario
Country [4] 0 0
France
State/province [4] 0 0
Lyon Cedex

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Takeda
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT06031259
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Takeda
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Takeda Contact
Address 0 0
Country 0 0
Phone 0 0
+1-877-825-3327
Fax 0 0
Email 0 0
medinfoUS@takeda.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT06031259