Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruiting
Participants planned
8
Countries
1
Sites
1
Hunter Syndrome and Cognitive Impairment
To collect long-term safety data in subjects with Hunter syndrome and cognitive impairment who are receiving intrathecal idursulfase-IT and IV Elaprase® enzyme replacement therapy
Key facts
- Sponsor
- Takeda Development Center Americas Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 8 Apr 2024 → ongoing
- Decision date (initial)
- 2024-02-13
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Takeda Development Center Americas, Inc
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
To collect long-term safety data in subjects with Hunter syndrome and cognitive impairment who are receiving intrathecal idursulfase-IT and IV Elaprase® enzyme replacement therapy
Conditions and MedDRA coding
Hunter Syndrome and Cognitive Impairment
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | LLT | 10056917 | Hunter's syndrome | 10010331 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- 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.
- 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
- 03. The subject has continued to receive Elaprase on a regular basis in studies HGT-HIT-046 or SHP609-302
Exclusion criteria 3
- 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
- 02. The subject has clinically relevant intracranial hypertension
- 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
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- 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).
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Idursulfase (idursulfase-IT [HGT-2310])
PRD5483124 · Product
- Active substance
- Idursulfase
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRATHECAL
- Max daily dose
- 30 mg milligram(s)
- Max total dose
- 3600 mg milligram(s)
- Max treatment duration
- 120 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- SHIRE HUMAN GENETIC THERAPIES, INC.
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Takeda Development Center Americas Inc.
- Sponsor organisation
- Takeda Development Center Americas Inc.
- Address
- 500 Kendall Street
- City
- Cambridge
- Postcode
- 02142-1108
- Country
- United States
Scientific contact point
- Organisation
- Takeda Development Center Americas Inc.
- Contact name
- Takeda
Public contact point
- Organisation
- Takeda Development Center Americas Inc.
- Contact name
- Takeda
Third parties 1
| Organisation | City, country | Duties |
|---|---|---|
| Bionical Limited ORG-100023296
|
Derby, United Kingdom | On site monitoring, Code 10, Code 11, Code 12, Code 5, Data management, E-data capture, Code 8, Code 9 |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 4 | 1 |
| Rest of world
Canada
|
— | 4 | — |
Investigational sites
Hospices Civils De Lyon
Pediatrics, 3 Quai Des Celestins, Bp 2251, Lyon Cedex 02
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2024-04-08 | 2024-04-10 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 13 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_TAK-609-3001_Protocol_2023-504127-90-00_Redacted | 4.0 |
| Recruitment arrangements (for publication) | K1_RecruitmentArrangements | 1.1 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_13 to 18yrs | 2.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_7 to 12yrs | 2.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Adult | 3.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Adult Lacking Capacity | 3.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Parent | 5.0 |
| Subject information and informed consent form (for publication) | L1_Other subject information material_Letter to Participants_V1_11Mar2025 | 1 |
| Subject information and informed consent form (for publication) | L2_ Other subject information material_Global Comparison Table | 1.0 |
| Subject information and informed consent form (for publication) | L2_ Other subject information material_Participant Card | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Lay Summary | 1.0 |
| Synopsis of the protocol (for publication) | D1_TAK-609-3001_Protocol synopsis_2023-504127-90-00 | 4.0 |
| Synopsis of the protocol (for publication) | D1_TAK-609-3001_Protocol synopsis_2023-504127-90-00_FR | 4.0 |
Application history
4 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-10-16 | France | Acceptable 2024-02-12
|
2024-02-13 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-04-23 | France | Acceptable 2024-02-12
|
2024-04-23 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-11-20 | France | Acceptable 2024-02-12
|
2025-11-20 |
| 4 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-11-28 | France | Acceptable 2026-02-09
|
2026-02-12 |