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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-504127-90-00 | EU Trial (CTIS) Number | EU CTIS Number |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Idursulfase-IT | Experimental | 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 4.8 years. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Idursulfase-IT | Drug | Idursulfase-IT intrathecally via IDDD. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events (AEs) by Type and Severity | An adverse event (AE) means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. The severity of AEs will be determined per the investigator's assessment. | Up to approximately 4.8 years |
| Number of Participants with AEs Related to Idursulfase-IT | An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to idursulfase-IT will be reported in this outcome measure. | Up to approximately 4.8 years |
| Number of Participants with AEs Related to the IDDD | An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to the IDDD will be reported in this outcome measure. | Up to approximately 4.8 years |
| Number of Participants with AEs Related to Device Surgical Procedure |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| M.A.G.I.C. Clinic | Calgary | Alberta | T2E 7Z4 | Canada | ||
| University of British Columbia |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here on this link | View source |
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Takeda does not provide access to Individual Participant Data when a study is in a very limited (small) study population due to participant privacy concerns such as potential reidentification of study participants.
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| ID | Term |
|---|---|
| D016532 | Mucopolysaccharidosis II |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
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| ID | Term |
|---|---|
| C517982 | idursulfase |
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| Elaprase |
| Drug |
Elaprase IV infusion. |
|
An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to device surgical procedure will be reported in this outcome measure.
| Up to approximately 4.8 years |
| Number of Participants with AEs Related to IT Administration Process | An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to IT administration process will be reported in this outcome measure. | Up to approximately 4.8 years |
| Number of Participants with AEs Related to IV Elaprase Infusion | An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to IV elaprase infusion will be reported in this outcome measure. | Up to approximately 4.8 years |
| Vancouver |
| British Columbia |
| V6T1Z3 |
| Canada |
| Hospital for Sick Children | Toronto | Ontario | M5G1X8 | Canada |
| Hopital Femme Mere Enfant | Bron | Auvergne-Rhône-Alpes | 02 69229 | France |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |