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The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and meet certain requirements for physical ability and independence.
This is an OLE study to investigate the long-term safety, tolerability, and efficacy of SKY-0515 administered orally in participants with HD. To be eligible for this OLE study, participants must have completed dosing in a prior SKY-0515 treatment study and meet eligibility criteria for this study at an approved clinical study site. The study is comprised of an enrollment visit, a treatment period, and a follow-up visit. Throughout the study, participants will be evaluated as specified in the Schedule of Activities. At the enrollment visit (which may be the same as the last treatment visit of the prior study), participants will be consented to enter the OLE study and initiate open label treatment with 9 mg tablets of SKY-0515. Open label SKY-0515 will be self-administered daily (QD).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 9mg dose | Experimental | Drug: SKY-0515 Type: Small molecule Route of Administration: Oral Dosage Frequency: Once daily Duration: until study drug is approved or study is discontinued Use: Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SKY-0515 | Drug | Study Drug Name (INN): SKY-0515 Type: Drug (small molecule) Dosage Level(s): 9mg Route of Administration: Oral Dosage Frequency: Once daily Treatment Duration: until drug becomes commercially available or until the development of SKY-0515 is terminated by the Sponsor. Use: Experimental Sourcing: SKY-0515 will be provided centrally by the Sponsor or subsidiary, or designee Packaging and Labeling: Study drug will be provided in bottles labeled as required per country requirement. All packaging and labeling operations for the study drug will be performed according to current Good Manufacturing Practice for Medicinal Products and the relevant regulatory requirements. |
| Measure | Description | Time Frame |
|---|---|---|
| Long-term safety and tolerability of SKY-0515 | To investigate the long-term safety and tolerability of SKY-0515 through the Incidence, severity, and relationship of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) (including withdrawals due to TEAEs) | Continuously from enrollment to study completion or approximately 36 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Z-score from the Composite Unified Huntington's Disease Rating Scale (cUHDRS) | cUHDRS includes the Total Functional Capacity (range, 0-13; higher score means better functioning), Total Motor Score (range, 0-124; higher score means worse motor severity), Symbol Digit Modality Test (range, 0-110, correctly paired numbers-symbols in 90 seconds; higher score means better cognitive performance), and Stroop Color and Word Test (range, 0-no max value, correctly read color words in 45 seconds; higher score means better cognitive performance) scores. A z-score for each test is calculated, which alone can be used to describe relationship between an individual's test score and the mean score of a target population. A z-score of 0 is the mean, and ±1 is 1 standard deviation from the mean. For cUHDRS, z-scores of each test are summed, whereby a higher cUHDRS score is better (score of -3.06-no max value) and a change of ≥1.2 is a meaningful worsening, shown to track functional decline. |
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Inclusion Criteria:
Exclusion Criteria:
-Have any other conditions that, in the opinion of the Investigator or Sponsor, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Westmead Hospital | Westmead | New South Wales | 2145 | Australia | ||
| Flinders Medical Centre |
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| Label | URL |
|---|---|
| Skyhawk Therapeutics Website | View source |
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| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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|
| Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months. |
| Change from Baseline in Total Motor Score (TMS) | The total motor score (TMS) assessed motor features of HD with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural stability. Some items (such as chorea and dystonia) required grading each extremity (face, bucco-oral-lingual, and trunk) separately. Eye movements require both horizontal and vertical grades. The total motor impairment scores was the sum of all the individual 31 motor sub-items (each rated from 0 to 4), with higher scores indicating more severe motor impairment than lower scores. The range of TMS is 0-124. | Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months. |
| Change from Baseline in Total Functional Capacity (TFC) | TFC consists of five ordinally scaled items assessing a person's capacity with: (1) occupation; (2) financial affairs; (3) domestic responsibilities; (4) activities of daily living; and (5) independent living. Total score ranges from zero (worst) to 13 (best). | Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months. |
| Change from Baseline in Independence Scale Score (IS) | The independence scale assesses independence on a 0 to 100 scale with higher scores indicating better functioning. | Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months. |
| Change from Baseline in Symbol Digit Modalities Test (SDMT) | The SDMT assesses attention, visuoperceptual processing, working memory, and cognitive/psychomotor speed. The score is the number of correctly paired abstract symbols and specific numbers in 90 seconds with higher scores indicating better cognitive functioning. | Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months. |
| Change from Baseline in the Stroop Color and Word Test (SCWT) | In the Stroop Color and Word Test (SCWT) the number of words and colors read correctly is counted, with a higher score indicating better cognitive performance scores. There is no upper limit for SWR as it is a time-based task. The lower limit (worst possible) however is 0; higher score is better meaning less severity. | Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months. |
| Adelaide |
| South Australia |
| 5042 |
| Australia |
| Calvary Health Care Bethlehem | Caulfield South | Victoria | 3162 | Australia |
| The Royal Melbourne Hospital | Parkville | Victoria | 3050 | Australia |
| D003704 | Dementia |
| D002819 | Chorea |
| D020820 | Dyskinesias |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |