An Extension of SKY-0515 in Participants With Huntington's Disease

Официальное название

An Open Label Extension of SKY-0515 in Participants With Huntington's Disease Who Completed a Prior SKY-0515 Treatment Study

Обзор

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.

Подробное описание

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).

Вмешательства

• Препарат SKY-0515
  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

Первичные конечные точки

• Long-term safety and tolerability of SKY-0515 [Срок оценки: Continuously from enrollment to study completion or approximately 36 months]

Вторичные конечные точки (6)

• Change From Baseline in the Z-score from the Composite Unified Huntington's Disease Rating Scale (cUHDRS) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months.]

Критерии участия

Критерии включения

• Must have completed treatment in a prior SKY-0515 treatment study and, in the opinion of the Investigator, have been compliant with the prior SKY-0515 treatment protocol study procedures.
• Must be able and willing to meet all study requirements in the opinion of the Investigator including:
• Participants must be able to tolerate blood draws
• Willing and able to comply with all scheduled visits, treatment plans,laboratory tests and other study procedures
• Women who can have children must have a negative pregnancy test before starting and use two types of birth control during the study and for 30 days after the last dose of the study drug.
• Men must agree to use birth control during the study and for 90 days after the last dose.

Критерии исключения

-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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Австралия · 7 центров

• John Hunter Hospital — New Lambton Heights
• Westmead Hospital — Westmead
• The University of Queensland — Herston
• Flinders Medical Centre — Adelaide
• Calvary Health Care Bethlehem — Caulfield South
• The Royal Melbourne Hospital — Parkville
• The Perron Institute for Neurology and Translational Science (Perron Institute) — Nedlands

Новая Зеландия · 3 центра

• Huntington's Disease Association — Auckland
• New Zealand Brain and Research Institute — Christchurch
• Wellington Hospital — Wellington

Идентификаторы

NCT: NCT07537075 · SKY-0515-008

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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