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

NCT07537075 | Enrolling By Invitation Phase 2 DMC

• 1 other identifier: SKY-0515-008
• Study Type: interventional
• Target: 500
• Locations: 1 country
• Sites: 4

Brief Summary

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.

Conditions

Huntington's Disease (HD)

Keywords

SKY-0515

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (6)

• Change From Baseline in the Z-score from the Composite Unified Huntington's Disease Rating Scale (cUHDRS)

  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)

  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)

  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)

  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)

  Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months.

Study Arms (1)

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

Drug: SKY-0515

Interventions

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.

9mg dose

Eligibility Criteria

• Age: 25 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

Sponsors & Collaborators

• Skyhawk Therapeutics, Inc.: lead

Study Sites (4)

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

Flinders Medical Centre

Adelaide, South Australia, 5042, Australia

Location

Calvary Health Care Bethlehem

Caulfield South, Victoria, 3162, Australia

Location

The Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

Location

Related Links

• Skyhawk Therapeutics Website

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 3, 2026
• First Posted: April 17, 2026
• Study Start: April 10, 2026
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (4)