Recruiting
Phase 2
Phase 3

SKY-0515

Sponsor:

Skyhawk Therapeutics, Inc.

Code:

NCT07378644

Conditions

Huntington Disease

Eligibility Criteria

Sex: All

Age: 25+

Healthy Volunteers: Not accepted

Interventions

SKY-0515

SKY-0515 Placebo

Study Details

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 Disease

Study ID

NCT07378644

Start date

Jan 6, 2026

Status verified date

Aug, 2026

Completion date

Aug, 2029

Anticipated

Primary completion date

Dec, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 25+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 25 years or older.
  • Huntington's Disease confirmed through genetic testing, with a specific change in exon 1 of the HTT gene (CAG repeat of 40 or more).
  • Total Functional Capacity (TFC) score of 10 or more).
  • Total Motor Score (TMS) of 6 or more).
  • Independence Score (IS) of 70 or more).
  • 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.
  • Agree to sign a consent form and follow the study's rules and schedule.

Exclusion Criteria:

  • Other Serious health problems or brain/spinal issues that could interfere with the study or make procedures unsafe.
  • Conditions that interfere with protocol-specified assessments, like an implanted medical device or difficulty getting an MRI.
  • Cancer, except for some types of skin cancer, or a history of cancer in the last five years.
  • Severe allergies or have reacted badly to similar drugs in the past.
  • Taking medications or treatments that might interfere with the study.
  • Participated in another study or taken experimental drugs in the last two months (or longer for some drugs).
  • Any kind of gene therapy.
  • History of suicidal thoughts, severe depression, or have attempted suicide in the past year.
  • Liver function tests show significant abnormalities.
  • Positive for hepatitis B, hepatitis C, or HIV.
  • Pregnancy, breastfeeding, or planning to become pregnant during the study.

Study Design

Enrollment

400 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: Active Comparator (1)

Dosage Level(s): Low Dose

active comparator: Active Comparator (2)

Dosage Level(s): Mid Dose

active comparator: Active Comparator (3)

Dosage Level(s): High Dose

placebo comparator: Placebo Comparator (4)

Matching placebo once daily oral

Interventions

SKY-0515

Type: Small molecule Dosage Level(s): Low, mid, high Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 18 months Use: Experimental

SKY-0515 Placebo

Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 18 months Use: Experimental

Primary outcome measure

  • Change From Baseline in the Z-score from the Composite Unified Huntington's Disease Rating Scale (cUHDRS) [ Time Frame: 72 weeks ]

Central Contacts and Locations

Central contacts

Study Director, MD

6178580041sky0515trials@skyhawktx.com

Locations

North York General Hospital

Recruiting

Toronto, Ontario, Canada, M2K 1E1

Contacts

Clare Gibbons

416-756-6788clare.gibbons@nygh.on.ca

Danica Nogo

danica.nogo@nygh.on.ca

Principal Investigator:

Phillipe Rizek

More Information

Sponsor

Skyhawk Therapeutics, Inc.

Last update posted

Sep 1, 2026

Last verified

Aug, 2026

Keywords

  • SKY-0515
  • Skyhawk
  • HTT
  • Neurodegenerative
  • Huntington Disease
  • mRNA
  • Splicing
  • Mutant Huntingtin protein

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Skyhawk Therapeutics, Inc. on 2026-09-01.