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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06873334




Registration number
NCT06873334
Ethics application status
Date submitted
6/03/2025
Date registered
12/03/2025

Titles & IDs
Public title
Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease
Scientific title
A Phase 2/3 Randomized, Double Blind, Placebo-Controlled, Dose Ranging Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
Secondary ID [1] 0 0
SKY-0515-004-ANZ
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Huntington Disease 0 0
Huntingtons Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SKY-0515
Treatment: Drugs - SKY-0515 Placebo

Active comparator: 1 (Active) - Dosage Level(s): Low dose once daily oral

Active comparator: 2 (Active) - Dosage Level(s): Mid dose once daily oral

Active comparator: 3 (Active) - Dosage Level(s): High dose once daily oral

Placebo comparator: 4 (Control) - Matching placebo once daily oral


Treatment: Drugs: SKY-0515
Study Drug Name (INN): SKY-0515 Type: Drug (small molecule) Dosage Level(s): Low, mid, high Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 12 months

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 blinded 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.

Treatment: Drugs: SKY-0515 Placebo
Route of Administration: Oral Dosage

Frequency: Once daily

Blinded Treatment Duration: 12 months

Use: Experimental

Sourcing: SKY-0515 placebo will be provided centrally by the Sponsor or subsidiary, or designee

Packaging and Labeling: Study drug will be provided in blinded 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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Changes in blood mHTT protein levels
Timepoint [1] 0 0
12 months
Primary outcome [2] 0 0
Changes in brain volume measured through MRI
Timepoint [2] 0 0
12 months
Primary outcome [3] 0 0
Changes in UHDRS
Timepoint [3] 0 0
12 months

Eligibility
Key inclusion criteria
* You must be 25 years or older.
* You must have Huntington's Disease confirmed through genetic testing, with a specific gene change (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.
* You must agree to sign a consent form and follow the study's rules and schedule.
Minimum age
25 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* You have other serious health problems or brain/spinal issues that could interfere with the study or make procedures unsafe.
* You have a condition that interferes with protocol-specified assessments, like an implanted medical device or difficulty getting an MRI.
* You have cancer, except for some types of skin cancer, or a history of cancer in the last five years.
* You have severe allergies or have reacted badly to similar drugs in the past.
* You are taking medications or treatments that might interfere with the study.
* You've been in another study or taken experimental drugs in the last two months (or longer for some drugs).
* You have had any kind of gene therapy.
* You have a history of suicidal thoughts, severe depression, or have attempted suicide in the past year.
* Your liver function tests show significant abnormalities.
* You have tested positive for hepatitis B, hepatitis C, or HIV.
* You are pregnant, breastfeeding, or planning to become pregnant during the study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/05/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2027
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
John Hunter Hospital - New Lambton Heights
Recruitment hospital [2] 0 0
Westmead Hospital - Westmead
Recruitment hospital [3] 0 0
The University of Queensland - Royal Brisbane Womens Hospital - Herston
Recruitment hospital [4] 0 0
Flinders Medical Centre - Adelaide
Recruitment hospital [5] 0 0
Calvary Health Care Bethlehem - Caulfield South
Recruitment hospital [6] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment hospital [7] 0 0
The Perron Institute for Neurology and Translational Science (Perron Institute) - Nedlands
Recruitment postcode(s) [1] 0 0
2305 - New Lambton Heights
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4006 - Herston
Recruitment postcode(s) [4] 0 0
5042 - Adelaide
Recruitment postcode(s) [5] 0 0
3162 - Caulfield South
Recruitment postcode(s) [6] 0 0
3050 - Parkville
Recruitment postcode(s) [7] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland
Country [2] 0 0
New Zealand
State/province [2] 0 0
Christchurch
Country [3] 0 0
New Zealand
State/province [3] 0 0
Wellington

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Skyhawk Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Masoud Mokhtarani
Address 0 0
Country 0 0
Phone 0 0
9253008881
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06873334