FALCON-HD 004-ANZ

15 evidence topics · 5 sources

Objective

Registered study goal

“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.” (opens the source at this quote in a new tab)

Location and study date

Study dates

Milestone	Registry record
Study start (actual)	“2025-05-27”
Primary completion (estimated)	“2027-05”
Study completion (estimated)	“2027-12”

Countries and first dosing site

“FALCON-HD 004-ANZ enrolled 144 participants with Stage 2 and early Stage 3 HD across sites in Australia and New Zealand, and enrollment is complete.” (opens the source at this quote in a new tab)

“The initial dosing took place at Flinders Medical Centre in Adelaide, Australia.” (opens the source at this quote in a new tab)

Study design

Phase 2/3 randomized, double-blind, placebo-controlled, dose-ranging design

“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” (opens the source at this quote in a new tab)
“They will be assigned randomly to one of four treatment arms (three doses of SKY-0515 and one placebo group) in a 1:1:1:1 ratio.” (opens the source at this quote in a new tab)

Study periods and safety oversight

“Screening Period (up to 4 weeks): Participants will undergo eligibility assessments.” (opens the source at this quote in a new tab)
“Double-Blind Treatment Period (12 months): Participants will take SKY-0515 or a placebo once daily and undergo regular clinic visits for monitoring.” (opens the source at this quote in a new tab)
“Follow-Up Period (4 weeks): After completing treatment, participants will be monitored for any remaining effects.” (opens the source at this quote in a new tab)
“An independent Data Safety Monitoring Board (DSMB) will oversee the trial to ensure participant safety.” (opens the source at this quote in a new tab)

Eligibility criteria

Key inclusion criteria

Criterion	Quoted registry text
Age	“You must be 25 years or older.”
Genetic diagnosis	“You must have Huntington's Disease confirmed through genetic testing, with a specific gene change (CAG repeat of 40 or more).”
Function	“Total Functional Capacity (TFC) score of 10 or more).”
Motor signs	“Total Motor Score (TMS) of 6 or more).”
Independence	“Independence Score (IS) of 70 or more).”

Key exclusion criteria

“You have a condition that interferes with protocol-specified assessments, like an implanted medical device or difficulty getting an MRI.” (opens the source at this quote in a new tab)
“You have had any kind of gene therapy.” (opens the source at this quote in a new tab)
“Your liver function tests show significant abnormalities.” (opens the source at this quote in a new tab)

Treatment

Treatment arms

Arm	Quoted registry description
1 (Active)	“Dosage Level(s): Low dose once daily oral”
2 (Active)	“Dosage Level(s): Mid dose once daily oral”
3 (Active)	“Dosage Level(s): High dose once daily oral”
4 (Control)	“Matching placebo once daily oral”

Blinded treatment duration

“Blinded Treatment Duration: 12 months” (opens the source at this quote in a new tab)

Numeric dose levels

No evidence found.

Study outcomes

Rationale for using blood mHTT protein, MRI brain volume, and UHDRS change as the primary endpoints

Registered primary outcomes

Primary outcome	Time frame
“Changes in blood mHTT protein levels”	“12 months”
“Changes in brain volume measured through MRI”	“12 months”
“Changes in UHDRS”	“12 months”

Primary goal as described at HDSA 2026

“The main goal of both FALCON-HD portions is to assess changes in the cUHDRS score after 72 weeks, or nearly 1.5 years.” (opens the source at this quote in a new tab)

Rationale for endpoint selection

No evidence found.

Statistical analysis description

Number of participants (Planned and analyzed)

Planned enrollment: registry record

Enrollment type	Participants
Estimated	“120”

Planned enrollment: independent trials review

“This multi-centre clinical trial will enrol 120 adults (≥25 years) with genetically confirmed HD (CAG repeat length ≥40)” (opens the source at this quote in a new tab)

Completed enrollment

“The Company also announces that the Australia and New Zealand (004-ANZ) portion of its Phase 2/3 FALCON-HD pivotal study completed enrollment six months ahead of schedule with 144 patients enrolled” (opens the source at this quote in a new tab)

Analyzed population

No evidence found.

Description of analysis sets

No evidence found.

Results

Participant disposition

No evidence found.

Baseline characteristics

No evidence found.

Efficacy results

No evidence found.

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

No evidence found.

Study limitations

Summary

No results have been reported. The registry lists three primary outcomes at 12 months (blood mHTT, MRI brain volume, and UHDRS change) and an estimated enrollment of 120 participants, while 144 participants were enrolled and conference coverage describes cUHDRS change at 72 weeks as the main goal; the numeric dose levels, final analysis plan, and multiplicity handling are not public. Eligibility requires TFC of 10 or more and TMS of 6 or more, so results will apply to stage 2 and early stage 3 HD in adults aged 25 years or older.