FALCON-HD 004-WW

14 evidence topics · 3 sources

Objective

Registered study goal

“This clinical trial aims to evaluate the safety, pharmacodynamics, and efficacy of SKY-0515, a novel, orally administered small molecule.” (opens the source at this quote in a new tab)

Location and study date

Study dates

Milestone	Registry record
Study start (actual)	“2026-01-06”
Primary completion (estimated)	“2028-12”
Study completion (estimated)	“2029-08”

Registered site countries

Country
“Argentina”
“Brazil”
“Canada”
“Chile”
“Colombia”
“Georgia”
“United States”

Expansion to the United Kingdom

“We are excited to now begin enrolling patients in our pivotal trial for Huntington's in the United States, the United Kingdom and Canada” (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).” (opens the source at this quote in a new tab)

Eligibility criteria

Key inclusion criteria

Criterion	Quoted registry text
Age	“25 years or older.”
Genetic diagnosis	“Huntington's Disease confirmed through genetic testing, with a specific change in exon 1 of the HTT gene (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).”

Treatment

Treatment arms and duration

“Type: Small molecule Dosage Level(s): Low, mid, high Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 18 months Use: Experimental” (opens the source at this quote in a new tab)

Numeric dose levels

No evidence found.

Study outcomes

Rationale for using cUHDRS change as the primary endpoint

Primary outcome definition

“Change From Baseline in the Z-score from the Composite Unified Huntington's Disease Rating Scale (cUHDRS)” (opens the source at this quote in a new tab)
“72 weeks” (opens the source at this quote in a new tab)

Rationale for cUHDRS: registry description

“For cUHDRS, z-scores of each test are summed, whereby a higher cUHDRS score is better (score of -3.06-no max value) and a change of ≥1.2 is a meaningful worsening, shown to track functional decline.” (opens the source at this quote in a new tab)

Secondary outcomes

Secondary outcome	Time frame
“Change from Baseline in Total Motor Score (TMS)”	“72 weeks”
“Change from Baseline in Total Functional Capacity (TFC)”	“72 weeks”
“Change from Baseline in Independence Scale Score (IS)”	“72 weeks”
“Change from Baseline in Symbol Digit Modalities Test (SDMT)”	“72 weeks”
“Change from Baseline in the Stroop Color and Word Test (SCWT)”	“72 weeks”

Statistical analysis description

Number of participants (Planned and analyzed)

Planned enrollment: registry record

Enrollment type	Participants
Estimated	“400”

Planned enrollment: company statement of up to an additional 400 participants

“FALCON-HD 004-WW plans to enroll up to an additional 400 participants with Stage 2 and early Stage 3 HD across more than 40 sites worldwide and is actively treating patients at a number of these sites presently.” (opens the source at this quote in a new tab)

Planned enrollment: company statement of approximately 600 participants

“FALCON-HD 004-WW plans to enroll approximately 600 participants with Stage 2 and Stage 3 HD across more than 40 sites worldwide and is actively recruiting.” (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

The trial is recruiting and has reported no results; the estimated primary completion is December 2028. Planned enrollment is 400 participants in the registry record and "up to an additional 400" in the July 2026 expansion announcement, whereas the September 2026 fifteen-month announcement states approximately 600. The 72-week primary endpoint is cUHDRS change, with TMS, TFC, IS, SDMT, and Stroop as secondary outcomes; numeric dose levels, the statistical hierarchy, and whether results will be pooled with the 004-ANZ trial have not been disclosed.