FORZETTO
14 evidence topics · 2 sources
Objective
Confirmatory study objective
Location and study date
Trial-initiation announcement date
Study design
FORZETTO: allocation
FORZETTO: extension
| Study element | Direct quotation |
|---|---|
| Open-label long-term extension | “96 weeks” |
Eligibility criteria
FORZETTO: population
| Study element | Direct quotation |
|---|---|
| Population | “Ambulatory males aged 4–18 years” |
Glucocorticoid stability requirement
Rise-from-floor threshold at both screening assessments
Treatment
Investigational dose and schedule
Study outcomes
Rationale for using Rise-from-Floor velocity as the primary endpoint
FORZETTO: primary endpoint
FORZETTO: endpoints
| Study element | Direct quotation |
|---|---|
| Primary endpoint | “Change from baseline in RFF velocity at Week 73” |
| Selected secondary endpoint | “SV95C” |
| Selected secondary endpoint | “NSAA total score” |
| Selected secondary endpoint | “10MWR velocity” |
| Selected secondary endpoint | “4SC velocity” |
Clinical relevance of rise-from-floor velocity
Statistical analysis description
Number of participants (Planned and analyzed)
FORZETTO: planned enrollment and follow-up
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 available evidence describes the design of an approximately 90-participant confirmatory trial, not completed efficacy or safety results. The population is restricted to ambulatory males aged 4–18 years, with a screening rise-from-floor threshold below 10 seconds and stable glucocorticoids for at least 24 weeks. Therefore, the planned population does not directly address non-ambulatory participants. The primary assessment is at Week 73, following the stated 72-week treatment period.