Stopped: Following the planned FALKON Part A primary analysis, the study did not meet its primary endpoint of reducing heterotopic ossification (HO) volume compared with placebo and met predefined futility criteria.
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by the presence of bone in soft tissue where bone normally does not exist, known as Heterotopic Ossification (HO). It is often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to abnormal stiffening and immobility (ankyloses) of major joints with cumulative and irreversible loss of movement and disability. This study will evaluate the efficacy of 2 dosing regimens of IPN60130 in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and paediatric participants with FOP. It will be assessed by a scan (provides internal images of the body) called low dose Whole Body Computed Tomography (WBCT), excluding head. Adults and participants 5 years of age or older are also eligible for a sub study to evaluate HO lesions assessed by another type of scan, Fluorine-18-labelled natrium fluoride Positron Emission Tomography-Computed Tomography (\[18F\]NaF PET-CT ).
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Annualized change in HO volume as assessed by low-dose WBCT (excluding the head) in treated participants receiving IPN60130 compared with placebo.
Timeframe: From baseline to 12 months
Incidence of Adverse Events / Serious Adverse Events (AEs/SAE)
Timeframe: From baseline until the end of study (63 months)
Change from baseline in clinically significant abnormal values in laboratory parameters (haematology, biochemistry, and urinalysis)
Timeframe: From baseline until the end of study (63 months)
Change from baseline in physical examination findings
Timeframe: From baseline until the end of study (63 months)
Change from baseline in clinically significant vital signs
Timeframe: From baseline until the end of study (63 months)
Change from baseline in clinically significant Electrocardiogram (ECG) readings
Timeframe: From baseline until the end of study (63 months)