Objectives:
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder caused by ALK2/ACVR1 mutation and characterized by heterotopic ossification (HO) and progressive disability. IPN60130 is a selective ALK2/ACVR1 inhibitor being investigated for FOP treatment.1 Here, we describe methodology of the FALKON trial (NCT05039515) designed to compare efficacy and safety of IPN60130 with placebo in patients (pts) with FOP.
Methods:
Pts will be randomized to oral placebo, or low or high dose IPN60130 for 12 months; pts receiving placebo will then transition to IPN60130 for 12 months. Enrollment criteria include: ≥5 years old, FOP diagnosis with disease-causing mutation, and either a flare-up, new HO or joint ankylosis, or increase in Cumulative Analogue Joint Involvement Scale (CAJIS) score in the prior year. Recruitment is ongoing to enroll 90 pts. The primary efficacy outcome will be annualized change from Baseline in HO volume to Month 12, assessed by low-dose whole-body computed tomography (CT). Secondary efficacy outcomes are presented in the Table. Safety will be assessed via adverse event (AE) and serious AE incidence over 25 months.
Summary:
Results from FALKON will allow evaluation of IPN60130 in FOP.
References
Funding: Sponsored by Ipsen.
Table: Secondary efficacy outcomes
|
Timeframe, monthsa |
Outcome |
Comparison |
|
12 |
Change from Baseline (CfB) in volume of new heterotopic ossification (HO) lesionsb |
IPN60130 vs placebo |
|
CfB in number of HO lesionsb |
||
|
Flare-up rate; number of flare-up days |
||
|
Number of body regions with new HO |
||
|
CfB in pain intensity |
||
|
Proportion of patients with new HO |
||
|
24 |
CfB in HO volumeb |
IPN60130 vs placebo and untreated natural history study (NCT02322255) participants |
aFrom Baseline up to the month given; bAssessed by low-dose whole-body computed tomography.
Disclosures: SP, NK, CP: Employees of Ipsen. FS: Employee and stockholder of Ipsen.