Characterization of flare-ups and impact of garetosmab in adults with fibrodysplasia ossificans progressiva: a post hoc analysis of the randomized, double-blind, placebo-controlled LUMINA-1 trial
Authors' conclusion
In this analysis, 71% of placebo-treated adults with FOP experienced flare-ups over 28 wk, which were associated with an increased volume of newly formed heterotopic bone. Garetosmab reduced the severity and duration of flare-ups, with effects sustained during the entire trial.
Study context
- Population
- Adults with fibrodysplasia ossificans progressiva (FOP)
- Comparison
- Garetosmab 10 mg/kg IV every 4 wk vs placebo over 28 wk
- Placebon = 24reference
Methodological detailsphase 2, double-blind, placebo-controlled, 28-wk open-label treatment and extension (Periods 2 and 3)
- Phase 2
- Double-blind
- Placebo-controlled
- 28-wk open-label treatment and extension (Periods 2 and 3)
Primary result
As reported in the abstract
Flare-ups in placebo-treated patients · 28 wk
Placebo
71%
17 of 24
- Placebo patients with flare-ups who developed a new heterotopic ossification (HO) lesion irrespective of flare-up location
- 59% (10/17)
- Flare-ups location-matched to new HO lesions
- 24%
- New HO lesions in placebo cohort by week 28
- 29
- New HO lesions in same location as new or ongoing flare-ups
- 12 (41%)
Further findings
Duration of patient-reported flares
Corticosteroid use frequency · numerically reduced with garetosmab
Volume of newly formed heterotopic bone in placebo patients, prior flare-up vs none · week 28
- Prior flare-up, median [Q1:Q3]
- 16.6 [12.0:31.1] cm³
- No flare-up, median
- 3.2 cm³
- Higher volume with prior flare-up
Duration of patient-reported flares
- Garetosmab reduced the severity of flare-ups vs placebo
Original publication
Characterization of flare-ups and impact of garetosmab in adults with fibrodysplasia ossificans progressiva: a post hoc analysis of the randomized, double-blind, placebo-controlled LUMINA-1 trial. J Bone Miner Res. Published 31 August 2024. doi:10.
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