Reduction of New Heterotopic Ossification ( Heterotopic Ossification (HO) ) in the Open‐Label , Phase 3 MOVE Trial of Palovarotene for Fibrodysplasia Ossificans Progressiva ( Fibrodysplasia Ossificans Progressiva (FOP) )
Authors' conclusion
Thus, post hoc analyses showed evidence for efficacy of palovarotene in reducing new HO in FOP, but high risk of PPC in skeletally immature patients.
Study context
- Population
- Patients aged ≥4 years with fibrodysplasia ossificans progressivawith post-baseline WBCT
- Participants
- 97 vs 101
- Comparison
- Palovarotene vs untreated natural history study participants
- MOVEn = 97
- Natural history study (NHS)n = 101reference
- Outcome
- Annualized change in new HO volume vs NHS participants (low-dose WBCT), Bayesian compound Poisson model with square-root transformation
Methodological detailsexternal comparison with natural history study, post hoc 18-month interim analyses, Independent Data Monitoring Committee recommended trial continuation
- External comparison with natural history study
- Post hoc 18-month interim analyses
- Independent Data Monitoring Committee recommended trial continuation
Main finding
As reported in the abstract
New HO volume · 18 months
Probability of any reduction (Bayesian compound Poisson model (BcPM) without transformation)
99.4%
with transformation: 65.4%
WLME fitted reduction
54%
nominal p = 0.039
New HO volume
- Mean annualized new HO volume lower in MOVE vs NHS
- 60%
Further findings
Vertebral bone (post hoc computational WBCT analyses)
- Decreased vertebral BMD, content and strength, and increased vertebral fracture risk with palovarotene
- Post hoc
Safety
Adverse events with palovarotene
- ≥1 adverse event (AE)
- All patients
- ≥1 retinoid-associated AE
- 97.0%
- ≥1 serious AE
- 29.3%
- Premature physeal closure/epiphyseal disorder, patients aged <14 years
- 21/57 (36.8%)
Original publication
Reduction of New Heterotopic Ossification ( HO ) in the Open‐Label , Phase 3 MOVE Trial of Palovarotene for Fibrodysplasia Ossificans Progressiva ( FOP ). J Bone Miner Res. Published 25 January 2023. doi:10.
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