Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial
Study context
- Population
- Adults with fibrodysplasia ossificans progressiva (FOP)43 in period 2
- Participants
- 20 vs 24
- Comparison
- Garetosmab vs placebo
- Garetosmabn = 20
- Placebon = 24reference
- Primary endpoint
- Safety and, for period 1, activity and size of HO lesions (total lesion activity by PET-CT)
- Follow-up
- Period 1 (28 weeks)
Methodological detailsdouble-blind, placebo-controlled, phase 2, open-label period 2
- Double-blind
- Placebo-controlled
- Phase 2
- Open-label period 2
Primary result
As reported in the abstract
Total lesion activity by PET-CT
- Garetosmab vs Placebo: P = 0.0741
Further findings
New heterotopic ossification (HO) lesions in placebo patients crossing over to garetosmab (period 2 primary efficacy end point, prospectively changed)
- New HO lesions, period 2 (on garetosmab)
- 0%
- New HO lesions, period 1 (on placebo)
- 40.9%
- P
- P = 0.0027
- Open-label period 2
Deaths · open-label period 2
- Deaths
- 5 of 44; 11.4%
- Considered unlikely to be related; causality cannot be ruled out
Safety
Treatment-emergent adverse events · period 1
- All patients had ≥1 TEAE, notably epistaxis, madarosis and skin abscesses
Original publication
Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial. Nat Med. Published 28 September 2023. doi:10.
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