ReviewDrug design, development and therapy2025
A Narrative Review of Phase II and III Clinical Trials for the Pharmacological Treatment of Fibrodysplasia Ossificans Progressiva (FOP): Efficacy, Safety, and Challenges in Treatment Strategies.
Review in Drug design, development and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
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Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Fibrodysplasia Ossificans Progressiva (FOP) is a rare genetic disorder marked by progressive heterotopic ossification (HO), causing severe disability and reduced life expectancy. Despite advancements in understanding its pathophysiology, effective treatments remain limited. Promising pharmacological approaches include retinoic acid receptor γ (RARγ) agonists, such as palovarotene, and monoclonal antibodies targeting activin A, like garetosmab. However, the rarity of FOP complicates the generation of robust clinical evidence. Methods: This narrative review analysed data from Phase II and III clinical trials on ClinicalTrials.gov, including completed and terminated interventional studies evaluating pharmacological treatments for FOP. Key trial details were systematically extracted and reviewed, including design, interventions, outcomes, and adverse events. Results: Five trials met the inclusion criteria, focusing on palovarotene and garetosmab. Palovarotene significantly reduced HO progression, particularly during active disease phases or flare-ups. Garetosmab showed potential in preventing new HO lesions and reducing lesion activity. However, both treatments posed safety concerns, including premature epiphyseal fusion in younger patients (palovarotene) and systemic adverse events (garetosmab). Terminated trials revealed logistical and ethical challenges, such as recruitment difficulties, small sample sizes, and limitations of placebo-controlled designs. Conclusion: Pharmacological advances, including palovarotene and garetosmab, show promise in managing FOP. Nevertheless, safety concerns and the complexities of clinical trial implementation highlight the need for innovative approaches to ensure effective and equitable treatments. Future research should prioritise long-term safety, efficacy, and patient-centred outcomes to improve FOP management.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.