Evidence map›Paper›PMID 42770123›Full record

Trial reportJBMR plus2026

Palovarotene impact on fibrodysplasia ossificans progressiva (FOP): data from month 48 of the phase III MOVE trial.

Robert J Pignolo, Edward C Hsiao, Mona Al Mukaddam, Geneviève Baujat, Staffan K Berglund, Angela M Cheung, Carmen De Cunto, Patricia Delai, Nobuhiko Haga, Peter Kannu and 5 more

2 registry-linked trialsAbstract readClinical Trial
In one paragraph

Trial report in JBMR plus, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

NCT02322255 completednot on this map

A Natural History, Non-Interventional, Two-Part Study in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)

TypeobservationalSponsorClementia Pharmaceuticals Inc.Ran2014 to 2020Enrolled114ConditionsFibrodysplasia Ossificans Progressiva
NCT03312634 phase3completednot on this map

A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)

TypeinterventionalSponsorClementia Pharmaceuticals Inc.Ran2017 to 2022Enrolled107ConditionsFibrodysplasia Ossificans ProgressivaArmsPalovarotene
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Robert J PignoloDepartment of Medicine, Mayo Clinic, Rochester, MN 55905, United States.ORCID https://orcid.org/0000-0002-8533-9438
Edward C HsiaoDivision of Endocrinology and Metabolism, the UCSF Metabolic Bone Clinic, the Eli and Edyth Broad Institute for Regeneration Medicine, and the Institute of Human Genetics, Department of Medicine, and the UCSF Program in Craniofacial Biology, University of California San Francisco, San Francisco, CA 94143, United States.ORCID https://orcid.org/0000-0001-8924-106X
Mona Al MukaddamDepartments of Orthopaedic Surgery and Medicine, Center for Research in FOP and Related Disorders, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, United States.ORCID https://orcid.org/0000-0002-7138-596X
Geneviève BaujatService de Médecine Génomique des Maladies Rares, Hôpital Necker-Enfants Malades, Paris 71015, France.ORCID https://orcid.org/0000-0001-7435-0298
Staffan K BerglundDepartment of Clinical Sciences, Pediatrics, Umeå University, 901 87 Umeå, Sweden.ORCID https://orcid.org/0000-0002-9263-9578
Angela M CheungDepartment of Medicine and Joint Department of Medical Imaging, University Health Network, University of Toronto, Toronto, ON M5G, Canada.ORCID https://orcid.org/0000-0001-8332-0744
Carmen De CuntoPediatric Rheumatology Section, Department of Pediatrics, Hospital Italiano de Buenos Aires, Buenos Aires C1199ACH, Argentina.ORCID https://orcid.org/0000-0002-1306-1852
Patricia DelaiCentro de Pesquisa Clinica, Hospital Israelita Albert Einstein, São Paulo 05652-000, Brazil.ORCID https://orcid.org/0000-0002-2841-493X
Nobuhiko HagaDepartment of Rehabilitation Medicine, The University of Tokyo Hospital, Tokyo 113-8655, Japan.ORCID https://orcid.org/0000-0001-7806-7641
Peter KannuHospital for Sick Children, Toronto, ON M5G 1E8, Canada.ORCID https://orcid.org/0000-0001-9479-4232
Edna E MancillaChildren's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, United States.ORCID https://orcid.org/0000-0003-1540-3745
Rose MarinoIpsen, Cambridge, MA 02142, United States.ORCID https://orcid.org/0009-0004-5255-2023
Fei ShihIpsen, Cambridge, MA 02142, United States.ORCID https://orcid.org/0009-0006-5821-1267
Andrew StrahsIpsen, Cambridge, MA 02142, United States.ORCID https://orcid.org/0009-0006-1665-7471
Frederick S KaplanDepartments of Orthopaedic Surgery and Medicine, Center for Research in FOP and Related Disorders, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, United States.ORCID https://orcid.org/0000-0003-1277-5420

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Palovarotene is approved in several countries for reducing heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP), an ultra-rare, severely debilitating, genetic disorder. Approval was based on interim phase III MOVE trial (NCT03312634) analyses at month 18 (IA3), the most complete dataset considering subsequent clinical holds for futility and study restart; however, long-term data remain limited. We report exploratory, post hoc analyses of once daily palovarotene over the 48-mo MOVE trial, including time on and off treatment. The primary efficacy outcome was annualized change in new HO volume, per low-dose whole-body CT. Safety was assessed throughout. Post-baseline HO volume measurements were available for 97 palovarotene-treated patients from MOVE and 101 untreated patients from a natural history study (NHS; NCT02322255). Across 48 mo, mean annualized new HO volume was numerically lower (46.4%) in palovarotene-treated patients compared with untreated patients; however, this difference was not significant (nominal

Indexed as

analysis/quantification of boneclinical trialfibrodysplasia ossificans progressivagrowth platetherapeutics

Identifiers

PMID42770123
PMCPMC13592773

What OpenQuestion holds

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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.