A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥ 14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.


About this study

The purpose of this study is to further evaluate the safety and effectiveness of palovarotene in adult and paediatric participants with FOP. Additionally, to ensure treatment continuity to participants who have completed one of the parent studies (Study PVO-1A-301, Study PVO-1A-202 and Study PVO-1A-204) and who, in the investigator's judgement, may benefit from palovarotene therapy.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- Participant has completed the EOS or End of Treatment Visit of Study PVO-1A-301 or
PVO-1A-202 (PVO-1A-202 Parts C and D correspond to Study PVO-1A-204 in France) and did
not previously withdraw consent from any of the parent studies to be eligible for
Study CLIN-60120-452.

- Participant must be ≥ 14 years of age (aligned with the age of treated participants in
the ongoing parent studies PVO-1A-301 and PVO-1A-202/PVO-1A-204) and qualify as 100%
skeletally mature (if < 18 years, based on assessments carried out at parent EOS Visit;
if ≥ 18 years, automatically considered 100% skeletally mature) or have reached final
adult height based on investigator's assessment, at the time the Study CLIN- 60120-452
informed consent is signed.

Exclusion Criteria:

- History of allergy or hypersensitivity to retinoids, gelatin, lactose (note that
lactose intolerance is not exclusionary) or palovarotene, or unresponsiveness to prior
treatment with palovarotene.

- Uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine,
metabolic, ophthalmologic, immunologic, psychiatric, or other significant disease.

- Intercurrent known or suspected non-healed fracture at any location;

- Any other medical condition/clinically significant abnormalities that would expose the
participant to undue risk or interfere with study assessments.

- Amylase or lipase > 2 × above the upper limit of normal (ULN) or with a history of
chronic pancreatitis.

- Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 2.5 × ULN.

- Fasting triglycerides > 400 mg/dL with or without therapy.

- Suicidal ideation (type 4 or 5) or any suicidal behaviour at the Inclusion Visit as
defined by the Columbia-Suicide Severity Rating Scale (C-SSRS).

- Current use of vitamin A or beta carotene, multivitamins containing vitamin A or beta
carotene, or herbal preparations, fish oil, and unable or unwilling to discontinue use
of these products during palovarotene treatment.

- Exposure to synthetic oral retinoids other than palovarotene within 4 weeks of the
Inclusion Visit.

- Concurrent treatment with tetracycline or any tetracycline derivatives due to the
potential increased risk of pseudotumor cerebri.

- Use of concomitant medications that are strong inhibitors or inducers of cytochrome
P450 (CYP450) 3A4 activity; or kinase inhibitors such as imatinib.

- Palovarotene is commercially available in the country where the study is being

- Any reason that, in the opinion of the investigator, would lead to the inability of
the participant and/or family to comply with the protocol.

Eligibility last updated 7/1/22. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Robert Pignolo, M.D., Ph.D.

Open for enrollment

Contact information:

Tamara Evans

(507) 284-1004


More information


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