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.
Key Inclusion Criteria:
- Participants must be at least 5 years of age, to be confirmed (entry for younger
paediatric participants <15 years of age will only be once safety in adult and older
paediatric participants ≥15 years of age has been established) at the time of signing
the informed participant/parent consent and, for participants who are minors,
age-appropriate assent.
- Participants must be at least 15 years of age at the time of signing the informed
participant/parent consent for the main study and, for participants who are minors,
age-appropriate assent
- Participants must be clinically diagnosed with FOP, with the R206H ACVR1 mutation or
other FOP variants associated with progressive HO.
- Participants must have disease progression in the preceding year of the screening
visit.
- Participants who have participated in a prior clinical study using another
investigational product for the treatment of FOP may be enrolled after a washout of at
least 5 half-lives of the other investigational product. Participants with prior
treatment such as, but not limited to, imatinib, isotretinoin, garetosmab, or
palovarotene may be enrolled 30 days after discontinuation or after washout of at
least 5 half-lives, whichever is longer.
1. Washout period for palovarotene is 30 days
2. Washout period for garetosmab is 4 months
- Participants must be able to perform pulmonary function tests adequately and reliably.
- Participants must be able to have an adequate echocardiography assessment at screening
for evaluation of left ventricular structure and function as defined by the protocol.
- Participants must be accessible for treatment and follow-up and be able to undergo all
study procedures. Participants living at distant locations from the investigational
site must be able and willing to travel to a site for the initial and all on-site
follow-up visits. Participants must be able to undergo low-dose WBCT (excluding head)
without sedation.
- Body weight ≥10 kg.
- Abstinent or using two highly effective forms of birth control. Females must also have
a negative blood or urine pregnancy test prior to administration of study drug.
- Participants must be capable of giving written, signed, and dated informed
participant/parent consent; and for participants who are minors, age-appropriate
assent and/or legal guardian consent (performed according to local regulations)
Key Exclusion Criteria:
- Participants with complete heart block and left bundle branch block on screening
electrocardiogram.
- Participants with screening echocardiography showing septal or left ventricular free
wall thickness >12 mm for adult participants or a z-score >3 compared with population
norms for children and adolescent participants or left ventricular ejection fraction
(LVEF) <50%.
- Participants with severe mitral or tricuspid regurgitation on echocardiography at
screening.
- Participants with significant underlying lung disease requiring supplementary oxygen
or forced vital capacity <35% of predicted at screening.
- Participants with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal,
endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or another significant
disease as judged by the investigator.
- Participants with severe hepatic impairment.
- Concomitant medications that are strong inhibitors (including grapefruit juice) or
inducers (including St John's Wort) of cytochrome P450 (CYP) 3A4 activity; or kinase
inhibitors such as imatinib.
- Prior use in the past year and concomitant use of bisphosphonates for participants in
the PET-CT sub study.
- Concurrent participation in another interventional clinical study, or a
noninterventional study with radiographic measures or invasive procedures (e.g.
collection of blood or tissue samples).
- Amylase or lipase >2× the upper limit of normal (ULN) or with a history of chronic
pancreatitis.
- Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >5×ULN.
- Participants with hematologic abnormalities:
- Hgb<10g/dL
- Platelets<75,000/mm3
- WBC<2000/mm3
- Participants with coagulation test measurements outside of the normal range at
screening.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Eligibility last updated 7/11/23. Questions regarding updates should be directed to the study team contact.