A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP). (FALKON)

  • STATUS
    Recruiting
  • End date
    Aug 30, 2029
  • participants needed
    98
  • sponsor
    Clementia Pharmaceuticals Inc.
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Updated on 27 August 2023
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Summary

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by the presence of bone in soft tissue where bone normally does not exist, known as Heterotopic Ossification (HO). It is often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to abnormal stiffening and immobility (ankyloses) of major joints with cumulative and irreversible loss of movement and disability.

This study will evaluate the efficacy of 2 dosing regimens of IPN60130 in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and paediatric participants with FOP. It will be assessed by a scan (provides internal images of the body) called low dose Whole Body Computed Tomography (WBCT), excluding head.

Adults and participants 5 years of age or older are also eligible for a sub study to evaluate HO lesions assessed by another type of scan, Fluorine-18-labelled natrium fluoride Positron Emission Tomography-Computed Tomography ([18F]NaF PET-CT ).

Details
Condition Fibrodysplasia Ossificans Progressiva
Treatment Placebo, IPN60130
Clinical Study IdentifierNCT05039515
SponsorClementia Pharmaceuticals Inc.
Last Modified on27 August 2023

Eligibility

 Yes No Not Sure

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 clinically diagnosed with FOP, with the R206H ACVR1 mutation or other FOP variants associated with progressive HO
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 have disease progression in the preceding year of the screening visit
Participants must be able to perform pulmonary function tests adequately and reliably
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
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
Washout period for palovarotene is 30 days
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
Washout period for garetosmab is 4 months
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)

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
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