A Phase 2 study to assess the efficacy and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of fibrodysplasia ossificans progressiva in male and female paediatric and adult participants - FALKO

egar Karimian0 sites4 target enrollmentOctober 26, 2022

Overview

No summary available.

Registry

who.int

Start Date

October 26, 2022

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional

Sex

All

Sponsor

egar Karimian

•Participants are eligible to be included in the study only if all of the following criteria apply:
•Age \- Main Study
•1\. 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.
•Age \- \[18F]NaF PET\-CT Imaging Substudy
•2\. 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, ageappropriate assent.
•Type of Participant and Disease Characteristics
•3\. Participants must be clinically diagnosed with FOP, with the R206H ACVR1 mutation or other FOP variants associated with progressive HO.
•4\. Participants must have disease progression in the preceding year of the screening visit by having at least one of the following:
•a. A self\-reported flare\-up with at least one major symptom of a flare\-up, including swelling, pain (a new onset pain in a new site), decreased movement, stiffness, warmth, or redness
•b. A new palpable HO

•Participants are excluded from the study if any of the following criteria apply:
•Medical Conditions
•1\. Participants with complete heart block and left bundle branch block on screening electrocardiogram.
•2\. Participants with screening echocardiograph 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 LVEF \<50%.
•3\. Participants with severe mitral or tricuspid regurgitation on echocardiograph at screening.
•4\. Participants with significant underlying lung disease requiring supplementary oxygen or forced vital capacity \<35% of predicted at screening.
•5\. Participants with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or other significant disease as judged by the investigator.
•6\. Participants with severe hepatic impairment.
•Prior/Concomitant Therapy
•7\. Concomitant medications that are strong inhibitors (including grapefruit juice) or inducers (including St Johns Wort) of cytochrome P450 (CYP) 3A4 activity or kinase inhibitors such as imatinib.