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

Clementia Pharmaceuticals Inc, an Ipsen Company0 sites98 target enrollmentSeptember 8, 2021

Conditionsfibrodysplasia ossificans progressiva (FOP)MedDRA version: 20.0Level: PTClassification code 10068715Term: Fibrodysplasia ossificans progressivaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: fibrodysplasia ossificans progressiva (FOP)
Sponsor: Clementia Pharmaceuticals Inc, an Ipsen Company
Enrollment: 98
Status: Active, not recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

September 8, 2021

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Clementia Pharmaceuticals Inc, an Ipsen Company

Eligibility Criteria

Inclusion Criteria

•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, age\-appropriate 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 at least one flare\-up in the preceding year of the screening visit.
•5\. 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, or palovarotene may be enrolled 30 days after discontinuation or after washout of at least 5 half\-lives, whichever is longer.
•6\. Participants must be able to perform pulmonary function tests as defined in the protocol adequately and reliably.

Exclusion Criteria

•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 John’s Wort) of cytochrome P450 (CYP) 3A4 activity and strong inhibitors or inducers of CYP2C19 activity or kinase inhibitors such as imatinib.