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.

Phase 1

Recruiting

• Conditions: Fibrodysplasia Ossificans Progressiva; MedDRA version: 20.0Level: PTClassification code: 10068715Term: Fibrodysplasia ossificans progressiva Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2024-511469-13-00
• Lead Sponsor: Clementia Pharmaceuticals Inc. An Ipsen Company

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-02-20
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 98

Inclusion Criteria

Age – Main Study: 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., Male and/or female participants: Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies., Male participants: Male participants of childbearing potential must agree to remain abstinent from heterosexual sex during treatment and for 90 days after treatment or, if sexually active, to use two effective methods of birth control, one of which must be highly effective during and for 90 days after treatment. The agreement to remain abstinent or use two effective methods (one of which must be highly effective) of birth control will be clearly defined in the informed consent; the participant or legally authorized representatives (e.g. parents, caregivers, or legal guardians) must sign this specific section., Female participants: Females of childbearing potential (defined in Appendix 10.5.1) must have a negative blood or urine pregnancy test (with sensitivity of at least 50 mIU/mL) prior to administration of study drug. FOCBP participants must agree to remain abstinent from heterosexual sex during treatment and for 1 month after treatment or, if sexually active, to use two effective methods of birth control, one of which must be highly effective during and for 1 month after treatment. Additionally, sexually active FOCBP participants in a heterosexual relationship must already be using two effective methods of birth control (one of which must be highly effective) 1 month before treatment is to start. Specific risks of study drug use during pregnancy as well as the agreement to remain abstinent or use two effective methods of birth control (one of which must be highly effective) will be clearly defined in the informed consent; the participant or legally authorized representatives (e.g. parents, caregivers, or legal guardians) must sign this specific section., Informed Consent: 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)., Age – [18F]NaF PET-CT Imaging Substudy: 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. 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 c.A new joint ankylosis d.An increase in Cumulative Analogue Joint Involvement Scale (CAJIS) score (if previous CAJIS assessment is available)., Participants who have participated in a prior clinical study using another investigational product for the treatment of FOP may be enro

Exclusion Criteria

Participants with complete heart block and left bundle branch block on screening electrocardiogram., 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, Female participants who are breastfeeding., 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 Individuals with disqualifying laboratory abnormalities may be rescreened once within the screening window. Rescreening may be repeated more than once if the results are atypical for the participant based on prior results from preceding year., 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 left ventricular ejection fraction (LVEF)<50%., Participants with severe mitral or tricuspid regurgitation on echocardiograph 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 other 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 substudy., 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).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified