A Study Assessing the Efficacy and Safety of 2 Dosage Regimens of Oral IPN60130 for FOP


About this study

The purpose of this study is to evaluate the effectiveness of IPN60130 monotherapy compared with placebo recipients in inhibiting new HO volume in adult and paediatric participants with FOP as assessed by low-dose WBCT (excluding the head), and to evaluate the safety of IPN60130 in adult and paediatric participants with FOP.

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare and severely disabling genetic disease characterized by heterotopic ossification (HO) in muscles, tendons, and ligaments often associated with painful, recurrent episodes of soft tissue swelling (formation of flare-ups). The formation of heterotopic bone is irreversible and leads to significant morbidity and progressive disability. Presently there are no approved medical treatment options to prevent the formation of heterotopic bone in FOP. Consequently, there is a great unmet medical need for new therapeutic treatments to improve clinical outcomes in patients with FOP.

It is hypothesized that daily treatment with IPN60130 will inhibit the formation of heterotopic bone, thereby lessening the development of functional limitations.

Participation eligibility

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:

- Written, signed, and dated informed subject/parent consent; and for subjects who are
minors, age-appropriate assent (performed according to local regulations).

- 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

- 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 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.

Key Exclusion Criteria:

- Participants with complete heart block and left bundle branch block on screening

- 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

- 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

- 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

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Eligibility last updated 9/19/22. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Robert Pignolo, M.D., Ph.D.

Open for enrollment

Contact information:

Tamara Evans

(507) 284-1004


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