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

The purpose of this study is to evaluate the effectiveness, safety, tolerability, and PK of INCB000928 over a 24-week treatment period followed by a 52-week, open-label extension period.

The purpose of this study to evaluate the efficacy of andecaliximab in FOP to inhibit the development of new HO lesions as measured by annualized new HO lesion rate.

The purpose of this study is test the effectiveness of the senolytic drug (Fisetin) in reducing senescent cell abundance in blood in elderly adults.

The purpose of this study is to create an older adult registry from a primary care population and to identify patients who may be eligible for participation in future research studies.

This is a pilot study to evaluate whether targeting inflammation will help reduce markers of insulin resistance inflammation, bone resorption and physical dysfunction in elderly women with gait disturbance. Positive results of this study would lead to the development of a larger clinical trial examining the effects of this intervention on age-related dysfunction.

The purpose of this study is to evaluate the effectiveness of fidrisertib (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 fidrisertib (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 except for palovarotene recently approved in some countries.  However, there are several drug candidates under clinical investigation. 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.

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation (heterotopic ossification or HO) in muscles, tendons, and ligaments. Flare-ups begin early in life and may occur spontaneously or after soft tissue trauma, vaccinations, or influenza infections. Recurrent flare-ups progressively restrict movement by locking joints leading to cumulative loss of function and disability. Mouse models of FOP have demonstrated the ability of retinoic acid receptor gamma (RARγ) agonists such as palovarotene to prevent HO following injury. This 24-month study will (1) continue to follow the 40 FOP subjects who completed Clementia Study PVO-1A-201; (2) enroll up to 20 additional new subjects who have achieved at least 90% skeletal maturity; and (3) evaluate the ability of different palovarotene dosing regimens to prevent HO in these subjects.

This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple osteochondromas (MO).

The purpose of this study is to measure changes of the anti- A/H1N1, anti-A/H3N2, and anti-B influenza virus strains serum circulating antibodies (as assessed using hemagglutination inhibition (HAI) assay) levels in elderly patients.

This is a two period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2). Primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP). Primary efficacy objective of the study is to assess the effect of REGN2477 versus placebo on the change from baseline in heterotopic ossification (HO) in patients with FOP, as determined by 18-NaF uptake in HO lesions by positron emission tomography (PET) and in total volume of HO lesions by computed tomography (CT). Secondary objectives are: - To assess the effect of REGN2477 versus placebo on the change from baseline in HO, as determined by the number of new HO lesions identified by 18F-NaF PET or by CT - To assess the effect of REGN2477 versus placebo on the change from baseline in 18F-NaF standardized uptake value maximum (SUVmax) of individual active HO site(s) by PET - To compare the effect of REGN2477 versus placebo on pain due to FOP, as measured by the area under the curve (AUC) for pain based on daily numeric rating scale (NRS) scores - To assess the effect of REGN2477 versus placebo on the change from baseline in biochemical markers of bone formation - To characterize the concentrations of total activin A at baseline and over time following the first dose of study drug - To characterize the concentration-time profile (pharmacokinetics [PK]) of REGN2477 in patients with FOP - To assess the immunogenicity of REGN2477

The purpose of this study is to conduct ACVR1 mutational analysis in individuals with heterotopic ossification of the spine.

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO) often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to ankyloses of major joints with cumulative and irreversible loss of movement and disability.

The purpose of this study is to further evaluate the safety and effectiveness of palovarotene in adult and paediatric participants with FOP. Additionally, to ensure treatment continuity to participants who have completed one of the parent studies (Study PVO-1A-301, Study PVO-1A-202 and Study PVO-1A-204) and who, in the investigator's judgement, may benefit from palovarotene therapy.