A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 40

Inclusion Criteria

1. Men and women 18 to 60 years of age at screening.
2. Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive heterotopic ossification).
3. Confirmation of classic FOP diagnosis with documentation of the ACVR1[R206H] mutation.
4. FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, and other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of heterotopic ossifications (increase in site or number of HO lesions) with/without being associated with flare-up episodes.
5. Willing and able to undergo PET and CT imaging procedures and other procedures as defined in this study.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Significant concomitant illness or history of significant illness such as, but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study.
2. Use of bisphosphonate within 1 year of screening.
3. Concurrent participation in another interventional clinical study, or a non-interventional study with radiographic measures or invasive procedures (eg collection of blood or tissue samples). Participation in the FOP Connection Registry or other studies in which patients complete study questionnaires are allowed.
4. Pregnant or breastfeeding women.
5. Male and women of childbearing potential patients who are unwilling to practice highly effective contraception.
6. Patients who are on concomitant antiplatelet therapy (e.g., clopidogrel), anti-coagulants (e.g., warfarin, heparin, factor Xa inhibitor, or thrombin inhibitors) in the last 30 days or within 5 half-lives of the therapy, whichever is longer. Low dose (=100 mg/day) acetylsalicylic acid (aspirin) is acceptable.
7. Patients with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise
8. Patients with a known pre-existing medical history of a bleeding diathesis (e.g., hemophilia A, von Willebrand’s Factor deficiency, platelet count =20x10^9/L).

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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