A randomized, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and effects on heterotopic bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva

Phase 2

Completed

• Conditions: Fibrodysplasia ossificans progressiva (FOP); fibrous tissue that is being progressively converted into bone tissue; 10028396

• Registration Number: NL-OMON50623
• Lead Sponsor: Regeneron Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 6

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 FOP diagnosis with documentation of any ACVR1 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.

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 (eg, clopidogrel),
anti-coagulants (eg, 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
(eg, hemophilia A, von Willebrand*s Factor deficiency, platelet count *20x10^9/
L).

Study & Design

• Study Type: Interventional
• Study Design: Not specified