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

Phase 1

• Conditions: Fibrodysplasia Ossificans Progressiva (FOP); MedDRA version: 20.0Level: PTClassification code 10068715Term: Fibrodysplasia ossificans progressivaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2016-005035-33-ES
• Lead Sponsor: Regeneron Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-01-22
• Study Completion: 2021-09-16
• Last Update Posted: 26 September 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 44

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.
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 (e.g. 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. Previous history or diagnosis of cancer.

Study & Design

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