Study to Assess the Efficacy and Safety of Garetosmab in Japanese Adult Patients With Fibrodysplasia Ossificans Progressiva (FOP)

Regeneron Pharmaceuticals

Overview

The primary safety objective of the study is to assess the safety and tolerability of garetosmab in Japanese male and female adult patients with FOP. The primary efficacy objective of the study is to assess the effect of garetosmab on Heterotopic ossification (HO) in Japanese adult patients with FOP, as determined by the number of new heterotopic bone lesions identified by computed tomography (CT).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Conditions

Fibrodyplasia Ossificans Progressiva (FOP)Heterotopic Ossification (HO)

Interventions

garetosmabDRUG

Repeated doses administered intravenously (IV) every four weeks (Q4W)

Eligibility

Sex: ALLMin age: 18 YearsMax age: 60 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive HO) * Confirmation of FOP diagnosis with documentation of any Type I activin A receptor (ACVR1) mutation * FOP disease activity, as defined in the protocol, within 1 year of screening visit * Willing and able to undergo PET and CT imaging procedures and other procedures as defined in this study * Able to understand and complete study-related questionnaires and diaries (assistance from caregivers is allowed) Key Exclusion Criteria: * Patient has 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 * Previous history or diagnosis of cancer * Severely impaired renal function defined as estimated glomerular filtration rate \<30 mL/min/1.73 m2 calculated by the Modification of Diet in Renal Disease equation (1 retest is allowed) * Uncontrolled diabetes defined as hemoglobin A1C (HbA1c) \>9% at screening (1 retest allowed) * History of severe respiratory compromise, as defined in protocol * Concurrent participation in another interventional clinical study or a non-interventional study with radiographic measures or invasive procedures * Pregnant or breastfeeding women NOTE: Other protocol defined inclusion/exclusion criteria apply.

Outcomes

Primary Outcomes

Incidence and severity of treatment-emergent adverse event (TEAEs)

Time frame: Through week 28

Number of new HO lesions as assessed by CT

Time frame: At week 28

Secondary Outcomes

Total volume of new HO lesions as assessed by CT

Time frame: At week 28

Number of new HO lesions as assessed by positron emission tomography (PET)

Time frame: At week 28

Total lesion activity in new HO lesions as assessed by PET

Time frame: At week 28

Percent of patients with new HO lesions as assessed by CT

Time frame: At week 28

Percent of patients with new HO lesions as assessed by PET

Time frame: At week 28

Percent of patients with investigator-assessed flare-ups

Time frame: Baseline to week 28

Percent of patients with investigator-assessed flare-ups

Time frame: Baseline to week 56

Percent of patients with flare-ups assessed by patient e-diary

Time frame: Baseline to week 28

Percent of patients with flare-ups assessed by patient e-diary

Time frame: Baseline to week 56

Data from ClinicalTrials.gov

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Number of new HO lesions as assessed by CT

Time frame: At week 56

Total volume of new HO lesions as assessed by CT

Time frame: At week 56

Percent of patients with new HO lesions as assessed by CT

Time frame: At week 56

Number of new HO lesions as assessed by PET

Time frame: At week 56

Total lesion activity in new HO lesions as assessed by PET

Time frame: At week 56

Percent of patients with new HO lesions as assessed by PET

Time frame: At week 56

Change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET

Time frame: Baseline and week 28

Change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET