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

Regeneron Pharmaceuticals

Status and phase: Withdrawn; Phase 3

Conditions: Fibrodyplasia Ossificans Progressiva (FOP); Heterotopic Ossification (HO)

Treatments: Drug: garetosmab

Study type: Interventional

Funder types: Industry

Identifiers

NCT04577820

R2477-FOP-1940

Details and patient eligibility

About

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).

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

garetosmab

Experimental group

Treatment:

Drug: garetosmab