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

NCT ID: NCT04577820

Last Updated: 2021-11-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE3

Study Classification

INTERVENTIONAL

Study Start Date

2021-10-13

Study Completion Date

2022-10-08

Brief Summary

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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).

Detailed Description

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Conditions

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Fibrodyplasia Ossificans Progressiva (FOP) Heterotopic Ossification (HO)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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garetosmab

Group Type EXPERIMENTAL

garetosmab

Intervention Type DRUG

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

Interventions

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garetosmab

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

Intervention Type DRUG

Other Intervention Names

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REGN2477

Eligibility Criteria

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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)

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trial Management

Role: STUDY_DIRECTOR

Regeneron Pharmaceuticals

Other Identifiers

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R2477-FOP-1940

Identifier Type: -

Identifier Source: org_study_id