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

NCT04577820 | Withdrawn Phase 3 FDA Drug

• 1 other identifier: R2477-FOP-1940
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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).

Conditions

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

Outcome Measures

Primary Outcomes (2)

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

  Through week 28

• Number of new HO lesions as assessed by CT

  At week 28

Secondary Outcomes (39)

• Total volume of new HO lesions as assessed by CT

  At week 28

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

  At week 28

• Total lesion activity in new HO lesions as assessed by PET

  At week 28

• Percent of patients with new HO lesions as assessed by CT

  At week 28

• Percent of patients with new HO lesions as assessed by PET

  At week 28

Study Arms (1)

garetosmab

EXPERIMENTAL

Drug: garetosmab

Interventions

garetosmab DRUG

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

Also known as: REGN2477

garetosmab

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

MeSH Terms

Conditions

Ossification, Heterotopic

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 30, 2020
• First Posted: October 8, 2020
• Study Start: October 13, 2021
• Primary Completion: March 1, 2022
• Study Completion: October 8, 2022
• Last Updated: November 1, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: When Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication, has made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry), has the legal authority to share the data, and has ensured the ability to protect participant privacy.
• Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf