NCT02745158

Brief Summary

The Fibrodysplasia Ossificans Progressiva (FOP) Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based patient portal. A physician portal (in development) will allow physicians to enter clinical data about their patients. The objectives are to organize the international FOP community for participation in clinical trials; to enable FOP patients worldwide to report data in a shared forum; to improve the collective understanding of FOP natural history; and to advance the understanding of FOP treatment outcomes.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
800

participants targeted

Target at P75+ for all trials

Timeline
179mo left

Started Jul 2015

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress43%
Jul 2015Dec 2040

Study Start

First participant enrolled

July 1, 2015

Completed
8 months until next milestone

First Submitted

Initial submission to the registry

March 6, 2016

Completed
2 months until next milestone

First Posted

Study publicly available on registry

April 20, 2016

Completed
19.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2035

Expected
5.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2040

Last Updated

April 8, 2026

Status Verified

April 1, 2026

Enrollment Period

20.1 years

First QC Date

March 6, 2016

Last Update Submit

April 2, 2026

Conditions

Keywords

Fibrodysplasia ossificans progressivaFOPInternational FOP AssociationPatient Advocacy GroupPatient RegistryNatural HistoryPatient-Reported OutcomesObservational DatabaseBone Morphogenetic ProteinBMPBone GrowthJointMobilityFlare-UpHeterotopic OssificationEctopic BoneOsteochondromaHallux ValgusALK2ACVR1Activin AIFOPAActivin A Receptor Type IActivin Receptor-like Kinase-2

Outcome Measures

Primary Outcomes (3)

  • Patient Reported Changes in New Bone Growth Using a Patient-Directed Survey Developed by the IFOPA

    The patient is able to report changes in new bone growth among 27 joints and body locations.

    Baseline, then every six months for up to 10 years

  • Patient Reported Changes in Episodic Flare-Ups Using a Patient-Directed Survey Developed by the IFOPA

    The patient is able to report changes in episodic flare-ups among 27 joints and body locations.

    Baseline, then every six months for up to 10 years

  • Patient Reported Changes in Mobility Using a Patient-Directed Survey Developed by the IFOPA

    The patient is able to report changes in mobility among 27 joints and body locations.

    Baseline, then every six months for up to 10 years

Secondary Outcomes (9)

  • Patient Reported Changes in Health Resource Utilization Using a Patient-Directed Survey Developed by the IFOPA

    Baseline, then every six months for up to 10 years

  • Patient Reported Changes in Signs Related to the Ear Using a Patient-Directed Survey Developed by the IFOPA

    Baseline, then every six months for up to 10 years

  • Patient Reported Changes in Signs Related to the Skin Using a Patient-Directed Survey Developed by the IFOPA

    Baseline, then every six months for up to 10 years

  • Patient Reported Changes in Signs Related to the Central Nervous System Using a Patient-Directed Survey Developed by the IFOPA

    Baseline, then every six months for up to 10 years

  • Patient Reported Changes in Signs Related to the Pulmonary System Using a Patient-Directed Survey Developed by the IFOPA

    Baseline, then every six months for up to 10 years

  • +4 more secondary outcomes

Other Outcomes (3)

  • Patient Reported Changes in Activities of Daily Living Using the FOP-Physical Functioning Questionnaire (PFQ)

    Baseline, then every six months for up to 10 years

  • Patient Reported Changes in Overall Health Using the PROMIS Global Health Scale

    Baseline, then every six months for up to 10 years

  • Patient Reported Changes in the Need for Assistive Devices, Aids, Attendants, and Adaptations Using a Survey Developed by the IFOPA

    Baseline, then every six months for up to 10 years

Study Arms (1)

FOP Patients

None. IFOPA-REG-001 is a FOP patient observational registry study.

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Individuals with Fibrodysplasia Ossificans Progressive (FOP) from the international FOP community will be invited to join the Registry.

You may qualify if:

  • Participants must have a confirmed diagnosis of FOP.
  • Participants (or a parent or legal guardian) must be willing and able to provide written informed consent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The International Fibrodysplasia Ossificans Progressiva Association (IFOPA)

North Kansas City, Missouri, 064116, United States

RECRUITING

Related Publications (1)

  • Pignolo RJ, Kimel M, Whalen J, Kawata AK, Artyomenko A, Kaplan FS. The Fibrodysplasia Ossificans Progressiva Physical Function Questionnaire (FOP-PFQ): A patient-reported, disease-specific measure. Bone. 2023 Mar;168:116642. doi: 10.1016/j.bone.2022.116642. Epub 2022 Dec 13.

Related Links

MeSH Terms

Conditions

Myositis OssificansOssification, HeterotopicOsteochondromaHallux Valgus

Condition Hierarchy (Ancestors)

MyositisMuscular DiseasesMusculoskeletal DiseasesPathologic ProcessesPathological Conditions, Signs and SymptomsNeoplasms, Bone TissueNeoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasmsOsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesFoot Deformities

Study Officials

  • Mark Hamilton, PhD

    International Fibrodysplasia Ossificans Progressiva Association

    STUDY DIRECTOR

Central Study Contacts

Mark S Hamilton, PhD

CONTACT

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Target Duration
20 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 6, 2016

First Posted

April 20, 2016

Study Start

July 1, 2015

Primary Completion (Estimated)

July 31, 2035

Study Completion (Estimated)

December 31, 2040

Last Updated

April 8, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

Formal data sharing agreements are planned between the International FOP Association and biopharmaceutical companies doing research in Fibrodysplasia Ossificans Progressiva (FOP). Publications are also planned with biopharmaceutical companies and physician researchers.

Locations