Urine Sample Collection From FOP Patients

The purpose of this study is to collect urine samples from patients with fibrodysplasia ossificans progressiva (FOP) for the assessment of biomarkers related to disease, disease progression and for prediction of flare-ups of the disease. Disease related biomarkers in these patients are currently unknown. This study aims to support the development of novel therapy/ies for this disease.

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Key information

Conditions

Fibrodysplasia Ossificans Progressiva Muscular Diseases Musculoskeletal Diseases Myositis Myositis Ossificans

Age range

5 year–35 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis Investigative Site

Philadelphia, Pennsylvania, 19104, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Male and female patients ≥ 5 and ≤ 35 years of age with a diagnosis of FOP.
Physically able to provide first-morning urine sample of at least 30 mL
Able to communicate well with the investigator, to understand and comply with the requirements of the study
About half of the patients enrolled should have a flare-up, which is defined as an acute exacerbation of disease activity characterized by two or more of the following symptoms: pain, swelling, decreased range of motion, impaired function

Exclusion criteria

Diagnosis of diabetes
Diagnosis of other systemic inflammatory disorder (juvenile idiopathic arthritis, systemic lupus erythematosus, etc.)
Diagnosis of cancer other than nonmelanomatous skin cancer.

Treatment and study plan

Primary outcomes

Analysis of pre-defined biomarker FGF2

Time frame: 3 months

The objective of this study is to collect urine samples from patients with FOP over a time period covering ideally quiescent disease status and progression through a flare-up for biomarker analysis
Analysis of pre-defined biomarker WEGF

Time frame: 3 months

The objective of this study is to collect urine samples from patients with FOP over a time period covering ideally quiescent disease status and progression through a flare-up for biomarker analysis
Analysis of microRNAs

Time frame: 3 months

The objective of this study is to collect urine samples from patients with FOP over a time period covering ideally quiescent disease status and progression through a flare-up for biomarker analysis

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

Urine Sample Collection From Patients With Fibrodysplasia Ossificans Progressiva (FOP) for Biomarker Analysis

Acronym: FOP

Important dates

Study start: 2013
Primary completion: 2014
Study completion: 2014
First posted: Feb 19, 2014
Registry last updated: Mar 12, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed