Urine Sample Collection From FOP Patients

Fibrodysplasia Ossificans Progressiva Clinical Trial

— FOP  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02066324
• Other study ID #: CPJMR0062203
• Status: Completed
• Phase: N/A
• First received: February 5, 2014
• Last updated: March 11, 2015
• Start date: November 2013
• Est. completion date: August 2014

Study information

• Verified date: March 2015
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: August 2014
• Est. primary completion date: August 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years to 35 Years

Eligibility

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.

Study Design

N/A

Related Conditions & MeSH terms

• Fibrodysplasia Ossificans Progressiva

Locations

Country	Name	City	State
United States	Novartis Investigative Site	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Analysis of pre-defined biomarker FGF2	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	3 months	No
Primary	Analysis of pre-defined biomarker WEGF	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	3 months	No
Primary	Analysis of microRNAs	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	3 months	No