Urine Sample Collection From FOP Patients

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT02066324
First received: February 5, 2014
Last updated: May 27, 2014
Last verified: May 2014
  Purpose

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.


Condition
Fibrodysplasia Ossificans Progressiva

Study Type: Observational
Official Title: Urine Sample Collection From Patients With Fibrodysplasia Ossificans Progressiva (FOP) for Biomarker Analysis

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Analysis of pre-defined biomarker FGF2 [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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


Biospecimen Retention:   Samples Without DNA

Urine samples


Estimated Enrollment: 20
Study Start Date: November 2013
Estimated Study Completion Date: May 2014
Estimated Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
  Eligibility

Ages Eligible for Study:   5 Years to 35 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Patients with Fibrodysplasia Ossificans Progressiva.

Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02066324

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals

Locations
United States, Pennsylvania
Novartis Investigative Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT02066324     History of Changes
Other Study ID Numbers: CPJMR0062203
Study First Received: February 5, 2014
Last Updated: May 27, 2014
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Myositis Ossificans
Myositis
Muscular Diseases
Musculoskeletal Diseases

ClinicalTrials.gov processed this record on August 19, 2014