NCT02961868

Cohort Follow-up of Patients With Renal or Craniocervical Fibromuscular Dysplasia

PROgression of FIbromuscular LEsions

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 340 (actual)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

PROFILE is a cohort study evaluating the progression of fibromuscular dysplasia lesions. This study is the prospective dimension of ARCADIA registry (ClinicalTrials.gov Identifier: NCT02884141), which aims to document phenotypic and genetic traits in patients with renal and/or cervical artery fibromuscular dysplasia.

Detailed description

Background Fibromuscular dysplasia (FMD) is a group of nonatherosclerotic, noninflammatory arterial diseases that usually involve renal and carotid arteries. Patients with FMD may present with renovascular hypertension and/or with cerebrovascular symptoms. The prevalence of FMD in hypertensive patients is estimated at 4/1000. Angiographic classification includes the multifocal type, with multiple stenoses and the 'string-of-beads' appearance that is related to medial FMD, and tubular and focal types which are not clearly related to specific histological lesions. FMD may affect one or more vascular beds and progress to more severe stenosis and to renal or cerebrovascular complications. FMD appears to be familial in 10% of cases (OMIM #135580). Renal artery FMD may progress to more severe stenosis and to renal atrophy, and/or to stenoses affecting more arteries within or outside the renal vasculature. The risk of progression as assessed from available studies was probably overestimated because documentation of progression was obtained from angiography, a procedure which is not routinely undertaken in patients with favourable clinical and biological outcomes. The disease is progressive, however, and literature stated that patients with FMD should undergo yearly duplex ultrasonography to detect progression of disease, restenosis, or loss of kidney volume. There are very few data on prognosis of patients with symptomatic carotid or vertebral artery FMD. The risk of arterial disease progression over time is unknown. The risk of ischemic stroke ranged from 0 to about 3% per year in the few studies which assessed that issue. Objectives The primary objective is to estimate the incidence and risk factors for progression of FMD lesions. This will be assessed by comparison between initial and 3 years abdominal and supra-aortic trunks vascular imaging (angiography, CT-angiography or Magnetic Resonance (MR) angiography), monitoring of downstream consequences development of lesions progression and clinical events. The secondary objectives are: * to estimate rate of genetic polymorphism that may influence disease progression or be associated with complications * to assess the frequency of multi-site FMD (common objective with the ARCADIA study) * to collect standardized clinical, radiological, and biological data in patients with FMD through a national registry (common objective with the ARCADIA study) * to organize a clinical, radiological and biological database and a biobank that will constitute a unique resource to initiate further clinical research (common objective with the ARCADIA study).

Conditions

• Fibromuscular Dysplasia

Interventions

Type	Name	Description
OTHER	Abdominal and supra-aortic trunks vascular imaging	Abdominal and supra-aortic trunks vascular imaging (angiography, CT-angiography or MR-angiography) will be performed 3 years after inclusion. This imaging will be compare to initial imaging (which is a part of usual care, not an intervention added by the study) in order to assess FMD progression.
GENETIC	Blood sampling (genetic)	A sample of blood will be taken to meet the objective of estimating the rate of genetic polymorphism that may influence disease progression or be associated with complications.
OTHER	Blood sampling (biomarkers)	A sample of blood will be taken to biomarkers analysis to meet the primary objective of assessing the risk factors for progression of FMD lesions.
OTHER	Urine sampling	A sample of urine will be taken to biomarkers analysis to meet the primary objective of assessing the risk factors for progression of FMD lesions.

Timeline

• Start date: 2009-11-01
• Primary completion: 2018-01-01
• Completion: 2018-01-01
• First posted: 2016-11-11
• Last updated: 2019-09-23

Locations

17 sites across 2 countries: Belgium, France