Idiopathic retroperitoneal fibrosis (IRF) MedDRA version: 20.1 Level: LLT Classification code 10021244 Term: Idiopathic retroperitoneal fibrosis System Organ Class: 100000004856
Conditions
Interventions
Pharmaceutical Form: Tablet
INN or Proposed INN: Prednisone
CAS Number: 53-03-2
Sponsors
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (APHP)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Patient over 18 years old • Diagnosis of active idiopathic retroperitoneal fibrosis (IRF) defined by the association of: o Related-disease symptoms (Appendix 17.2) or elevated CRP level (>20 mg/l) AND o Retroperitoneal peri-aortic mass that surrounds the abdominal vessels on CT-scan Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 21
Exclusion criteria
Exclusion criteria: • Secondary retroperitoneal fibrosis including drug-related retroperitoneal fibrosis, active infections (such as tuberculosis) or malignancies, systemic vasculitis (such as ANCA-associated vasculitis), Erdheim-Chester disease (Appendix 17.3), • Relapse of an already treated IRF, • Contraindication to perform FDG-PET/CT, • Contraindication to perform CT scan with injection of contrast agent, • Contraindication to treatment by prednisone • Active infection, • Acute or chronic liver disease that is deemed sufficiently severe to impair their ability to participate in the trial, • Active or history of malignancy in last 5 years. Individuals with squamous cell or basal cell skin carcinomas and individuals with cervical carcinoma in situ may be enrolled if they have received curative surgical treatment, • Serum creatinine level greater than 400 µmol/L that cannot be attributed to underlying IRF, • Live vaccination received from 4 weeks before inclusion, • Inhaled glucocorticoids (except for patients with documented asthma), • Any previous treatment with rituximab, methotrexate, alemtuzumab, cyclophosphamide, azathiorpine, mycophenolate mofetil, infliximab, adalimumab, etanercept within the past 3 months, • Pregnancy or breastfeeding, • Non-affiliation to a social security regime, • Subject deprived of freedom, subject under a legal protective measure • Refusal to participate
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the cumulative IRF relapse rate 12 months after discontinuation of steroids. ;Secondary Objective: 1.To analyze the characteristics of hypermetabolism of IRF in FDG-PET/CT and their evolution at diagnosis, remission (M9), M21 and relapse, 2.To assess the performance of hypermetabolism of IRF in FDG-PET/CT for diagnosis of disease activity, 3.To compare at M21 the corticosteroids therapy – related adverses events between patients who continue or discontinue the treatment at M9. ;Primary end point(s): The primary endpoint is the cumulate IRF relapse rate measured at the end of the study (M21). The diagnosis of IRF relapse is based on the association of a clinical or biological criterion with a radiological criterion (i.e. composite criteria): -Clinical or biological criteria orecurrent or new-onset disease related symptoms oincrease in CRP >20mg/L without other cause -And a Radiological criterion oincreased of retroperitoneal fibrosis size as compared with the CT scan performed at remission. The primary endpoint will be centrally adjudicated. ;Timepoint(s) of evaluation of this end point: M21 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1a. Visual grades of retroperitoneal fibrosis FDG uptake as compared to liver FDG uptake (which consist of one item that yields a score of 0 to III), maximal standardized uptake value (SUVmax) within the retroperitoneal fibrosis (regions of interest- ROI) at diagnosis (M0), remission (M9), M21 and relapse, 1b. Metabolic volume (i.e. ratio of metabolically active volume (MAV) to global lesion volume) of retroperitoneal fibrosis FDG uptake at diagnosis (M0), remission (M9), M21 and relapse, 2. Diagnostic performance of SUVmax and MAV (area under the curve (AUC) and performance values for the Youden index) for the disease activity, 3. Frequency of diabetes, severe infection, osteoporotic fracture and major cardiovascular events 12 months after remission (M21). Serious cardiovascular adverse events are defined as a composite of nonfatal stroke, nonfatal myocardial infarction, and cardiovascular death and will be assessed at M12,M15 and M21 ;Timepoint(s) of evaluation of this end point: M9, M21 or relapse | — |
Countries
France
Contacts
Public ContactDRCI - Hôpital Saint Louis
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (APHP)
Outcome results
None listed