Clinical trial NCT01240850: Methotrexate as a Steroid-sparing Agent in Idiopathic Retroperitoneal Fibrosis: a Randomised, Multicenter Trial
Official title: Medical Therapy in Idiopathic Retroperitoneal Fibrosis: a Multicenter, Randomized, Controlled Trial of Prednisone vs Low-dose Prednisone Plus Methotrexate
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Idiopathic Retroperitoneal Fibrosis
Perianeurysmal Retroperitoneal Fibrosis
Chronic Periaortitis
Keywords: Retroperitoneal fibrosis
Inflammatory aneurysms
Methotrexate
Prednisone
Aorta
Lead sponsor: University of Parma
Sponsor class: OTHER
First posted: 2010-11-15
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Primary purpose: TREATMENT

Brief summary: Chronic periaortitis is a clinico-pathological entity encompassing idiopathic retroperitoneal fibrosis and perianeurysmal retroperitoneal fibrosis. The treatment of this disease is generally based on the use of glucocorticoids, which are often effective. However, prolonged steroid treatments are usually needed to achieve a sustained remission; additionally, patients frequently develop disease relapses following treatment discontinuation, therefore they may be exposed to high cumulative doses of glucocorticoids.

Preliminary data reported in the literature show that methotrexate may be effective in combination with prednisone for retroperitoneal fibrosis. In addition, methotrexate is often used as a steroid-sparing agent in different inflammatory diseases.

The aim of this study is to evaluate whether a treatment with low-dose prednisone plus methotrexate is non-inferior to conventional dose-prednisone in achieving remission in retroperitoneal fibrosis patients.


Eligibility criteria: Inclusion Criteria:

* New diagnosis of idiopathic retroperitoneal fibrosis or chronic periaortitis; written informed consent

Exclusion Criteria:

* Secondary forms of retroperitoneal fibrosis (e.g. drugs, surgery, neoplasms, infections)
* Previous medical therapy for retroperitoneal fibrosis
* Renal failure with creatinine \>2 mg/dl which proved not to be reversible after ureteral decompression
* Hypersensitivity to the study drugs
* Pregnancy
* Active infections or malignant neoplasms

Primary outcomes:
measure: Remission rate by the end of treatment
Secondary outcomes:
measure: Reduction in size of the retroperitoneal mass on CT/MRI scans
measure: Rate of post-treatment relapses
measure: Treatment-related toxicity
Other outcomes:
Locations:
city: Parma
country: Italy
facility: Nephrology Unit, University Hospital of Parma
Study officials:
affiliation: Parma University Hospital
name: Augusto Vaglio, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: van Bommel EF, Siemes C, Hak LE, van der Veer SJ, Hendriksz TR. Long-term renal and patient outcome in idiopathic retroperitoneal fibrosis treated with prednisone. Am J Kidney Dis. 2007 May;49(5):615-25. doi: 10.1053/j.ajkd.2007.02.268.
citation: Vaglio A, Palmisano A, Corradi D, Salvarani C, Buzio C. Retroperitoneal fibrosis: evolving concepts. Rheum Dis Clin North Am. 2007 Nov;33(4):803-17, vi-vii. doi: 10.1016/j.rdc.2007.07.013.
citation: Vaglio A, Salvarani C, Buzio C. Retroperitoneal fibrosis. Lancet. 2006 Jan 21;367(9506):241-51. doi: 10.1016/S0140-6736(06)68035-5.
citation: Warnatz K, Keskin AG, Uhl M, Scholz C, Katzenwadel A, Vaith P, Peter HH, Walker UA. Immunosuppressive treatment of chronic periaortitis: a retrospective study of 20 patients with chronic periaortitis and a review of the literature. Ann Rheum Dis. 2005 Jun;64(6):828-33. doi: 10.1136/ard.2004.029793.