Methotrexate as a Steroid-sparing Agent in Idiopathic Retroperitoneal Fibrosis: a Randomised, Multicenter Trial

NCT01240850 | Unknown Phase 3

• 2 other identifiers: FARM79BKPY2008-007353-12
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 1

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.

Conditions

Idiopathic Retroperitoneal Fibrosis; Perianeurysmal Retroperitoneal Fibrosis; Chronic Periaortitis

Keywords

Retroperitoneal fibrosis; Inflammatory aneurysms; Methotrexate; Prednisone; Aorta

Outcome Measures

Primary Outcomes (1)

• Remission rate by the end of treatment

Secondary Outcomes (3)

• Reduction in size of the retroperitoneal mass on CT/MRI scans

• Rate of post-treatment relapses

• Treatment-related toxicity

Study Arms (2)

Prednisone

ACTIVE COMPARATOR

Drug: Prednisone

Methotrexate+Prednisone

EXPERIMENTAL

Drug: Methotrexate+Prednisone

Interventions

Methotrexate+Prednisone DRUG

Methotrexate will be added at a dose of 0.3 mg/kg/week (maximum dose, 20 mg/week, orally) to a reduced-dose prednisone regimen

Methotrexate+Prednisone

Prednisone DRUG

Prednisone

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Parma: lead

Study Sites (1)

Nephrology Unit, University Hospital of Parma

Parma, Parma, 43126, Italy

RECRUITING

Related Publications (5)

• 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.

  PMID: 17472843 BACKGROUND

• 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.

  PMID: 18037118 BACKGROUND

• Vaglio A, Salvarani C, Buzio C. Retroperitoneal fibrosis. Lancet. 2006 Jan 21;367(9506):241-51. doi: 10.1016/S0140-6736(06)68035-5.

  PMID: 16427494 BACKGROUND

• 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.

  PMID: 15897305 BACKGROUND

• Peyronel F, Palmisano A, Maritati F, Alberici F, Urban ML, Gianfreda D, Rossi GM, Fenaroli P, Bettiol A, Moroni G, Vaglio A. Methotrexate and low-dose prednisone in idiopathic retroperitoneal fibrosis: a randomised clinical trial. J Autoimmun. 2025 Dec;157:103487. doi: 10.1016/j.jaut.2025.103487. Epub 2025 Sep 19.

  PMID: 40974866 DERIVED

MeSH Terms

Conditions

Retroperitoneal Fibrosis

Interventions

Prednisone

Condition Hierarchy (Ancestors)

Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Augusto Vaglio, MD, PhD

  Parma University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Carlo Buzio, MD

CONTACT

+39 0521 702345; carlo.buzio@unipr.it

Augusto Vaglio, MD, PhD

CONTACT

+39 0521 702345; augusto.vaglio@virgilio.it

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: November 12, 2010
• First Posted: November 15, 2010
• Study Start: May 1, 2007
• Primary Completion: May 1, 2012
• Study Completion: December 1, 2013
• Last Updated: November 15, 2010

Record last verified: 2010-11

Locations

IT (1)