A Prospective Study of Cyclophosphamide Treatment for Idiopathic Retroperitoneal Fibrosis

NCT04762810 | Unknown Phase 4 DMC

• 1 other identifier: Cyclophosphamide for IRPF
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

This prospective, interventional, controlled study is aimed to evaluate the efficacy and safety of cyclophosphamide in the treatment of idiopathic retroperitoneal fibrosis, which refers to the chronic nonspecific inflammation of retroperitoneal fascia and adipose tissue that gradually evolves into fibroproliferative disease.

Conditions

Idiopathic Retroperitoneal Fibrosis

Outcome Measures

Primary Outcomes (1)

• remission

  Patients achieve remission in symptoms and laboratory tets or radiologic images and have a low diseae activity after treatment.

  5 years

Secondary Outcomes (1)

• relapse

  5 years

Other Outcomes (1)

• adverse events

  5 years

Study Arms (2)

Cyclophosphamide and Glucocorticoids

EXPERIMENTAL

Through the selective depletion of proliferating lymphocytes, cyclophosphamide has an inhibitory effect on both humoral and cellular immunity. Cyclophosphamide 50mg per day for 6 months and decrease to 50mg Qod for 6 months.

Drug: Cyclophosphamide and glucocorticoids

Glucocorticoids monotherapy

ACTIVE COMPARATOR

Prednisone/prednisolone: started at 0.6-0. 8mg/kg.d for 2 to 4 weeks, tapered at 5mg per 1-2 weeks before 15mg per day, and tapered at 2.5-5mg per 2 weeks to equal to or less than 5mg per day in 6 months.

Drug: Glucocorticoids

Interventions

Cyclophosphamide and glucocorticoids DRUG

Prednisone/prednisolone: started at 0.6-0. 8mg/kg.d for 2 to 4 weeks, tapered at 5mg per 1-2 weeks before 15mg per day, and tapered at 2.5-5mg per 2 weeks to equal to or less than 5mg per day in 6 months. Cyclophosphamide 50mg per day for 6 months and decrease to 50mg Qod for 6 months.

Cyclophosphamide and Glucocorticoids

Glucocorticoids DRUG

Steroids have fast onset of action and multiple anti-inflammatory effects. Prednisone/prednisolone: started at 0.6-0. 8mg/kg.d for 2 to 4 weeks, tapered at 5mg per 1-2 weeks before 15mg per day, and tapered at 2.5-5mg per 2 weeks to equal to or less than 5mg per day in 6 months.

Glucocorticoids monotherapy

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• age 18-75 diagnosed as IRPF. The diagnosis of idiopathic retroperitoneal fibrosis is based on the following aspects :(1) the swelling of retroperitoneal tissue , which seems neoplastic; (2) A large number of lymphocytes proliferated and infiltrated in the affected tissues and organs, and tissues showed inflammation, fibrosis and sclerosis, in which IgG4-positive cells accounted for less than 50% of plasma cells; (3) Increased inflammatory markers, such as ESR and CRP; (4) Good response to glucocorticoid therapy.

You may not qualify if:

• malignancy retroperitoneal fibrosis secondary to other diseases or drugs or abdominal surgeries.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100032, China

RECRUITING

Related Publications (10)

• Marzano A, Trapani A, Leone N, Actis GC, Rizzetto M. Treatment of idiopathic retroperitoneal fibrosis using cyclosporin. Ann Rheum Dis. 2001 Apr;60(4):427-8. doi: 10.1136/ard.60.4.427.

  PMID: 11247880 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

• Jagadesham VP, Scott DJ, Carding SR. Abdominal aortic aneurysms: an autoimmune disease? Trends Mol Med. 2008 Dec;14(12):522-9. doi: 10.1016/j.molmed.2008.09.008. Epub 2008 Nov 6.

  PMID: 18980864 BACKGROUND

• Qian Q, Kashani KB, Miller DV. Ruptured abdominal aortic aneurysm related to IgG4 periaortitis. N Engl J Med. 2009 Sep 10;361(11):1121-3. doi: 10.1056/NEJMc0905265. No abstract available.

  PMID: 19741239 BACKGROUND

• Scheel PJ Jr, Feeley N, Sozio SM. Combined prednisone and mycophenolate mofetil treatment for retroperitoneal fibrosis: a case series. Ann Intern Med. 2011 Jan 4;154(1):31-6. doi: 10.7326/0003-4819-154-1-201101040-00005.

  PMID: 21200036 BACKGROUND

• Vaglio A, Palmisano A, Alberici F, Maggiore U, Ferretti S, Cobelli R, Ferrozzi F, Corradi D, Salvarani C, Buzio C. Prednisone versus tamoxifen in patients with idiopathic retroperitoneal fibrosis: an open-label randomised controlled trial. Lancet. 2011 Jul 23;378(9788):338-46. doi: 10.1016/S0140-6736(11)60934-3. Epub 2011 Jul 4.

  PMID: 21733570 BACKGROUND

• Binder M, Uhl M, Wiech T, Kollert F, Thiel J, Sass JO, Walker UA, Peter HH, Warnatz K. Cyclophosphamide is a highly effective and safe induction therapy in chronic periaortitis: a long-term follow-up of 35 patients with chronic periaortitis. Ann Rheum Dis. 2012 Feb;71(2):311-2. doi: 10.1136/annrheumdis-2011-200148. Epub 2011 Aug 22. No abstract available.

  PMID: 21859695 BACKGROUND

• Alberici F, Palmisano A, Urban ML, Maritati F, Oliva E, Manenti L, Ferretti S, Cobelli R, Buzio C, Vaglio A. Methotrexate plus prednisone in patients with relapsing idiopathic retroperitoneal fibrosis. Ann Rheum Dis. 2013 Sep 1;72(9):1584-6. doi: 10.1136/annrheumdis-2013-203267. Epub 2013 May 21. No abstract available.

  PMID: 23696631 BACKGROUND

• Fatima J, Gota C, Clair DG, Billings SD, Gornik HL. Inflammatory abdominal aortic aneurysm with retroperitoneal fibrosis. Circulation. 2014 Oct 7;130(15):1300-2. doi: 10.1161/CIRCULATIONAHA.114.010173. No abstract available.

  PMID: 25287771 BACKGROUND

• Fernando A, Pattison J, Horsfield C, D'Cruz D, Cook G, O'Brien T. [18F]-Fluorodeoxyglucose Positron Emission Tomography in the Diagnosis, Treatment Stratification, and Monitoring of Patients with Retroperitoneal Fibrosis: A Prospective Clinical Study. Eur Urol. 2017 Jun;71(6):926-933. doi: 10.1016/j.eururo.2016.10.046. Epub 2016 Nov 18.

  PMID: 27876167 BACKGROUND

MeSH Terms

Conditions

Retroperitoneal Fibrosis

Interventions

Cyclophosphamide; Glucocorticoids

Condition Hierarchy (Ancestors)

Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 17, 2021
• First Posted: February 21, 2021
• Study Start: January 3, 2020
• Primary Completion: February 10, 2023
• Study Completion: February 10, 2023
• Last Updated: March 19, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)