A Prospective Study of Cyclophosphamide Treatment for Idiopathic Retroperitoneal Fibrosis

NCT ID: NCT04762810

Last Updated: 2021-03-19

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE4
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-03
• Study Completion Date: 2023-02-10

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.

Detailed Description

Idiopathic retroperitoneal fibrosis is a chronic disease that requires treatment for a few years. Patients with this disease respond well to glucocorticoid therapy, so you need to take glucocorticoid medication once the diagnosis is established. In addition, most patients need to use immunosuppressants when taking glucocorticoids. At present, the most commonly used immunosuppressants in the world are cyclophosphamide, azathioprine, methotrexate and mycophoranate, etc. The function of the above drugs is to improve the disorder of the immune system function, in order to restore the normal immune state, so that the inflammation and swelling of the affected organs disappear, restore the normal function. It should be emphasized that if the use of hormones and immunosuppressant is not standard or stopped too early, it can lead to disease recurrence. Therefore, after the disease is under control, long-term adherence to medication is needed to maintain a stable state of the disease.

At present, there is no clear treatment guideline for idiopathic retroperitoneal fibrosis in the world, and the addition of immunosuppressant is also out of debate. Therefore, a comprehensive evaluation of its pathogenesis, clinical characteristics and treatment response is needed.

Conditions

Idiopathic Retroperitoneal Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cyclophosphamide and Glucocorticoids

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.

Group Type: EXPERIMENTAL

Cyclophosphamide and glucocorticoids

Intervention Type: 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.

Glucocorticoids monotherapy

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.

Group Type: ACTIVE_COMPARATOR

Glucocorticoids

Intervention Type: 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.

Interventions

Cyclophosphamide and glucocorticoids

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.

Intervention Type: DRUG

Glucocorticoids

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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.

Exclusion Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking Union Medical College Hospital

OTHER

Sponsor Role: lead

Responsible Party

Wen Zhang

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Countries

China

Facility Contacts

Yunyun Fei

Role: primary

feiyunyun2013@sina.com

+8613681125226

Wen Zhang

Role: backup

zhangwen91@sina.com

References

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Reference Type: BACKGROUND

PMID: 11247880 (View on PubMed)

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

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PMID: 16427494 (View on PubMed)

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Reference Type: BACKGROUND

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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.

Reference Type: BACKGROUND

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Reference Type: BACKGROUND

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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.

Reference Type: BACKGROUND

PMID: 27876167 (View on PubMed)

Other Identifiers

Cyclophosphamide for IRPF

Identifier Type: -

Identifier Source: org_study_id