Study of Sirolimus in Idiopathic Retroperitoneal Fibrosis

Phase

2/3

Condition

Scar Tissue

Treatment

Corticosteroid

Sirolimus

Clinical Study ID

NCT04047576

Sirolimus for RPF

Ages > 18
All Genders

Study Summary

Retroperitoneal fibrosis refers to a group of diseases characterized by hyperplasia of the fibrosclerotic tissues in the retroperitoneal space, which can compress the surrounding ureters and inferior vena cava and cause serious complications such as aortic aneurysm, renal failure, and even death. The lesion is diffuse and difficult to resect. corticosteroid is the first-line medication, but the recurrence rate of the disease is high, especially after dose reduction of corticosteroid. Therefore, the combined use of immunosuppressants is very important in preventing disease recurrence and reducing the toxic and side effects of long-term corticosteroid. Sirolimus plays dual roles in inhibiting lymphocyte activation and fibroblast proliferation. It is inferred from its mechanism that sirolimus is a good potential treatment option for idiopathic retroperitoneal fibrosis. Therefore, we conducted this RCT on patients with idiopathic retroperitoneal fibrosis to determine the efficacy and safety of sirolimus.

Eligibility Criteria

Inclusion

Inclusion Criteria:

Idiopathic retroperitoneal fibrosis diagnosed on CT or MRI. For patients withsuspected secondary retroperitoneal fibrosis or atypical idiopathic retroperitonealfibrosis suggested by imaging, idiopathic retroperitoneal fibrosis should beconfirmed by puncture biopsy
Increased ESR and CRP levels caused by this disease and/or active lesions suggestedon imaging

Exclusion

Exclusion Criteria:

Secondary retroperitoneal fibrosis
Having used corticosteroid (equivalent to >10 mg per day of prednisone),immunosuppressant, or biologic within 3 months prior to enrollment
Having any contraindication of prednisone or sirolimus, or allergy to sirolimus, orhaving experienced serious adverse reactions from the previous use of any of theabove drugs
Massive proteinuria (24-hour urine protein quantitation ≥3 g), moderate-to-severeanemia (hemoglobin <90 g/L), agranulocytosis (white blood cell count <1.5×10^9/L orneutrophil count <0.5×10^9/L), platelet count <50×10^9/L, interstitial pneumonia
Uncontrollable diabetes, hypertension, hyperlipidemia, infection, or heart failure,or other serious complications
Malignancy
Pregnancy or need for pregnancy in the near future
Unable to adhere to follow-up or refuses to provide consent

Study Design

Corticosteroid

2/3

July 01, 2018

December 31, 2026

Study Description

Retroperitoneal fibrosis refers to a group of diseases characterized by hyperplasia of the fibrosclerotic tissues in the retroperitoneal space, which mostly involve the abdominal aorta and iliac artery distal to the kidneys. The hyperplastic tissues can compress the surrounding ureters and inferior vena cava and cause serious complications such as aortic aneurysm, renal failure, and even death. There is no clear boundary between the lesion and its surrounding tissues. The lesion is diffuse and difficult to resect. corticosteroid is the first-line medication, but the recurrence rate of the disease is high, especially after dose reduction of corticosteroid. Therefore, the combined use of immunosuppressants is very important in preventing disease recurrence and reducing the toxic and side effects of long-term high- and medium-dose corticosteroid. In recent years, as the immunological characteristics of retroperitoneal fibrosis have gradually been recognized, some rheumatologists and immunologists have attempted to use immunosuppressants commonly used for autoimmune diseases in this population, including biologics. However, high-level evidences of evidence-based medicine such as randomized controlled trials (RCTs) were still lacking. Sirolimus plays dual roles in inhibiting T lymphocyte activation and fibroblast proliferation. It is inferred from its mechanism that sirolimus is a good potential treatment option for idiopathic retroperitoneal fibrosis. Therefore, we conducted this RCT on patients with idiopathic retroperitoneal fibrosis to determine the efficacy and safety of sirolimus.

Connect with a study center

Peking University International Hospital
Beijing, Beijing 100000
China
Active - Recruiting

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