Study of Sirolimus in Idiopathic Retroperitoneal Fibrosis

NCT04047576 · tracked via the Priya Life Science China tracker

Sponsor

Peking University International Hospital

Phase

Phase 2/3

Started

2018-07-01

Last updated

2026-09-22

Condition(s) studied

Retroperitoneal Fibrosis

Investigational drug(s) / intervention(s)

Sirolimus →Corticosteroid →

Sirolimus: The efficacy is evaluated at 12 weeks, and treatment will be adjusted according to the control of disease and adverse effects.For experimental group, if a patient is assessed as treatment failure (TS), the patient should be withdrawn from the study and receive rescue treatment. Whereas, a patient would be transferred to the control group if he/ she cann't stand the side effects of sirolimus but not serious adverse event (SAE).

Corticosteroid: The efficacy is evaluated at 12 weeks, and treatment will be adjusted according to the control of disease and adverse effects. For active comparator group, if a patient is assessed as treatment failure (TF), the patient should be withdrawn from the study and receive rescue treatment.

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

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: 1. Idiopathic retroperitoneal fibrosis diagnosed on CT or MRI. For patients with suspected secondary retroperitoneal fibrosis or atypical idiopathic retroperitoneal fibrosis suggested by imaging, idiopathic retroperitoneal fibrosis should be confirmed by puncture biopsy 2. Increased ESR and CRP levels caused by this disease and/or active lesions suggested on imaging Exclusion Criteria: 1. Secondary retroperitoneal fibrosis 2. Having used corticosteroid (equivalent to \>10 mg per day of prednisone), immunosuppressant, or biologic within 3 months prior to enrollment 3. Having any contraindication of prednisone or sirolimus, or allergy to sirolimus, or having experienced serious adverse reactions from the previous use of any of the above drugs 4. Massive proteinuria (24-hour urine protein quantitation ≥3 g), moderate-to-severe anemia (hemoglobin \<90 g/L), agranulocytosis (white blood cell count \<1.5×10\^9/L or neutrophil count \<0.5×10\^9/L), platelet count \<50×10\^9/L, interstitial pneumonia 5. Uncontrollable diabetes, hypertension, hyperlipidemia, infection, or heart failure, or other serious complications 6. Malignancy 7. Pregnancy or need for pregnancy in the near future 8. Unable to adhere to follow-up or refuses to provide consent

Primary outcome measure(s)

Recurrence rate — through study completion, an average of 1 year

Trial sites (1)

Facility	City	Region	Status
Peking University International Hospital	Beijing	Beijing Municipality

More Peking University International Hospital trials in China

Neoadjuvant Chemotherapy Combined With Targeted Treatment in High-risk Retroperitoneal Sarcoma Enrolling by invitation

Other trials for the same condition

Distinguishing Retroperitoneal Fibrosis and Sarcoma from Other Retroperitoneal Diseases Via Radiomics Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04047576 on ClinicalTrials.gov ↗ ← All trials in China