Study of Sirolimus in Idiopathic Retroperitoneal Fibrosis

NCT04047576 | Recruiting Phase 2

• 1 other identifier: Sirolimus for RPF
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Retroperitoneal Fibrosis

Keywords

mTOR inhibitor; Sirolimus; Retroperitoneal Fibrosis

Outcome Measures

Primary Outcomes (1)

• Recurrence rate

  through study completion, an average of 1 year

Secondary Outcomes (3)

• Treatment failure rate

  12 weeks of treatment

• Cumulative dose of prednisone

  48 weeks

• Size of retroperitoneal fibrotic mass

  48 weeks

Study Arms (2)

sirolimus group

EXPERIMENTAL

Sirolimus: 2 mg/day for the first 3 days and 1 mg/day thereafter. The plasma drug concentration was monitored at 14 days, 12 weeks, and 48 weeks of medication to maintain a plasma drug concentration of 4-15 ug/L. Prednisone: 0.8 mg/kg/d (maximum dose: 60 mg/d), reduced by 5 mg every 14 days, by 2.5 mg every 2 weeks after 30 mg/d until discontinuation.

Drug: Sirolimus; Drug: Corticosteroid

corticosteroid group

ACTIVE COMPARATOR

Prednisone: 0.8 mg/kg/d (maximum dose: 60 mg/d), reduced by 5 mg every 14 days, by 2.5 mg every 2 weeks after 30 mg/d until 5-7.5 mg/d.

Drug: Corticosteroid

Interventions

Sirolimus DRUG

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).

sirolimus group

Corticosteroid DRUG

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.

Also known as: Prednisone

corticosteroid group; sirolimus group

Eligibility Criteria

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

You may qualify if:

• 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
• Increased ESR and CRP levels caused by this disease and/or active lesions suggested on imaging

You may not qualify if:

• 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, or having experienced serious adverse reactions from the previous use of any of the above drugs
• 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
• 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

Sponsors & Collaborators

• Peking University International Hospital: lead

Study Sites (1)

Peking University International Hospital

Beijing, Beijing Municipality, 100000, China

RECRUITING

MeSH Terms

Conditions

Retroperitoneal Fibrosis

Interventions

Sirolimus; Adrenal Cortex Hormones; Prednisone

Condition Hierarchy (Ancestors)

Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Hui Gao, Doctor

  Peking University International Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Hui Gao, Doctor

CONTACT

8613811833264; gh841017@126.com

Zhan-guo Li, Professor

CONTACT

8610-88324372; li99@bjmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 25, 2019
• First Posted: August 6, 2019
• Study Start: July 1, 2018
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: June 4, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)