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Study of Sirolimus in Idiopathic Retroperitoneal Fibrosis

ConditionRetroperitoneal Fibrosis
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorPeking University International Hospital

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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).

  • 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.

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: sirolimus groupExperimental treatment 2 interventions
Group B: corticosteroid groupActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

Recurrence rate

Time frame
through study completion, an average of 1 year

Secondary outcomes

1

Treatment failure rate

Time frame
12 weeks of treatment
2

Cumulative dose of prednisone

Time frame
48 weeks
3

Size of retroperitoneal fibrotic mass

Time frame
48 weeks

Other outcomes

Sponsors and contacts

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Peking University International Hospital

Lead sponsor

Source ClinicalTrials.gov
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