Using Sirolimus to treat Idiopathic Retroperitoneal Fibrosis
Prospective Study of Prednisone Versus Sirolimus in the Treatment of Idiopathic Retroperitoneal Fibrosis
This study is testing if Sirolimus can help people with idiopathic retroperitoneal fibrosis by reducing the growth of fibrous tissue and lowering the need for long-term steroid use.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Peking University International Hospital Academic / other |
| Drugs / interventions | prednisone |
| Locations | 1 site (Beijing, Beijing Municipality) |
| Trial ID | NCT04047576 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the efficacy of Sirolimus, an mTOR inhibitor, in treating idiopathic retroperitoneal fibrosis, a condition characterized by the abnormal growth of fibrous tissue in the retroperitoneal space. The study aims to determine whether Sirolimus can effectively reduce disease recurrence and minimize the side effects associated with long-term corticosteroid use. Patients diagnosed with idiopathic retroperitoneal fibrosis will be enrolled, and their responses to Sirolimus will be monitored through imaging and laboratory tests. The trial is designed as a randomized controlled trial to provide high-level evidence for this treatment approach.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with idiopathic retroperitoneal fibrosis confirmed by imaging and exhibiting elevated inflammatory markers.
Not a fit: Patients with secondary retroperitoneal fibrosis or those who have recently used corticosteroids or immunosuppressants may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve outcomes for patients with idiopathic retroperitoneal fibrosis by reducing recurrence rates and reliance on corticosteroids.
How similar studies have performed: While there is limited high-level evidence for the use of immunosuppressants in this condition, the approach of using Sirolimus is novel and has not been extensively tested in this specific context.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Beijing, Beijing Municipality
- Peking University International Hospital — Beijing, Beijing Municipality, China (Recruiting)
Study contacts
- Principal investigator: Hui Gao, Doctor — Peking University International Hospital
- Study coordinator: Hui Gao, Doctor
- Email: gh841017@126.com
- Phone: 8613811833264
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.