Efficacy and Safety of Sirolimus in the Management of pediatric Vascular Tumors and Malformations- A Prospective Study
Overview
- Phase
- Phase 2
- Status
- Not yet recruiting
- Sponsor
- Dr Saurav Sharma(self)
- Enrollment
- 30
- Locations
- 1
- Primary Endpoint
- Reduction in size of vascular anomaly
Overview
Brief Summary
Vascular anomalies represent a broad spectrum of disorders ranging from a tiny “birthmark†to life threatening entities. They are difficult to treat clinical entities causing significant morbidity and functional impairment. Currently available treatment modalities like sclerotherapy/ embolization/ surgical excision are seldom curative with high recurrence rates. Medical management can be a way forward in this regard. Sirolimus has shown promising results in the past studies as an effective drug in management of vascular anomalies leading to decrease in size and improvement in functional status of the patients. Thus our research hypothesis is that the use of sirolimus will improve clinical symptoms, reduce the size, and improve the quality of life in symptomatic patients with vascular anomalies in 70% of patients after 3 months of use. This will be a single arm trial to be conducted at Department of paediatrics, AIIMS Delhi for a period of 2 years from july 2024 to july 2026. The primary objective will be to assesss the efficacy of Sirolimus in decreasing the size of vascular anomalies. The secondary objectives will be to assess the efficacy of sirolimus in decreasing the signs and symptoms of vascular anomalies, in improving the quality of life of the patients; and to assess its safety during the treatment. Additionally, data regarding the distribution of types of vascular anomalies will be recorded.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Masking
- None
Eligibility Criteria
- Ages
- 1.00 Day(s) to 18.00 Year(s) (—)
- Sex
- All
Inclusion Criteria
- •Children aged ≤18 years diagnosed with Newly diagnosed vascular malformation.
- •2.Patients with benign vascular tumors who are refractory to 3 months of conventional therapy.
- •3.Patients with locally aggressive vascular tumors 4.Written informed consent.
Exclusion Criteria
- •1.Children less than 1 year of age with infantile hemangioma.
- •2.Known hypersensitivity to sirolimus 3.Concurrent nephrotoxic agent use 4.CYP3A4 inducers or inhibitors while receiving sirolimus 5.Baseline cytopenias (ANC less than 500/mm3, Hb less than 8 gm/dl, and platelet less than 50,000/mm3).
- •6.Baseline SGOT/SGPT more than 5 times ULN or serum bilirubin more than 1.5 times ULN 7.Abnormal lipid profiles: triglycerides (TG) more than 130mg/dl, cholesterol more than 170 mg/dl.
Outcomes
Primary Outcomes
Reduction in size of vascular anomaly
Time Frame: 6 month
Secondary Outcomes
- Improvement in clinical signs and symptoms of vascular mnalformation.(3 month)
- Improvement in quality of life of the patients.(3 month)
- Evaluation of side effect of the Sirolimus during the treatment.(3 month)
- Distribution of types of vascular anomaly.(at the end of study.)
Investigators
Dr Saurav Sharma
AIIMS, New Delhi