Using Sirolimus to treat lymphatic and vascular malformations in children
Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations
This study is testing if Sirolimus can help children aged 6 months to 12 years with hard-to-treat lymphatic malformations that haven’t improved with other treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 10 (estimated) |
| Ages | 6 Months to 12 Years |
| Sex | All |
| Sponsor | Sohag University Academic / other |
| Locations | 1 site (Sohag) |
| Trial ID | NCT06160739 on ClinicalTrials.gov |
What this trial studies
This observational study investigates the effectiveness of Sirolimus, an oral medication, in treating microcystic and mixed lymphatic malformations in children aged 6 months to 12 years. These malformations are challenging to treat surgically and often do not respond to other treatments. Sirolimus has shown promise in preventing the growth of abnormal lymphatics and inducing regression of lesions. The study aims to evaluate the outcomes of patients who have not benefited from previous treatments.
Who should consider this trial
Good fit: Ideal candidates are children aged 6 months to 12 years diagnosed with microcystic or mixed lymphatic malformations who have not responded to other treatments.
Not a fit: Patients with macrocystic lymphatic malformations or high-flow vascular malformations will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new effective option for managing difficult lymphatic and vascular malformations in children.
How similar studies have performed: Previous studies have indicated that Sirolimus can be effective in treating similar conditions, suggesting a potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * from age 6 months to 12 years , not before 6 months to complete their vaccination program \& maturation of hepatic enzymes . * Patients diagnosed with microcystic \& mixed Lymphatic malformations . * After failure of other lines of treatment as regard propranolol , steroid for infantile haemangiomas \& Vascular malformations and lymphovascular malformations . * After failure of surgical excision \& injection of bleomycin of Lympho-vascular malformations Exclusion Criteria: * Macrocystic Lymphatic malformations \& high flow vascular malformations like arterio-venous malformations . * An active infection that requires systemic treatment during the attack . * Side effects of the drug as ( history of an allergic reaction to sirolimus or patients who develop severe allergic reaction to drug during treatment , hyperlipidemia , leucopenia , etc... ) * Chronic liver or kidney disease or on chronic drug treatment as (steroids, interferon or chemotherapeutic agents) . * An immunodeficiency condition such as a human immunodeficiency viral infection or primary immunodeficiency disease. * Patients who received drug less than 6 months duration .
Where this trial is running
Sohag
- Sohag university Hospital — Sohag, Egypt (Recruiting)
Study contacts
- Study coordinator: Tarek A Abd elsalam, resident
- Email: tarikahmed@med.sohag.eg
- Phone: 01064411709
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.