Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations

Vascular Malformations Clinical Trial

Official title:

Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06160739
• Other study ID #: soh-Med-23-11-16MS
• Status: Recruiting
• Start date: November 20, 2023
• Est. completion date: September 1, 2024

Study information

• Verified date: December 2023
• Source: Sohag University
• Contact: Tarek A Abd elsalam, resident
• Phone: 01064411709
• Email: tarikahmed[@]med.sohag.eg
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Lympho-vascular malformations result from errors in embryologic vasculogenesis involving capillaries, veins, arteries, lymphatics, or a combination of these. Infantile haemangiomas & Vascular malformations like : Capillary malformations & Venous malformations : they increase in size and never regress on their own. & They are generally present at birth, they enlarge in response to infection, hormonal changes or trauma . Lymphatic malformations can be classified into macrocystic (cyst diameter >1cm), microcystic (cyst diameter <1 cm), or mixed , in macrocystic lymphatic malformations, surgery and sclerotherapy are effective . Surgery of microcystic lymphatic malformations remains challenging due to their infiltrative nature & Sclerotherapy is often impossible. As especially large microcystic and mixed malformations are still a therapeutic challenge, pharmaceutical treatment as sirolimus is used in last years as main line of treatment with great efficacy.

Description:

Lympho-vascular malformations result from errors in embryologic vasculogenesis involving capillaries, veins, arteries, lymphatics, or a combination of these. Infantile haemangiomas & Vascular malformations like : Capillary malformations & Venous malformations : they increase in size and never regress on their own. & They are generally present at birth, they enlarge in response to infection, hormonal changes or trauma . Lymphatic malformations can be classified into macrocystic (cyst diameter >1cm), microcystic (cyst diameter <1 cm), or mixed , in macrocystic lymphatic malformations, surgery and sclerotherapy are effective . Surgery of microcystic lymphatic malformations remains challenging due to their infiltrative nature & Sclerotherapy is often impossible. As especially large microcystic and mixed malformations are still a therapeutic challenge, pharmaceutical treatment as sirolimus is used in last years as main line of treatment with great efficacy. Sirolimus is a natural macrolide isolated from a bacteria strain of the Streptomyces genus & Streptomyces hygroscopicus . It was initially used as an antibiotic and antifungal agent, subsequent studies have revealed impressive cytostatic, antiproliferative, and immunosuppressive properties . Sirolimus not only prevents the growth of abnormal lymphatics but also induces the partial regression of lesions, without apparent effects on normal lymphatics .

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: September 1, 2024
• Est. primary completion date: September 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 12 Years

Eligibility

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 .

Related Conditions & MeSH terms

• Congenital Abnormalities
• Lymphangioma
• Lymphatic Abnormalities
• Microcystic Lymphatic Malformation
• Vascular Malformations

Intervention

Drug:
• Sirolimus 1Mg Oral Tablet
patients with Microcystic , Mixed Lymphatic and Vascular Malformations will be given sirolimus 1 mg oral tab for 3-6 months with follow up of lesion size by clinical exam & ultrasound & MRI to compare lesion size before & after use of the drug with observation of potential side effects & after exclusion of it before use of the drug

Locations

Country	Name	City	State
Egypt	Sohag university Hospital	Sohag

Sponsors (1)

Lead Sponsor	Collaborator
Sohag University

Country where clinical trial is conducted

Egypt, 

References & Publications (4)

Adams DM, Wentzel MS. The role of the hematologist/oncologist in the care of patients with vascular anomalies. Pediatr Clin North Am. 2008 Apr;55(2):339-55, viii. doi: 10.1016/j.pcl.2008.01.007. — View Citation

Muller-Wille R, Wildgruber M, Sadick M, Wohlgemuth WA. Vascular Anomalies (Part II): Interventional Therapy of Peripheral Vascular Malformations. Rofo. 2018 Feb 7. doi: 10.1055/s-0044-101266. Online ahead of print. — View Citation

Sadick M, Muller-Wille R, Wildgruber M, Wohlgemuth WA. Vascular Anomalies (Part I): Classification and Diagnostics of Vascular Anomalies. Rofo. 2018 Sep;190(9):825-835. doi: 10.1055/a-0620-8925. Epub 2018 Jun 6. — View Citation

Vlahovic AM, Vlahovic NS, Haxhija EQ. Sirolimus for the Treatment of a Massive Capillary-Lymphatico-Venous Malformation: A Case Report. Pediatrics. 2015 Aug;136(2):e513-6. doi: 10.1542/peds.2014-3469. Epub 2015 Jul 6. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	changes in size of the lesion	efficacy of sirolimus in decreasing size of the lesion over time , by clinical exam of size lesion & measurement of size of leesion by ultrasound & MRI	1 year