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

NCT06160739 | Unknown

• 1 other identifier: soh-Med-23-11-16MS
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Microcystic Lymphatic Malformation; Combined Vascular Malformation; Vascular Malformations

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

group A

microcystic \& mixed lymphatic malformation

Drug: Sirolimus 1Mg Oral Tablet

group B

vascular malformation

Drug: Sirolimus 1Mg Oral Tablet

Interventions

Sirolimus 1Mg Oral Tablet DRUG

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

group A; group B

Eligibility Criteria

• Age: 6 Months - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

* 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

You may qualify if:

• 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

You may not qualify if:

• 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 .

Sponsors & Collaborators

• Sohag University: lead

Study Sites (1)

Sohag university Hospital

Sohag, Egypt

RECRUITING

Related Publications (4)

• 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.

  PMID: 29874693 BACKGROUND

• 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.

  PMID: 26148957 BACKGROUND

• 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.

  PMID: 29415296 BACKGROUND

• 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.

  PMID: 18381090 BACKGROUND

MeSH Terms

Conditions

Lymphangioma; Vascular Malformations

Interventions

Sirolimus; Tablets

Condition Hierarchy (Ancestors)

Neoplasm, Lymphatic Tissue; Neoplasms by Histologic Type; Neoplasms; Cardiovascular Abnormalities; Cardiovascular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals; Dosage Forms; Pharmaceutical Preparations

Central Study Contacts

Tarek A Abd elsalam, resident

CONTACT

01064411709; tarikahmed@med.sohag.eg

Mohamed yousef, Ass. Prof.

CONTACT

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Resident-pediatric surgery department-sohag hospital university

Study Record Dates

• First Submitted: November 29, 2023
• First Posted: December 7, 2023
• Study Start: November 20, 2023
• Primary Completion: September 1, 2024
• Study Completion: September 1, 2024
• Last Updated: December 15, 2023

Record last verified: 2023-12

Locations

EG (1)