MedDataX Logo

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

NCT ID: NCT06160739

Last Updated: 2023-12-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

10 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-11-20

Study Completion Date

2024-09-01

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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 .

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Microcystic Lymphatic Malformation Combined Vascular Malformation Vascular Malformations

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

group A

microcystic \& mixed lymphatic malformation

Sirolimus 1Mg Oral Tablet

Intervention Type 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 B

vascular malformation

Sirolimus 1Mg Oral Tablet

Intervention Type 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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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 .
Minimum Eligible Age

6 Months

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Sohag University

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Tarek Ahmed Abd Elsalam

Resident-pediatric surgery department-sohag hospital university

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Sohag university Hospital

Sohag, , Egypt

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Egypt

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Tarek A Abd elsalam, resident

Role: CONTACT

Phone: 01064411709

Email: [email protected]

Mohamed yousef, Ass. Prof.

Role: CONTACT

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Magdy M Amin, professor

Role: primary

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type BACKGROUND
PMID: 29874693 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 26148957 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 29415296 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18381090 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

soh-Med-23-11-16MS

Identifier Type: -

Identifier Source: org_study_id