Microcystic Lymphatic MalformationNCT06160739
Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations
An observational study of Microcystic Lymphatic Malformation and Combined Vascular Malformation, run by Sohag University at 1 site in Egypt.
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. Read more about this study
- Who is it for?
- Ages 6 months to 12 years
- Conditions the study is for
- Microcystic Lymphatic Malformation, Combined Vascular Malformation, Vascular Malformations
- What is being tested?
- Sirolimus 1Mg Oral Tablet
- Type of treatment
- Pill or oral medicine
- How many people
- 10 participants
- Where
- 1 siteEgypt
- Study dates
- 20 Nov 2023to 1 Sep 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 12 years
- All sexes
- No healthy volunteers
- Child
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
Who may be able to join
- 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
Who cannot join
- 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 .
Trial locations and contacts
1 site in 1 country.
Mohamed yousef, Ass. Prof. contact
Egypt1 site, 1 recruiting
- Recruiting
Sohag university Hospital
Sohag
Magdy M Amin, professor
About this study
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.
Read the detailed 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 .
- Conditions
- Microcystic Lymphatic Malformation, Combined Vascular Malformation, Vascular Malformations
- Being tested
- Sirolimus 1Mg Oral Tablet
- Medical subject headings
- Lymphangioma, Vascular Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sirolimus 1Mg Oral TabletDrug
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
| Study group | Role | What they receive |
|---|---|---|
| group A | microcystic \& mixed lymphatic malformation Receives: Drug: Sirolimus 1Mg Oral Tablet | |
| group B | vascular malformation Receives: Drug: Sirolimus 1Mg Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2023
- Study start20 Nov 2023
- Primary completion1 Sep 2024 (estimated)
- Study completion1 Sep 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Microcystic Lymphatic MalformationStudies of the same condition, open and under way.
- More trials run by Sohag UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sohag University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tarek Ahmed Abd Elsalam, Sohag University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06160739
- Sponsor's protocol ID
- soh-Med-23-11-16MS
- Status last verified
- Dec 2023
- First submitted
- 29 Nov 2023
- Last updated
- 15 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 29874693Vlahovic 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.
PubMed 26148957Muller-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.
PubMed 29415296Adams 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.
PubMed 18381090
Study at a glance
From the registry record, last updated by the sponsor on 15 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations
- ClinicalTrials.gov ID
- NCT06160739
- Acronym / sponsor ID
- soh-Med-23-11-16MS
- Conditions
- Microcystic Lymphatic Malformation, Combined Vascular Malformation, Vascular Malformations
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Sohag UniversityUniversity, hospital or other
- Study start
- 20 Nov 2023 (actual)
- Primary completion
- 1 Sep 2024 (estimated)
- Study completion
- 1 Sep 2024 (estimated)
- First posted
- 7 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 .
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| group A | – | Drug: Sirolimus 1Mg Oral Tabletmicrocystic \& mixed lymphatic malformation |
| group B | – | Drug: Sirolimus 1Mg Oral Tabletvascular malformation |
Who may qualify
- Ages
- 6 months to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sirolimus 1Mg Oral Tabletpatients 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 | Drug | group A, group B |
Primary outcome
changes in size of the lesion
Time frame: 1 year
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Dec 2023.
- Background: PubMed 29874693Sadick 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.
- Background: PubMed 26148957Vlahovic 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.
- Background: PubMed 29415296Muller-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.
- Background: PubMed 18381090Adams 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mohamed yousef, Ass. Prof. contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Microcystic Lymphatic Malformation and Combined Vascular Malformation, run by Sohag University at 1 site in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 15 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Tarek A Abd elsalam, resident (01064411709, tarikahmed@med.sohag.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
changes in size of the lesion (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06160739, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Dec 2023.