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mTOR inhibitor

Weekly Sirolimus for Lymphatic and Venous Malformations

Phase 2
Recruiting
Led By Alexandra Ritter, BS
Research Sponsored by Medical University of South Carolina
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline and 6 months
Awards & highlights

Summary

This trial is testing if taking sirolimus once a week can help people with venous and lymphatic malformations. These patients have few treatment options. Sirolimus aims to reduce abnormal growths by affecting the immune system. The study will also check for side effects and patient satisfaction over several months. Sirolimus has been used in various studies to treat lymphatic malformations and has shown potential in stabilizing lung function in patients with lymphangioleiomyomatosis.

Who is the study for?
This trial is for individuals aged 2 years and older with venous, lymphatic, or venolymphatic malformations. It excludes those with contraindications to sirolimus, on certain other medications, pregnant women or those who might become pregnant without effective contraception, and children with a history of transplant or immunodeficiency.
What is being tested?
The study tests if taking the oral medication Sirolimus once a week can treat venous and lymphatic malformations effectively. The treatment lasts for six months with an option to continue thereafter. Patient satisfaction and side effects will also be evaluated.
What are the potential side effects?
Sirolimus may cause serious side effects including low white blood cell count (neutropenia), painful mouth sores (oral ulcerations), and changes in lab test results which could indicate harm to organs.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month six
This trial's timeline: 3 weeks for screening, Varies for treatment, and month six for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in size of lesion
Change in size of lesion through photograph
Secondary study objectives
Change in quality of life as assessed by questionnaire
Number of participants with laboratory abnormalities
Number of side effects experienced

Side effects data

From 2008 Phase 4 trial • 293 Patients • NCT00118742
29%
Diarrhoea
18%
Abdominal Pain
16%
Nausea
16%
Headache
16%
Fatigue
16%
Hepatitis C
14%
Vomiting
14%
Pyrexia
14%
Leukopenia
12%
Oedema Peripheral
11%
Insomnia
10%
Anaemia
10%
Hyperkalaemia
10%
Tremor
10%
Back Pain
10%
Hypertension
9%
Cough
9%
Pruritis
9%
Arthralgia
8%
Neutropenia
8%
Abdominal Pain Upper
8%
Dizziness
8%
Pain in Extremity
8%
Hepatic Enzyme Increased
7%
Dyspnoea
7%
Constipation
7%
Sinusitis
7%
Weight Decreased
6%
Blood Creatinine Increased
6%
Liver Function Test Abnormal
6%
White Blood Cell Count Decreased
5%
Muscle Spasms
5%
Jaundice
5%
Renal Failure
5%
Decreased Appetite
5%
Weight Increased
5%
Upper Respiratory Tract Infection
5%
Nasopharyngitis
5%
Asthenia
5%
Incision Site Pain
5%
Depression
4%
Anorexia
4%
Night Sweats
4%
Oropharyngeal Pain
4%
Rhinorrhoea
3%
Pleural Effusion
3%
Myalgia
3%
Hyperlipidaemia
3%
Thrombocytopenia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Sepsis
2%
Pneumonia
2%
Hypokalaemia
1%
Renal Failure Acute
1%
Hepatic Neoplasm Malignant
1%
Non-Small Cell Lung Cancer Metastatic
1%
Cerebral Haemorrhage
1%
Ventricular Tachycardia
1%
Urinary Retention
1%
Benign Prostatic Hyperplasia
1%
Clostridium Difficile Colitis
1%
Hypoglycaemia
1%
Blood Alkaline Phosphatase Increased
1%
Chest Pain
1%
Gastritis
1%
Crohn's Disease
1%
Abdominal Hernia
1%
Hepatic Failure
1%
Multi-Organ Failure
1%
Inappropriate Antidiuretic Hormone Secretion
1%
Gastrointestinal Haemorrhage
1%
Cardiac Failure Congestive
1%
Hepatic Artery Stenosis
1%
Portal Vein Thrombosis
1%
Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%
Gastrointestinal Tract Adenoma
1%
Febrile Neutropenia
1%
Encephalopathy
1%
Atrial Flutter
1%
Blood Glucose Increased
1%
Transplant Rejection
1%
Confusional State
1%
Spinal Osteoarthritis
1%
Hypercholesterolaemia
1%
Convulsion
1%
Peritonitis
1%
Haemorrhage Intracranial
1%
Deep Vein Thrombosis
1%
Inguinal Hernia
1%
Viral Infection
1%
Acarodermatitis
1%
Atrial Fibrillation
1%
Malaise
1%
Hepatic Cancer Metastatic
1%
Adenocarcinoma
1%
B-Cell Lymphoma
1%
Desmoid Tumour
1%
Pulmonary Embolism
1%
Stomatitis
1%
Influenza
1%
Staphylococcal Infection
1%
Umbilical Hernia
1%
Hepatic Function Abnormal
1%
Hyponatraemia
1%
Bacteraemia
1%
Cellulitis
1%
Clostridial Infection
1%
Diverticulitis
1%
Escherichia Urinary Tract Infection
1%
Lactobacillus Infection
1%
Lobar Pneumonia
1%
Pseudomonal Sepsis
1%
Post Procedural Haemorrhage
1%
Procedural Pain
1%
Biliary Anastomosis Complication
1%
Complications of Transplanted Kidney
1%
Bile Duct Obstruction
1%
Bile Duct Stenosis
1%
Biliary Tract Disorder
1%
Autoimmune Hepatitis
1%
Cholestasis
1%
Lung Disorder
1%
Pulmonary Oedema
1%
Sinus Congestion
1%
Embolism Venous
1%
Orthostatic Hypotension
1%
Vasculitis
1%
Hyperglycaemia
1%
Graft Versus Host Disease
100%
80%
60%
40%
20%
0%
Study treatment Arm
CellCept + CNI (Tacrolimus or Cyclosporine)
CellCept + Sirolimus

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment GroupExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
2013
Completed Phase 4
~2750

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Lymphatic malformations are often treated with mTOR inhibitors such as sirolimus. These drugs work by inhibiting the mammalian target of rapamycin (mTOR) pathway, which is crucial for cell growth, proliferation, and survival. By blocking this pathway, sirolimus reduces the abnormal growth of lymphatic vessels, thereby decreasing the size and symptoms of the malformations. This mechanism is particularly important for patients as it offers a targeted approach to manage the condition, potentially reducing the need for invasive procedures and improving quality of life.
Analysis of current data on the use of topical rapamycin in the treatment of facial angiofibromas in tuberous sclerosis complex.

Find a Location

Who is running the clinical trial?

Medical University of South CarolinaLead Sponsor
965 Previous Clinical Trials
7,399,591 Total Patients Enrolled
Alexandra Ritter, BSPrincipal InvestigatorMedical University of South Carolina
Chelsea Shope, MSCRPrincipal InvestigatorMedical University of South Carolina

Media Library

Sirolimus (mTOR inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04861064 — Phase 2
Lymphatic Malformations Research Study Groups: Treatment Group
Lymphatic Malformations Clinical Trial 2023: Sirolimus Highlights & Side Effects. Trial Name: NCT04861064 — Phase 2
Sirolimus (mTOR inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04861064 — Phase 2
~2 spots leftby Feb 2025