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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 weeks3 visits
Treatment ~ Varies
Follow Up ~ month six
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month six
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 • NCT0011874229%
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.
Analysis of current data on the use of topical rapamycin in the treatment of facial angiofibromas in tuberous sclerosis complex.
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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
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Eligibility Criteria:
This trial includes the following eligibility criteria:- Children who have had previous immune system problems.Children who have had a severe or life-threatening infection in the past.If you are a child taking medications that inhibit CYP3A4.You have malformations in your veins, lymphatic system, or both.You have had an organ transplant in the past as a child.Children who have had their immune system deliberately weakened in the past.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment Group
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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