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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
Study Summary
This trial will test if a lower dose of sirolimus, given once a week, can treat venous and lymphatic malformations effectively, with fewer side effects.
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.Check my eligibility
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.See study design
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
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in size of lesion
Change in size of lesion through photograph
Secondary outcome measures
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%
Jaundice
5%
Renal Failure
5%
Muscle Spasms
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%
Myalgia
3%
Pleural Effusion
3%
Hyperlipidaemia
3%
Thrombocytopenia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Sepsis
2%
Pneumonia
2%
Hypokalaemia
1%
Gastritis
1%
Urinary Retention
1%
Abdominal Hernia
1%
Multi-Organ Failure
1%
Benign Prostatic Hyperplasia
1%
Non-Small Cell Lung Cancer Metastatic
1%
Hypoglycaemia
1%
Crohn's Disease
1%
Clostridium Difficile Colitis
1%
Inappropriate Antidiuretic Hormone Secretion
1%
Hepatic Neoplasm Malignant
1%
Ventricular Tachycardia
1%
Blood Alkaline Phosphatase Increased
1%
Hepatic Failure
1%
Renal Failure Acute
1%
Chest Pain
1%
Cerebral Haemorrhage
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
Find a Location
Who is running the clinical trial?
Medical University of South CarolinaLead Sponsor
933 Previous Clinical Trials
7,394,591 Total Patients Enrolled
Alexandra Ritter, BSPrincipal InvestigatorMedical University of South Carolina
Chelsea Shope, MSCRPrincipal InvestigatorMedical University of South Carolina
Media Library
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.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any more patients needed for this research project?
"Correct. At the moment, this study is looking for 24 patients from 1 site, as indicated by the clinicaltrials.gov listing which was created on 1/18/2022 and last updated on 11/7/2022."
Answered by AI
To date, how many individuals have signed up to be a part of this research project?
"Indeed, this upcoming clinical trial is still recruiting patients. According to the posting on clinicaltrials.gov, the first 18th of January 2022 was when recruitment began and it will continue until the 7th of November of the same year. They are looking for a total 24 participants that will be split between 1 site."
Answered by AI
Has the FDA cleared Sirolimus for general medical use?
"Sirolimus has received a safety score of 2. While there is some evidence that the drug is safe, none exists to support its efficacy in humans."
Answered by AI
Are there any other existing studies that have used Sirolimus?
"There are a total of 130 clinical trials underway that are researching Sirolimus. 13 of those trials are currently in Phase 3. The majority of the research is taking place in Cincinnati, Ohio; however, there are 1,084 other locations around the world conducting similar studies."
Answered by AI
What has been the most successful application of Sirolimus?
"Sirolimus is a medication that helps patients who have received liver transplants and frequently experience rejection as well as those with kidney disease or renal angiomyolipomas."
Answered by AI
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