What side effects are associated with this type of intervention?

The most common treatments for lymphatic malformations, particularly those involving mTOR inhibitors like sirolimus, are associated with several side effects. These include neutropenia (a reduction in white blood cells), oral ulcerations, and various laboratory abnormalities. These side effects can be significant and require careful monitoring during treatment.

What prior FDA approvals exist?

Sirolimus, an mTOR inhibitor, has shown promise in treating lymphatic malformations, although it is not specifically FDA-approved for this indication. It is used off-label due to its efficacy in reducing the size and symptoms of these malformations. Other mTOR inhibitors, such as everolimus, have similar mechanisms of action and are being explored for similar uses. These drugs work by inhibiting the mTOR pathway, which is involved in cell growth and proliferation, thereby helping to manage the abnormal growths associated with lymphatic malformations.

What other types of research exist?

Rapamycin has shown promise in reducing and normalizing abnormal lymphatics in lymphatic malformations without affecting normal lymphatics. Additionally, ongoing research into other mTOR inhibitors and targeted therapies may provide new options. However, specific alternatives to Sirolimus were not detailed in the provided context.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and month six for reporting.

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 • 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

Cough

Pruritis

Arthralgia

Neutropenia

Abdominal Pain Upper

Dizziness

Pain in Extremity

Hepatic Enzyme Increased

Dyspnoea

Constipation

Sinusitis

Weight Decreased

Blood Creatinine Increased

Liver Function Test Abnormal

White Blood Cell Count Decreased

Jaundice

Renal Failure

Muscle Spasms

Decreased Appetite

Weight Increased

Upper Respiratory Tract Infection

Nasopharyngitis

Asthenia

Incision Site Pain

Depression

Anorexia

Night Sweats

Oropharyngeal Pain

Rhinorrhoea

Myalgia

Pleural Effusion

Hyperlipidaemia

Thrombocytopenia

Rash

Acne

Incisional Hernia

Sepsis

Pneumonia

Hypokalaemia

Gastritis

Abdominal Hernia

Urinary Retention

Multi-Organ Failure

Renal Failure Acute

Ventricular Tachycardia

Cerebral Haemorrhage

Hypoglycaemia

Benign Prostatic Hyperplasia

Non-Small Cell Lung Cancer Metastatic

Crohn's Disease

Clostridium Difficile Colitis

Hepatic Neoplasm Malignant

Hepatic Failure

Chest Pain

Blood Alkaline Phosphatase Increased

Inappropriate Antidiuretic Hormone Secretion

Gastrointestinal Haemorrhage

Cardiac Failure Congestive

Hepatic Artery Stenosis

Portal Vein Thrombosis

Epstein-Barr Virus Associated Lymphoproliferative Disorder

Gastrointestinal Tract Adenoma

Febrile Neutropenia

Encephalopathy

Atrial Flutter

Blood Glucose Increased

Transplant Rejection

Confusional State

Spinal Osteoarthritis

Hypercholesterolaemia

Convulsion

Peritonitis

Haemorrhage Intracranial

Deep Vein Thrombosis

Inguinal Hernia

Viral Infection

Acarodermatitis

Atrial Fibrillation

Malaise

Hepatic Cancer Metastatic

Adenocarcinoma

B-Cell Lymphoma

Desmoid Tumour

Pulmonary Embolism

Stomatitis

Influenza

Staphylococcal Infection

Umbilical Hernia

Hepatic Function Abnormal

Hyponatraemia

Bacteraemia

Cellulitis

Clostridial Infection

Diverticulitis

Escherichia Urinary Tract Infection

Lactobacillus Infection

Lobar Pneumonia

Pseudomonal Sepsis

Post Procedural Haemorrhage

Procedural Pain

Biliary Anastomosis Complication

Complications of Transplanted Kidney

Bile Duct Obstruction

Bile Duct Stenosis

Biliary Tract Disorder

Autoimmune Hepatitis

Cholestasis

Lung Disorder

Pulmonary Oedema

Sinus Congestion

Embolism Venous

Orthostatic Hypotension

Vasculitis

Hyperglycaemia

Graft Versus Host Disease

100%

80%

60%

40%

20%

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

Do I qualify?Apply below to find out

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.