Your session is about to expire
← Back to Search
mTOR inhibitor
Sirolimus for Cowden Syndrome with Colon Polyposis
Phase 2
Waitlist Available
Led By Peter P Stanich, MD
Research Sponsored by Ohio State University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Confirmed pathogenic or likely pathogenic PTEN germline mutation on genetic testing
Cowden syndrome or other PTEN hamartoma tumor syndrome spectrum disorder
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
Study Summary
This trial is investigating the use of sirolimus to reduce the number of colon polyps in patients with Cowden syndrome over a 1 year period.
Who is the study for?
This trial is for adults over 18 with Cowden syndrome or related disorders, who have a genetic mutation called PTEN and more than 50 colon polyps that can't be removed endoscopically. It's not for those with chronic kidney disease, past high-grade colon tumors, colectomy history, or current/pending pregnancy.Check my eligibility
What is being tested?
The study tests Sirolimus on patients with Cowden syndrome to see if it reduces the number of colon polyps over one year. Participants must have many polyps and a specific genetic mutation linked to their condition.See study design
What are the potential side effects?
Sirolimus may cause side effects such as mouth sores, increased risk of infection, rash, diarrhea, abnormal blood test results (like elevated cholesterol), and potentially lung problems.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have a confirmed PTEN gene mutation.
Select...
I have Cowden syndrome or a related genetic condition.
Select...
I am able to understand and agree to participate in the study.
Select...
I am 18 years old or older.
Select...
I have had a colonoscopy showing more than 50 polyps.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in colon polyp burden by number
Colon structure (body structure)
Secondary outcome measures
Change in well-being assessment
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%
Ventricular Tachycardia
1%
Renal Failure Acute
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%
Blood Alkaline Phosphatase Increased
1%
Hepatic Failure
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 armExperimental Treatment1 Intervention
There will be a clinic visit and colonoscopy at study entrance with standard of care sampling and assessment of polyps, including resection of concerning polyps. The investigators will also collect data on well-being via the SF-36 health survey (a validated questionnaire to help monitor this aspect given anecdotal patient-level reports of improvement while on therapy).
Study subjects will then begin sirolimus 2 mg by mouth daily for 1 year.
Laboratories will be checked at 4 days after initiation, at 2 weeks after initiation, then every 4 weeks for 3 months, then every 3 months to complete the year of therapy
Participants will have a clinic visit at 3, 6 and 9 months and include well-being assessment with the SF-36 health survey.
Participants will have a clinic visit with well-being assessment and perform colonoscopy at study closure at 12 months. The investigators will perform standard of care sampling and assessment of polyps, including resection of concerning polyps.
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?
PTEN ResearchUNKNOWN
1 Previous Clinical Trials
46 Total Patients Enrolled
PfizerIndustry Sponsor
4,570 Previous Clinical Trials
10,915,975 Total Patients Enrolled
Ohio State UniversityLead Sponsor
829 Previous Clinical Trials
505,625 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had colon cancer or a serious type of colon polyp.I have a confirmed PTEN gene mutation.I have Cowden syndrome or a related genetic condition.I have long-term kidney disease.You are pregnant, planning to become pregnant, or planning to father a child within 3 months after treatment.I am able to understand and agree to participate in the study.I am 18 years old or older.I have chronic kidney disease.I have had a colonoscopy showing more than 50 polyps.I have had surgery to remove part or all of my colon.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment arm
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.
How many participants has this clinical trial enrolled thus far?
"Yes, the records on clinicaltrials.gov indicate that this trial is presently recruiting patients. It was initially posted on September 16th 2019 and amended on November 12th 2021. The research requires 10 participants from a single medical centre."
Answered by AI
Does this research encompass individuals aged 65 or greater?
"According to the qualifications for this trial, only patients aged between 18 and 80 can enroll. There are 746 clinical trials available for individuals under the age of majority, while there are 4024 studies open to those above 65 years old."
Answered by AI
Are there opportunities to still join this experiment?
"As indicated by clinicaltrials.gov, this research initiative is actively looking for participants; the study was posted on September 16th 2019 and revised most recently on November 12th 2021."
Answered by AI
Can you explain the common applications of Sirolimus?
"Sirolimus has a range of therapeutic applications, such as preventing organ transplant rejections and treating kidney diseases like renal angiomyolipomas."
Answered by AI
Does this clinical trial have any specific qualifications for participants?
"Applicants for this clinical trial should have been diagnosed with pten hamartoma tumor syndrome and must be of age between 18-80 years. Approximately 10 people are being sought out to complete the study."
Answered by AI
Has further research been conducted regarding the effects of Sirolimus?
"Presently, 13 Phase 3 clinical trials are investigating the effects of Sirolimus with a total of 125 active studies. The majority of these research efforts are based in Cincinnati, Ohio; however 1074 other locations have joined their investigation into this medication's efficacy."
Answered by AI
Has Sirolimus been given the go-ahead by the Food and Drug Administration?
"Our analysts at Power have assigned sirolimus a score of 2 on their safety scale, since clinical data exists to support its security but efficacy is still unproven."
Answered by AI
Recent research and studies
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger