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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
Must not have
History of colon cancer or colon adenoma with high grade dysplasia
Chronic renal disease
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
No Placebo-Only Group

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.
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.
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 below
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I have a confirmed PTEN gene mutation.
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I have Cowden syndrome or a related genetic condition.
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I am able to understand and agree to participate in the study.
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I am 18 years old or older.
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I have had a colonoscopy showing more than 50 polyps.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had colon cancer or a serious type of colon polyp.
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I have long-term kidney disease.
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I have chronic kidney disease.
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I have had surgery to remove part or all of my colon.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year 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 colon polyp burden by number
Colon structure (body structure)
Secondary study objectives
Change in well-being assessment

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%
Decreased Appetite
5%
Renal Failure
5%
Jaundice
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%
Hyperlipidaemia
3%
Thrombocytopenia
3%
Pleural Effusion
3%
Myalgia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Hypokalaemia
2%
Sepsis
2%
Pneumonia
1%
Urinary Retention
1%
Hypoglycaemia
1%
Renal Failure Acute
1%
Cerebral Haemorrhage
1%
Cardiac Failure Congestive
1%
Hypercholesterolaemia
1%
Hepatic Artery Stenosis
1%
Portal Vein Thrombosis
1%
Encephalopathy
1%
Transplant Rejection
1%
Blood Alkaline Phosphatase Increased
1%
Multi-Organ Failure
1%
Chest Pain
1%
Crohn's Disease
1%
Non-Small Cell Lung Cancer Metastatic
1%
Atrial Flutter
1%
Benign Prostatic Hyperplasia
1%
Ventricular Tachycardia
1%
Febrile Neutropenia
1%
Hepatic Failure
1%
Gastritis
1%
Gastrointestinal Tract Adenoma
1%
Clostridium Difficile Colitis
1%
Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%
Confusional State
1%
Hepatic Neoplasm Malignant
1%
Abdominal Hernia
1%
Inappropriate Antidiuretic Hormone Secretion
1%
Gastrointestinal Haemorrhage
1%
Blood Glucose Increased
1%
Spinal Osteoarthritis
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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
4,675 Previous Clinical Trials
28,717,171 Total Patients Enrolled
Ohio State UniversityLead Sponsor
871 Previous Clinical Trials
655,722 Total Patients Enrolled
Peter P Stanich, MDPrincipal InvestigatorThe Ohio State University Wexner Medical Center

Media Library

Sirolimus (mTOR inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04094675 — Phase 2
Cowden Syndrome Research Study Groups: Treatment arm
Cowden Syndrome Clinical Trial 2023: Sirolimus Highlights & Side Effects. Trial Name: NCT04094675 — Phase 2
Sirolimus (mTOR inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04094675 — Phase 2
~0 spots leftby Feb 2025
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