Sirolimus for PTEN Hamartoma Tumor Syndrome
Phase-Based Progress Estimates
Effectiveness
Safety
PTEN Hamartoma Tumor Syndrome+12 More
Sirolimus - DrugEligibility
18+
All Sexes
What conditions do you have?
Select
Eligibility
18+
All Sexes
What conditions do you have?
Select
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.
Eligible Conditions
- PTEN Hamartoma Tumor Syndrome
- PTEN Gene Mutation
- Bannayan Syndrome
- Polyposis
- Hamartoma
- Syndrome
- Cowden's Disease
Treatment Effectiveness
Effectiveness Progress
Study Objectives
2 Primary · 1 Secondary · Reporting Duration: 1 year
1 year
Change in colon polyp burden by number
Colon structure (body structure)
Change in well-being assessment
Trial Safety
Safety Progress
This is further along than 68% of similar trials
Side Effects for
CellCept + CNI (Tacrolimus or Cyclosporine)
29%Diarrhoea
18%Abdominal Pain
16%Hepatitis C
16%Nausea
16%Fatigue
16%Headache
14%Vomiting
14%Pyrexia
14%Leukopenia
12%Oedema Peripheral
11%Insomnia
10%Back Pain
10%Hyperkalaemia
10%Tremor
10%Anaemia
10%Hypertension
9%Arthralgia
9%Pruritis
9%Cough
8%Pain in Extremity
8%Abdominal Pain Upper
8%Hepatic Enzyme Increased
8%Dizziness
8%Neutropenia
7%Constipation
7%Sinusitis
7%Weight Decreased
7%Dyspnoea
6%White Blood Cell Count Decreased
6%Blood Creatinine Increased
6%Liver Function Test Abnormal
5%Nasopharyngitis
5%Depression
5%Jaundice
5%Asthenia
5%Upper Respiratory Tract Infection
5%Decreased Appetite
5%Weight Increased
5%Muscle Spasms
5%Incision Site Pain
5%Renal Failure
4%Night Sweats
4%Oropharyngeal Pain
4%Anorexia
4%Rhinorrhoea
3%Hyperlipidaemia
3%Thrombocytopenia
3%Rash
3%Incisional Hernia
3%Pleural Effusion
3%Acne
3%Myalgia
2%Sepsis
2%Pneumonia
2%Hypokalaemia
1%Bile Duct Stenosis
1%Biliary Tract Disorder
1%Acarodermatitis
1%Lobar Pneumonia
1%Inguinal Hernia
1%Bile Duct Obstruction
1%Hepatic Failure
1%Ventricular Tachycardia
1%Procedural Pain
1%Hyperglycaemia
1%Blood Glucose Increased
1%Clostridium Difficile Colitis
1%Viral Infection
1%Bacteraemia
1%Escherichia Urinary Tract Infection
1%Cellulitis
1%Clostridial Infection
1%Diverticulitis
1%Influenza
1%Lactobacillus Infection
1%Pseudomonal Sepsis
1%Staphylococcal Infection
1%Gastrointestinal Haemorrhage
1%Umbilical Hernia
1%Crohn's Disease
1%Abdominal Hernia
1%Peritonitis
1%Gastritis
1%Biliary Anastomosis Complication
1%Complications of Transplanted Kidney
1%Post Procedural Haemorrhage
1%Cholestasis
1%Hepatic Artery Stenosis
1%Autoimmune Hepatitis
1%Portal Vein Thrombosis
1%Hepatic Function Abnormal
1%Chest Pain
1%Multi-Organ Failure
1%Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%Desmoid Tumour
1%Gastrointestinal Tract Adenoma
1%Adenocarcinoma
1%Malaise
1%Hepatic Cancer Metastatic
1%B-Cell Lymphoma
1%Hepatic Neoplasm Malignant
1%Pulmonary Embolism
1%Febrile Neutropenia
1%Non-Small Cell Lung Cancer Metastatic
1%Sinus Congestion
1%Embolism Venous
1%Lung Disorder
1%Pulmonary Oedema
1%Atrial Fibrillation
1%Cardiac Failure Congestive
1%Encephalopathy
1%Orthostatic Hypotension
1%Cerebral Haemorrhage
1%Deep Vein Thrombosis
1%Vasculitis
1%Hypoglycaemia
1%Convulsion
1%Haemorrhage Intracranial
1%Blood Alkaline Phosphatase Increased
1%Atrial Flutter
1%Hyponatraemia
1%Transplant Rejection
1%Confusional State
1%Urinary Retention
1%Graft Versus Host Disease
1%Renal Failure Acute
1%Spinal Osteoarthritis
1%Benign Prostatic Hyperplasia
1%Stomatitis
1%Inappropriate Antidiuretic Hormone Secretion
1%Hypercholesterolaemia
Trial Design
1 Treatment Group
Treatment arm
1 of 1
Experimental Treatment
10 Total Participants · 1 Treatment Group
Primary Treatment: Sirolimus · No Placebo Group · Phase 2
Treatment arm
Drug
Experimental Group · 1 Intervention: Sirolimus · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
FDA approved
Trial Logistics
Trial Timeline
Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year
Who is running the clinical trial?
PTEN ResearchUNKNOWN
1 Previous Clinical Trials
46 Total Patients Enrolled
1 Trials studying PTEN Hamartoma Tumor Syndrome
46 Patients Enrolled for PTEN Hamartoma Tumor Syndrome
Ohio State UniversityLead Sponsor
704 Previous Clinical Trials
382,293 Total Patients Enrolled
Peter P Stanich, MDPrincipal InvestigatorThe Ohio State University Wexner Medical Center
Eligibility Criteria
Age 18+ · All Participants · 5 Total Inclusion Criteria
Mark “yes” if the following statements are true for you:You have had a previous colonoscopy with a burden of colon polyps that are too numerous to clear endoscopically.
You have a confirmed pathogenic or likely pathogenic PTEN germline mutation on genetic testing.
About The Reviewer
Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.
References
- Lynch, Patrick M., Jeffrey S. Morris, Sijin Wen, Shailesh M. Advani, William Ross, George J. Chang, Miguel Rodriguez-Bigas, et al.. 2016. “A Proposed Staging System and Stage-specific Interventions for Familial Adenomatous Polyposis”. Gastrointestinal Endoscopy. Elsevier BV. doi:10.1016/j.gie.2015.12.029.
- Stanich, Peter P., Victoria L. Owens, Seth Sweetser, Sherezade Khambatta, Thomas C. Smyrk, Ronald L. Richardson, Matthew P. Goetz, and Mrinal M. Patnaik. 2011. “Colonic Polyposis and Neoplasia in Cowden Syndrome”. Mayo Clinic Proceedings. Elsevier BV. doi:10.4065/mcp.2010.0816.
- Heald, Brandie, Jessica Mester, Lisa Rybicki, Mohammed S. Orloff, Carol A. Burke, and Charis Eng. 2010. “Frequent Gastrointestinal Polyps and Colorectal Adenocarcinomas in a Prospective Series of PTEN Mutation Carriers”. Gastroenterology. Elsevier BV. doi:10.1053/j.gastro.2010.06.061.
- Komiya, Takefumi, Gideon M. Blumenthal, Roopa DeChowdhury, Susan Fioravanti, Marc S. Ballas, John Morris, Thomas J. Hornyak, et al.. 2019. “A Pilot Study of Sirolimus in Subjects with Cowden Syndrome or Other Syndromes Characterized by Germline Mutations in pten”. The Oncologist. Oxford University Press (OUP). doi:10.1634/theoncologist.2019-0514.
- Stanich, Peter P. 2014. “Colonic Manifestations Ofptenhamartoma Tumor Syndrome: Case Series and Systematic Review”. World Journal of Gastroenterology. Baishideng Publishing Group Inc.. doi:10.3748/wjg.v20.i7.1833.
- Peter P Stanich 2019. "Sirolimus for Cowden Syndrome With Colon Polyposis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04094675.
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