Sirolimus for PTEN Hamartoma Tumor Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
PTEN Hamartoma Tumor Syndrome+12 More
Sirolimus - Drug
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

1 of 3

Other trials for PTEN Hamartoma Tumor Syndrome

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

2 of 3
This is further along than 68% of similar trials

Other trials for PTEN Hamartoma Tumor Syndrome

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
This histogram enumerates side effects from a completed 2008 Phase 4 trial (NCT00118742) in the CellCept + CNI (Tacrolimus or Cyclosporine) ARM group. Side effects include: Diarrhoea with 29%, Abdominal Pain with 18%, Hepatitis C with 16%, Nausea with 16%, Fatigue with 16%.

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: Drug
Treatment
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 preview

Michael Gill - B. Sc.

First Published: October 21st, 2021

Last Reviewed: October 11th, 2022

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