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mTOR inhibitor

Sirolimus for Nosebleeds in HHT

Phase 2
Waitlist Available
Led By Marie E Faughnan, MD MSc FRCPC
Research Sponsored by Unity Health Toronto
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Epistaxis at least 15 min per week
COVID-19 Vaccine (2 doses)
Must not have
Specific contra-indications for study drug (detailed in the product monograph)
Male participants of reproductive potential whose female partners are of childbearing potential and are not planning to use highly effective contraceptive method
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 9 months
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing if the drug sirolimus can help people with HHT who have moderate or severe nosebleeds. The study will last 3 months, and people will take sirolimus once a day.

Who is the study for?
This trial is for adults over 18 with Hereditary Hemorrhagic Telangiectasia (HHT) who suffer from moderate to severe nosebleeds lasting at least 15 minutes per week. Participants must have received two COVID-19 vaccine doses and not be pregnant, breastfeeding, or planning pregnancy without effective contraception. They should not have a history of cancer, acute infections, unstable illnesses, high creatinine levels, liver issues twice above normal limits or untreated dyslipidemia.
What is being tested?
The study tests the safety and effectiveness of oral sirolimus in reducing nosebleeds in HHT patients. Participants will take a daily dose of sirolimus to maintain specific blood levels for three months. The impact on nosebleeds will be measured against their situation before the trial using patient-reported outcomes.
What are the potential side effects?
Sirolimus may cause side effects such as mouth sores, diarrhea, nausea, lower blood cell counts increasing infection risk; it can also affect kidney function and cholesterol levels. Side effects vary among individuals.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I experience nosebleeds lasting over 15 minutes each week.
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I have received 2 doses of a COVID-19 vaccine.
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I am older than 18 years.
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I have been diagnosed with HHT either through clinical evaluation or genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have no known allergies or reactions to the study drug.
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I am a man and my partner can have children but we are not using effective birth control.
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My immune system is weak.
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I am not pregnant, breastfeeding, nor planning to become pregnant during the study.
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I currently have an infection.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~9 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 9 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Blood glucose level
Electrolytes
Hematology
+4 more
Secondary study objectives
Biomarkers
Epistaxis

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

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: Oral sirolimus tabletsExperimental Treatment1 Intervention
Sirolimus starting dose of 2 mg once daily, orally adjusted as need to maintain drug blood levels of 6-10 ng/ ml The first dose will be given at the week 12 visit and participants will be observed for 30 min
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?

University of California, San FranciscoOTHER
2,585 Previous Clinical Trials
15,084,179 Total Patients Enrolled
Unity Health TorontoLead Sponsor
556 Previous Clinical Trials
454,396 Total Patients Enrolled
National Institutes of Health (NIH)NIH
2,811 Previous Clinical Trials
8,161,248 Total Patients Enrolled

Media Library

Sirolimus (mTOR inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05269849 — Phase 2
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Osler-Weber-Rendu Syndrome Clinical Trial 2023: Sirolimus Highlights & Side Effects. Trial Name: NCT05269849 — Phase 2
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