Sirolimus for Osler-Weber-Rendu Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Osler-Weber-Rendu Syndrome+1 MoreSirolimus - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study 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.

Eligible Conditions
  • Osler-Weber-Rendu Syndrome
  • Nosebleed

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

Study Objectives

7 Primary · 2 Secondary · Reporting Duration: 9 months

9 months
Biomarkers
Blood glucose level
Electrolytes
Epistaxis
Hematology
Liver function
Renal function
ferritin level
lipid assessment

Trial Safety

Safety Progress

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

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%Sinusitis
7%Constipation
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%Rash
3%Hyperlipidaemia
3%Thrombocytopenia
3%Incisional Hernia
3%Pleural Effusion
3%Acne
3%Myalgia
2%Sepsis
2%Pneumonia
2%Hypokalaemia
1%Bile Duct Stenosis
1%Gastrointestinal Haemorrhage
1%Autoimmune Hepatitis
1%Convulsion
1%Lung Disorder
1%Blood Glucose Increased
1%Benign Prostatic Hyperplasia
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%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%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%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%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%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%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

Oral sirolimus tablets
1 of 1

Experimental Treatment

10 Total Participants · 1 Treatment Group

Primary Treatment: Sirolimus · No Placebo Group · Phase 2

Oral sirolimus tablets
Drug
Experimental Group · 1 Intervention: Sirolimus · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
2013
Completed Phase 4
~2750

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 9 months

Who is running the clinical trial?

National Institutes of Health (NIH)NIH
2,444 Previous Clinical Trials
24,578,433 Total Patients Enrolled
Unity Health TorontoLead Sponsor
496 Previous Clinical Trials
375,910 Total Patients Enrolled
Marie E Faughnan, MD MSc FRCPCPrincipal InvestigatorUnity Health Toronto
2 Previous Clinical Trials
35 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 5 Total Inclusion Criteria

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You are able to give informed consent.
References