ATERO : A Randomised Study With Tranexamic Acid in Epistaxis of Rendu Osler Syndrome

NCT00355108 | Completed Phase 3 DMC

• 1 other identifier: 2005.405
• Study Type: interventional
• Target: 170
• Locations: 1 country
• Sites: 1

Brief Summary

Scientific Objectives Primary objective : Demonstrate that oral intake of tranexamic acid significantly reduces the risk of epistaxis occurrence, estimated by the average monthly duration of episodes of epistaxis. Secondary objectives :

• Document the benefit of tranexamic acid on the amount of haemoglobine and quality of life of patients.
• Identify scalable and genetic factors of response to the treatment by tranexamic acid.
• Describe compliance and tolerance of the treatment. Method Experimental Design We suggest the realisation of a randomised comparative clinical trial versus placebo, with a crossover of random alternated periods of three months over a total of six months. Study Population 213 affected patients, displaying sufficiently invalidating epistaxis to require a basal treatment, will be recruited and followed every 3 months. Recruitment will lean on teams who are part of the national French network in close collaboration with the reference centre for the Rendu-Osler disorder, appointed to Lyon on the 19th of November 2004. Follow up of the study will be carried out by the Clinical Investigational Centre of the related towns. Outcome measures The main criterion of efficacy is the average duration of epistaxis, the secondary criterion of efficacy is the average number of epistaxis measured per month. Tolerance will be analysed according to the occurrence of venal or arterial thrombosis and allergic accidents. Venal thrombosis will systematically be sought by an inferior limbs echodoppler. Response markers will be sought through modelisation incorporating environmental, phenotypic and genetic factors.

Conditions

Telangiectasia, Hereditary Hemorrhagic; Osler-Rendu Disease

Keywords

Rendu Osler syndrome; Epistaxis; Tranexamic acid; clinical trial

Outcome Measures

Primary Outcomes (1)

• The primary efficacy criterion is the average monthly duration of epistaxis.

  after first 3 month period treatment or placebo and after second 3 month period treatment or placebo

Secondary Outcomes (5)

• the average monthly number of epistaxis,

  after first 3 month period treatment or placebo and after second 3 month period treatment or placebo

• the average duration of one episode of epistaxis measured over a month,

  after first 3 month period treatment or placebo and after second 3 month period treatment or placebo

• the Haemoglobin level

  after first 3 month period treatment or placebo and after second 3 month period treatment or placebo

• estimation of Quality of life.

  over first 3 month period treatment or placebo and over second 3 month period treatment or placebo

• Outcome of following Adverse Event: arterial or veinous thrombosis, allergic accident

  during first 3 month period treatment or placebo and over second 3 month period treatment or placebo

Interventions

tranexamic acid DRUG

2 3-month-periods in cross-over: placebo or 3g/day of tranexamic acid

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Repeated, spontaneous epistaxis in a known Rendu Osler patient who wants global caring, these must be daily epistaxis (\> 28 a month) or a total amount of 60 minutes of bleeding a month (50 % Rendu-Osler patients bleed before 20 and 99 % at 50 when penetrance is complete).
• Good completion of the grid of control by recording the number and duration of epistaxis over a period of three month.

You may not qualify if:

• Rendu Osler syndrome with specific phenotype : absence of epistaxis or occasional epistaxis, no need for particular care.
• Maintained expectations of an ENT treatment of epistaxis in a delay of three months.
• Incapacity of detailing the number of epistaxis, especially, at least, within three months before entering the study.
• No informed consent obtained after informing the patient on his participation to the study.
• Expected lack of observance because of an incapacity to compel to the daily treatment.
• Formal indication to Tranexamic Acid (angioneurotic oedema as a disease modifying treatment of 3 to 4 g/ day).
• Contra-indications to Tranexamic acid: history of convulsion, arterial or veinous thrombosis, , positivity of veinous echo doppler of the inferior limbs; Serum creatinine \> 250 µmol/l.
• N.B. Cases of bad observance are not frequent, patients being highly concerned with a treatment to reduce their discomfort and anaemia. They are more likely ready to move to a centre insuring this handling. Nevertheless, lack of observance will be detected over a period of 15 days of placebo in simple blind, before randomization.

Sponsors & Collaborators

• Hospices Civils de Lyon: lead

Study Sites (1)

Henri PLAUCHU

Lyon, 69002, France

Location

Related Publications (1)

• Gaillard S, Dupuis-Girod S, Boutitie F, Riviere S, Moriniere S, Hatron PY, Manfredi G, Kaminsky P, Capitaine AL, Roy P, Gueyffier F, Plauchu H; ATERO Study Group. Tranexamic acid for epistaxis in hereditary hemorrhagic telangiectasia patients: a European cross-over controlled trial in a rare disease. J Thromb Haemost. 2014 Sep;12(9):1494-502. doi: 10.1111/jth.12654. Epub 2014 Jul 29.

  PMID: 25040799 RESULT

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic; Epistaxis

Interventions

Tranexamic Acid

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nose Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Respiratory; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cyclohexanecarboxylic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals

Study Officials

• Henri PLAUCHU, MD

  Hospices Civils de Lyon

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: July 20, 2006
• First Posted: July 21, 2006
• Study Start: September 1, 2006
• Primary Completion: May 1, 2010
• Study Completion: May 1, 2010
• Last Updated: May 28, 2019

Record last verified: 2010-12

Locations

FR (1)