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Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02690246
Recruitment Status : Active, not recruiting
First Posted : February 24, 2016
Last Update Posted : February 15, 2019
Information provided by (Responsible Party):
Urban Geisthoff, University Hospital, Essen

Brief Summary:
Hereditary haemorrhagic telangiectasia (HHT), also known as Rendu-Osler-Weber syndrome, is an inherited multisystemic disorder with recurrent epistaxis, mucocutaneous telangiectasia and visceral arteriovenous malformations. The purpose of this study is to provide data about multiple clinical aspects of HHT and responses to treatment. For comparison of some aspects also data of non-affected relatives is collected (second cohort). the questionnaire has been designed primarily for web based entry, but can also be circulated in paper format on request.

Condition or disease Intervention/treatment
Hereditary Haemorrhagic Telangiectasia (HHT) Other: questionnaire

Detailed Description:
Specific aspects include potential consequences from iron deficiency, efficacy and safety of self-packing, effect of female sex hormones, mortality, effects on the immune system.

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Study Type : Observational
Actual Enrollment : 915 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Questionnaire Based Study on Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia (HHT)
Study Start Date : February 2016
Actual Primary Completion Date : August 14, 2017
Estimated Study Completion Date : December 2019

Group/Cohort Intervention/treatment
patients with HHT
patients with Hereditary Haemorrhagic Telangiectasia
Other: questionnaire
a questionnaire based study

control cohort
indirectly only as a control cohort in questionnaire of affected persons

Primary Outcome Measures :
  1. Effect of nasal self-packing to treat epistaxis on the quality of life in patients with HHT measured mainly by Glasgow Benefit Inventory (GBI) [ Time Frame: 34 months ]
    Hereditary Haemorrhagic Telangiectasia is characterised by recurrent epistaxis which can lead to a feeling to lose control. The investigators examine whether the use of high volume low pressure nasal packing is a secure and practical method to improve patients' quality of life.

Secondary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 34 months ]

Other Outcome Measures:
  1. Number of patients with restless legs syndrome according to a questionnaire including the criteria by Allen et al., Sleep Medicine 4(2003) 101-119 [ Time Frame: 34 months ]
  2. Number of infectious diseases and complications in comparison to healthy spouses [ Time Frame: 34 months ]
  3. Hormonal changes: Change of number of visible telangiectases during menstrual cycle and pregnancy [ Time Frame: 34 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Patients with HHT; Indirectly we accept healthy volunteers only as control cohort in questionnaires of affected persons.

Inclusion Criteria:

  • a diagnosis of Hereditary Haemorrhagic Telangiectasia

Exclusion Criteria:

  • unable to provide informed consent

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02690246

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University Hospital Essen
Essen, Germany, 45147
Sponsors and Collaborators
University Hospital, Essen
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Study Director: Urban Geisthoff, Prof. Dr. University Hospital, Essen

Additional Information:
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Responsible Party: Urban Geisthoff, Prof. Dr. med., University Hospital, Essen Identifier: NCT02690246     History of Changes
Other Study ID Numbers: University Clinic Essen
First Posted: February 24, 2016    Key Record Dates
Last Update Posted: February 15, 2019
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Additional relevant MeSH terms:
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Telangiectasia, Hereditary Hemorrhagic
Vascular Diseases
Cardiovascular Diseases
Hemostatic Disorders
Hemorrhagic Disorders
Hematologic Diseases
Vascular Malformations
Cardiovascular Abnormalities
Congenital Abnormalities