Trial document




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  DRKS00013943

Trial Description

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Title

Descriptive Analysis of the Expert Opinion Regarding the Initiation of Augmentation Therapy in Alpha-1-Antitrypsin-Deficiency

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Trial Acronym

NA

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URL of the Trial

http://NA

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Brief Summary in Lay Language

This is a questionnaire directed to experts in the field of alpha-1-antitrypsin-deficiency. We want to know what kind of patients would be treated with augmentation therapy (replacing the protein that is lacking in that disease) by experts.

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Brief Summary in Scientific Language

Alpha-1-antitrypsin deficiency (AATD) is a hereditary disorder that is characterized by a low serum level of AAT. The loss of anti-inflammatory and anti-proteolytic functions, together with pro-inflammatory effects of polymerized AAT contributes to the development of a progressively destructive process that results in an increased risk of developing chronic obstructive pulmonary disease (COPD) and emphysema, especially in smokers.
Specific therapy includes the possibility of "Augmentation" therapy, i.e. replacing alpha-1-antitrypsin by means of the plasma-purified protein of healthy donors.
While some patients clearly fall into the indication for augmentation therapy, others are borderline cases.
In our study, 50 experts will evaluate 500 virtual clinical cases (10 / per expert) and decide whether or not augmentation therapy should be started.
The planned analysis tries do identify the factors that are most influencal on the decision of the experts.

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Do you plan to share individual participant data with other researchers?

[---]*

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Description IPD sharing plan:

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Organizational Data

  •   DRKS00013943
  •   2018/02/05
  •   [---]*
  •   yes
  •   Approved
  •   162/17, Ethik-Kommission des Fachbereichs Medizin der Philipps-Universität Marburg
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Secondary IDs

  • [---]*
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Health Condition or Problem studied

  •   E88.0 -  Disorders of plasma-protein metabolism, not elsewhere classified
  •   J44.8 -  Other specified chronic obstructive pulmonary disease
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Interventions/Observational Groups

  •   The study Population consists of 50 expertes for AATD (Alpha-1-antitrypsin deficiency) that will evaluate 500 virtual clinical cases (10 cases per expert) and make the decision to start augmentation therapy or not.
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Characteristics

  •   Non-interventional
  •   Observational study
  •   Single arm study
  •   Open (masking not used)
  •   [---]*
  •   Uncontrolled/Single arm
  •   Other
  •   Single (group)
  •   N/A
  •   N/A
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Primary Outcome

Descriptive analysis of all those factors which would lead to an augmentation therapy. Due to the lack of a prespecified questionnaire, we created one. After a phone call, experts in the field of AATD will get the questionnaire and complete it once. No follow-up is planned.

The Analyse factors include AAT serum level, genotype, FEV1, diffusion capacity, FEV1-decline, diffusion capacity decline, age, lung function decline, CT-scan, smoking history, symptoms and exacerbations.

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Secondary Outcome

Individual factors of the experts (Age, gender, country of origin). These will be taken from the questionnaire.

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Countries of Recruitment

  •   Germany
  •   Spain
  •   Netherlands
  •   Italy
  •   France
  •   United Kingdom
  •   Czech Republic
  •   Portugal
  •   Ireland
  •   Poland
  •   Switzerland
  •   Belgium
  •   Turkey
  •   Croatia
  •   Romania
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Locations of Recruitment

  • University Medical Center 
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Recruitment

  •   Actual
  •   2018/04/13
  •   50
  •   Multicenter trial
  •   International
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Inclusion Criteria

  •   Both, male and female
  •   no minimum age
  •   no maximum age
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Additional Inclusion Criteria

(1) 5-year work experience in the field of AATD
(2) 5 treated AATD-patients/year

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Exclusion Criteria

none

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Addresses

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    • Philipps-Universität Marburg FB HumanmedizinInnere Medizin, Pneumologie
    • Mr.  PD  Timm  Greulich 
    • Baldingerstraße
    • 35043  Marburg
    • Germany
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    • Philipps-Universität Marburg FB Humanmedizin, Innere Medizin, Pneumologie
    • Mr.  PD  Timm  Greulich 
    • Baldingerstraße
    • 35043  Marburg
    • Germany
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    • Philipps-Unversität Marburg FB Humanmedizin, Innere Medizin, Pneumologie
    • Mr.  PD  Timm  Greulich 
    • Baldingerstraße
    • 35043  Marburg
    • Germany
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Sources of Monetary or Material Support

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    • Philipps-Universität Marburg, FB 20, Humanmedizin
    • Mr.  PD Dr. med.  Timm  Greulich 
    • Baldingerstraße
    • 35043  Marburg
    • Germany
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Status

  •   Recruiting complete, follow-up complete
  •   2018/12/25
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Trial Publications, Results and other Documents

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* This entry means the parameter is not applicable or has not been set.