Trial document




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  DRKS00010438

Trial Description

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Title

HELP Study (TM): A Multicenter, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study to Evaluate DX-2930 For Long-Term Prophylaxis Against Acute Attacks of Hereditary Angioedema (HAE)

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Trial Acronym

HELP

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URL of the Trial

[---]*

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Brief Summary in Lay Language

In this study it will be investigated if DX-2930 can prevent attacks of hereditary angioedema (HAE). In addition the safety of DX-2930 therapy shall be investigated. Patient will be randomly assigned to one of the four treatment arms and will receive the study medication DX-2930 or placebo accordingly for a period of 182 days (6 months).

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Brief Summary in Scientific Language

Phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.

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Organizational Data

  •   DRKS00010438
  •   2016/04/27
  •   2015/10/23
  •   no
  •   Approved
  •   837.458.15 (10226), Ethik-Kommission bei der Landesärztekammer Rheinland-Pfalz
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Secondary IDs

  •   2015-003943-20 
  •   NCT02586805  (ClinicalTrials.gov)
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Health Condition or Problem studied

  •   D84.1 -  Defects in the complement system
  •   Hereditary Angioedema
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Interventions/Observational Groups

  •   DX-2930 300 mg every 2 weeks by subcutaneous injection
  •   DX-2930 300 mg every 4 weeks by subcutaneous injection
  •   DX-2930 150 mg every 4 weeks by subcutaneous injection
  •   Placebo
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Characteristics

  •   Interventional
  •   [---]*
  •   Randomized controlled trial
  •   Blinded
  •   patient/subject, investigator/therapist
  •   Placebo
  •   Prevention
  •   Parallel
  •   III
  •   No
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Primary Outcome

Number of investigator confirmed HAE (Hereditary Angioedema) attacks per week observed in each DX-2930 treatment arm versus placebo arm.

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Secondary Outcome

- Time to first HAE attack for each DX-2930 treatment arm versus placebo arm
- Number per week of HAE attacks requiring acute attack therapy use for each DX-2930 treatment arm versus placebo arm
- Number per week of moderate or severe HAE attacks requiring acute attack therapy use for each DX-2930 treatment arm versus placebo arm
- Reduction in high-morbidity HAE attacks for each DX-2930 treatment arm versus placebo arm

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Countries of Recruitment

  •   United States
  •   Germany
  •   United Kingdom
  •   Italy
  •   Canada
  •   Jordan
  •   Puerto Rico
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Locations of Recruitment

  • University Medical Center 
  • University Medical Center 
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Recruitment

  •   Actual
  •   2016/05/26
  •   15
  •   Multicenter trial
  •   International
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Inclusion Criteria

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

- Documented diagnosis of HAE, Type I or II

- Baseline rate of at least 1 Investigator-confirmed HAE attack per 4 weeks

- Executed informed consent

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Exclusion Criteria

– Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema
– Treatment with any other investigational drug or exposure to an investigational device within 4 weeks prior screening
– Exposure to ACE inhibitors or any estrogen-containing medications within 4 weeks prior to screening.
– Exposure to androgens or long-term prophylactic therapy for HAE within 2 weeks prior to entering the run-in period.
– Use of short-term prophylaxis for HAE within 7 days prior to entering the run-in period.
– Pregnancy or breastfeeding.

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Addresses

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    • Dyax Corp.
    • Ms.  PharmD  Carolyn  Soo 
    • 55 Network Drive
    • MA 01803  Burlington
    • United States
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    • Dyax Corp.
    • Ms.  PharmD  Carolyn  Soo 
    • 55 Network Drive
    • MA 01803  Burlington
    • United States
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    • Charité Campus Charité Mitte
    • Mr.  Prof. Dr. med.  Markus  Magerl 
    • Charitéplatz 1
    • 10117  Berlin
    • Germany
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    • Universitätsmedizin der Johannes Gutenberg-Universität Mainz
    • Ms.  PD Dr. med.  Petra  Staubach 
    • Langenbeckstraße 1
    • 55131  Mainz
    • Germany
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    • HZRM Hämophilie Zentrum Rhein Main GmbH
    • Ms.  Dr. med.  Inmaculada  Martinez-Saguer 
    • Hessenring 13a
    • 64546  Mörfelden-Walldorf
    • Germany
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Sources of Monetary or Material Support

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    • Dyax Corp.
    • Ms.  PharmD  Carolyn  Soo 
    • 55 Network Drive
    • MA 01803  Burlington
    • United States
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Status

  •   Recruiting complete, follow-up continuing
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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.