Trial document




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  DRKS00015436

Trial Description

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Title

RETROSPECTIVE CHART ANALYSIS OF HYQVIA USAGE
IN PEDIATRIC PATIENTS WITH PID OR SID

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Trial Acronym

RAHPP

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URL of the Trial

http://(keine)

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Brief Summary in Lay Language

This study assesses the usage of HyQiva in children mit PID und SID under clinical practice conditions. Only available data from patient charts are used.

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Brief Summary in Scientific Language

HyQvia is a product consisting of recombinant human hyaluronidase (rHuPH20, Hylenex®), and a human normal immunoglobulin (IG). The subcutaneous (SC) IG is a 10% solution prepared from human plasma consisting of at least 98% IgG, which contains a broad spectrum of antibodies (Kiovig®) .The two components are packaged together as a dual vial unit: IG provides the therapeutic effect and the recombinant human hyaluronidase facilitates the dispersion and absorption of the IG, increasing the bioavailability.

HyQvia is infused at 1-2 sites per administration (e.g. abdomen, thigh). Often it is sufficient if the infusion is applied once a month. After appropriate training, patients can self-administer HyQiva at home.


In a phase III study, also 26 patients under 18 years were included (Wasserman RL et al: Recombinant human hyaluronidase-facilitated subcutaneous infusion of human immunoglobulins for primary immunodeficiency. J Allergy Clin Immunol 2012, 130(4):951-957 e911). The youngest kid was 4 years old, and median age was 11 years. Nineteen patients completed 14-18 months HyQvia treatment in the study; of these, 15 patients opted to continue in the extension study with maximum treatment duration of 3.3 years. Validated acute serious bacterial infections were reported at 0.08/patient-year (four pneumonia episodes in three patients). No serious adverse drug reaction (ADR) was reported, and rates of local and systemic ADRs were low (0.09/infusion and 0.1/infusion). Infection rates were low (3.02/patient-year) with sustained Ig trough levels (median: 1009 mg/dl). Of 674 HyQvia infusions, 97.2% required no change of administration due to ADR, in most (82.5%) with one infusion site. No patient developed neutralizing anti-rHuPH20 antibodies. After conclusion of that study, 100% of patients aged <14 years or their caregivers and 85.7% of patients aged 14 to <18 years expressed preference for HyQvia compared with Ig administered intravenously or Ig administered subcutaneously. Information about the utilization pattern of HyQvia in pediatric patients under routine clinical practice conditions is lacking to date. The current retrospective chart data analysis aims to close this gap.

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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

  •   DRKS00015436
  •   2018/08/27
  •   [---]*
  •   no
  •   Approved
  •   7966_BO_K_2018, Ethikkommission der Medizinischen Hochschule Hannover
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Secondary IDs

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Health Condition or Problem studied

  •   Primary immunodeficiency, secondary immnunodeficiency
  •   D80 -  Immunodeficiency with predominantly antibody defects
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Interventions/Observational Groups

  •   Data from children with PID or SID who received HyQvia for at least 6 months are taken retrospectively from the patient files. Demographic and clinical data as well as detailed data on the use of HyQvia are collected.
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Characteristics

  •   Non-interventional
  •   Observational study
  •   Single arm study
  •   Open (masking not used)
  •   [---]*
  •   Uncontrolled/Single arm
  •   Treatment
  •   Single (group)
  •   IV
  •   No
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Primary Outcome

HyQvia usage by patient characteristics (age, diagnosis, medical history), IgG treatment initiation, administration, and tolerability

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Secondary Outcome

• Adverse drug reactions (ADR) related to the full infusion interval of the 2 HyQvia infusions that are being captured)
• Utilization of health resources (training sessions, nurse visits at home)
• Physician’s therapy assessment

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Countries of Recruitment

  •   Germany
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Locations of Recruitment

  • University Medical Center 
  • Medical Center 
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Recruitment

  •   Actual
  •   2019/05/29
  •   30
  •   Multicenter trial
  •   National
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Inclusion Criteria

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

1.Patient with PID or SID has been on HyQvia infusion for at least 6 months
2.Patients was <18 years at the date of the 2nd documented HyQvia infusion
3.Patients/parent/guardian provided written informed consent for documentation. Data on the first HyQvia infusion and on the most recent HyQvia infusion are documented.

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Exclusion Criteria

none

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Addresses

  • start of 1:1-Block address primary-sponsor
    • GWT-TUD GmbH, Medizin
    • Ms.  Doris  Breiner 
    • Freiberger Str. 33
    • 01067  Dresden
    • Germany
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    • GWT-TUD GmbH, Medizin
    • Ms.  Doris  Breiner 
    • Freiberger Str. 33
    • 01067  Dresden
    • Germany
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    • GWT-TUD GmbH, Medizin
    • Ms.  Doris  Breiner 
    • Freiberger Str. 33
    • 01067  Dresden
    • Germany
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Sources of Monetary or Material Support

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    • Shire Deutschland GmbH
    • Friedrichstr. 149
    • 10117  Berlin
    • Germany
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Status

  •   Recruiting ongoing
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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.