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Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox

Primary Purpose

Cartilage-hair Hypoplasia

Status
Unknown status
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Varilrix
Sponsored by
Helsinki University Central Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Cartilage-hair Hypoplasia

Eligibility Criteria

12 Months - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • genetically confirmed cartilage-hair hypoplasia diagnosis
  • age >12 months
  • no history of chickenpox
  • informed concent of the patient/caregiver.

Exclusion Criteria:

  • positive serum IgG for varicella zoster virus
  • low CD4+ cell counts (<15% or <200 cells/mm3)
  • clinical or laboratory signs of severe immunodeficiency
  • ongoing intravenous or subcutaneous immunoglobulin treatment

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Cartilage-hair hypoplasia (CHH)

    Arm Description

    Selected CHH patients will be vaccinated against varicella with Varilrix, one dose of 0,5 ml subcutaneously. If no response is documented to the first dose, the second dose of 0,5 ml can be administered.

    Outcomes

    Primary Outcome Measures

    Humoral response to vaccination
    Cell-mediated response to vaccination
    Number and severity of adverse events to vaccination

    Secondary Outcome Measures

    Full Information

    First Posted
    February 10, 2015
    Last Updated
    October 18, 2019
    Sponsor
    Helsinki University Central Hospital
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02383797
    Brief Title
    Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox
    Official Title
    Immunodeficiency in Cartilage-hair Hypoplasia: Correlation With Pulmonary Disease, Infections and Malignancy
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    October 2019
    Overall Recruitment Status
    Unknown status
    Study Start Date
    February 2015 (undefined)
    Primary Completion Date
    October 2019 (Actual)
    Study Completion Date
    February 2020 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Helsinki University Central Hospital

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    The investigatoris will carefully select cartilage-hair hypoplasia patients unexposed to varicella (VZV) and immunize patients in a controlled manner with VZV vaccine. Patients will be selected on the basis of disease severity and the degree of immunodeficiency (including CD4+ T-cell counts). Any potential complication of VZV immunization, such as rash, will be discussed with the patients/caregivers beforehand and acyclovir will be used to treat any VZV related symptoms, consistent with the current practices. The investigators will verify the development of immune response to vaccination by testing for VZV antibodies and cell-mediated immunity.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cartilage-hair Hypoplasia

    7. Study Design

    Primary Purpose
    Prevention
    Study Phase
    Phase 4
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    5 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Cartilage-hair hypoplasia (CHH)
    Arm Type
    Experimental
    Arm Description
    Selected CHH patients will be vaccinated against varicella with Varilrix, one dose of 0,5 ml subcutaneously. If no response is documented to the first dose, the second dose of 0,5 ml can be administered.
    Intervention Type
    Biological
    Intervention Name(s)
    Varilrix
    Other Intervention Name(s)
    ATC J07BK01
    Intervention Description
    Vaccination against varicella in selected cartilage-hair hypoplasia patients
    Primary Outcome Measure Information:
    Title
    Humoral response to vaccination
    Time Frame
    4-6 weeks post-vaccination
    Title
    Cell-mediated response to vaccination
    Time Frame
    4-6 wks post-vaccination
    Title
    Number and severity of adverse events to vaccination
    Time Frame
    0-60 days

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    12 Months
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: genetically confirmed cartilage-hair hypoplasia diagnosis age >12 months no history of chickenpox informed concent of the patient/caregiver. Exclusion Criteria: positive serum IgG for varicella zoster virus low CD4+ cell counts (<15% or <200 cells/mm3) clinical or laboratory signs of severe immunodeficiency ongoing intravenous or subcutaneous immunoglobulin treatment
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Outi Mäkitie, PhD
    Organizational Affiliation
    Helsinki University Central Hospital and University of Helsinki, Folkhälsan Research Centre, Helsinki Finland
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    32849667
    Citation
    Vakkilainen S, Kleino I, Honkanen J, Salo H, Kainulainen L, Grasbeck M, Kekalainen E, Makitie O, Klemetti P. The Safety and Efficacy of Live Viral Vaccines in Patients With Cartilage-Hair Hypoplasia. Front Immunol. 2020 Aug 11;11:2020. doi: 10.3389/fimmu.2020.02020. eCollection 2020.
    Results Reference
    derived

    Learn more about this trial

    Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox

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