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
About this trial
This is an interventional prevention trial for Cartilage-hair Hypoplasia
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
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
NCT ID
NCT02383797
First Posted
February 10, 2015
Last Updated
October 18, 2019
Sponsor
Helsinki University Central Hospital
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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