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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02383797
Recruitment Status : Active, not recruiting
First Posted : March 9, 2015
Last Update Posted : October 22, 2019
Sponsor:
Information provided by (Responsible Party):
Paula Klemetti, Helsinki University Central Hospital

Study Description
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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.

Condition or disease Intervention/treatment Phase
Cartilage-hair Hypoplasia Biological: Varilrix Phase 4

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Immunodeficiency in Cartilage-hair Hypoplasia: Correlation With Pulmonary Disease, Infections and Malignancy
Study Start Date : February 2015
Actual Primary Completion Date : October 2019
Estimated Study Completion Date : February 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Chickenpox

Arms and Interventions
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Arm Intervention/treatment
Experimental: Cartilage-hair hypoplasia (CHH)
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.
 Biological: Varilrix
Vaccination against varicella in selected cartilage-hair hypoplasia patients
Other Name: ATC J07BK01


Outcome Measures
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Primary Outcome Measures :
  1. Humoral response to vaccination [ Time Frame: 4-6 weeks post-vaccination ]
  2. Cell-mediated response to vaccination [ Time Frame: 4-6 wks post-vaccination ]
  3. Number and severity of adverse events to vaccination [ Time Frame: 0-60 days ]

Eligibility Criteria
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Ages Eligible for Study:   12 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02383797


Sponsors and Collaborators
Helsinki University Central Hospital
Investigators
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Study Director: Outi Mäkitie, PhD Helsinki University Central Hospital and University of Helsinki, Folkhälsan Research Centre, Helsinki Finland
More Information
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Responsible Party: Paula Klemetti, MD, PhD, Consultant in pediatric gastroenterology and immunology, Helsinki University Central Hospital
ClinicalTrials.gov Identifier: NCT02383797    
Other Study ID Numbers: TYH2013336
First Posted: March 9, 2015    Key Record Dates
Last Update Posted: October 22, 2019
Last Verified: October 2019
Additional relevant MeSH terms:
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Osteochondrodysplasias
Hirschsprung Disease
Immunologic Deficiency Syndromes
Immune System Diseases
Digestive System Abnormalities
Digestive System Diseases
Megacolon
Colonic Diseases
 Intestinal Diseases
Gastrointestinal Diseases
Congenital Abnormalities
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn