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Trial record 1 of 2 for:    Congenital Varicella Syndrome
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Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox

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ClinicalTrials.gov Identifier: NCT02383797
Recruitment Status : Unknown
Verified March 2017 by Paula Klemetti, Helsinki University Central Hospital.
Recruitment status was:  Enrolling by invitation
First Posted : March 9, 2015
Last Update Posted : March 3, 2017
Sponsor:
Information provided by (Responsible Party):
Paula Klemetti, Helsinki University Central Hospital

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

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Study Type : Interventional  (Clinical Trial)
Estimated 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
Estimated Primary Completion Date : February 2019
Estimated Study Completion Date : February 2019


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




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 ]


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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

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

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Responsible Party: Paula Klemetti, MD, PhD, Consultant in pediatric gastroenterology and immunology, Helsinki University Central Hospital
ClinicalTrials.gov Identifier: NCT02383797     History of Changes
Other Study ID Numbers: TYH2013336
First Posted: March 9, 2015    Key Record Dates
Last Update Posted: March 3, 2017
Last Verified: March 2017

Additional relevant MeSH terms:
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Immunologic Deficiency Syndromes
Congenital Abnormalities
Hirschsprung Disease
Osteochondrodysplasias
Immune System Diseases
Digestive System Abnormalities
Digestive System Diseases
Megacolon
Colonic Diseases
Intestinal Diseases
Gastrointestinal Diseases
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn