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

Helsinki University Central Hospital (HUCH)

Status and phase: Unknown; Phase 4

Conditions: Cartilage-hair Hypoplasia

Treatments: Biological: Varilrix

Study type: Interventional

Funder types: Other

Identifiers

NCT02383797

TYH2013336

Details and patient eligibility

About

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.

Enrollment: 5 patients

Sex: All

Ages: 12+ months old

Volunteers: No 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

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

Cartilage-hair hypoplasia (CHH)

Experimental group

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.

Treatment:

Biological: Varilrix