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

NCT02383797 | Unknown Phase 4

• 1 other identifier: TYH2013336
• Study Type: interventional
• Target: 5
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Cartilage-hair Hypoplasia

Outcome Measures

Primary Outcomes (3)

• Humoral response to vaccination

  4-6 weeks post-vaccination

• Cell-mediated response to vaccination

  4-6 wks post-vaccination

• Number and severity of adverse events to vaccination

  0-60 days

Study Arms (1)

Cartilage-hair hypoplasia (CHH)

EXPERIMENTAL

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

Interventions

Varilrix BIOLOGICAL

Vaccination against varicella in selected cartilage-hair hypoplasia patients

Also known as: ATC J07BK01

Cartilage-hair hypoplasia (CHH)

Eligibility Criteria

• Age: 12 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Helsinki University Central Hospital: lead

Related Publications (1)

• 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.

  PMID: 32849667 DERIVED

MeSH Terms

Conditions

Cartilage-hair hypoplasia

Study Officials

• Outi Mäkitie, PhD

  Helsinki University Central Hospital and University of Helsinki, Folkhälsan Research Centre, Helsinki Finland

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD, Consultant in pediatric gastroenterology and immunology

Study Record Dates

• First Submitted: February 10, 2015
• First Posted: March 9, 2015
• Study Start: February 1, 2015
• Primary Completion: October 1, 2019
• Study Completion: February 1, 2020
• Last Updated: October 22, 2019

Record last verified: 2019-10