Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)
Status:
Completed
Trial end date:
2008-03-01
Target enrollment:
Participant gender:
Summary
Ataxia Telangiectasia (AT) is an autosomal recessive inherited condition caused by mutations
in the ATM gene1. Patients suffer from neuro-degenerative problems, usually commencing in the
second year of life, and affecting predominantly the cerebellum. They also develop the
characteristic superficial telangiectases. Between 60 and 80% of affected children are
immunodeficient. This is associated with deficiency of immunoglobulin A (IgA ) 2, of IgG23
and of antibody responses to pneumococcal polysaccharides4. Patients suffer recurrent
sino-pulmonary infections but a recent study suggests poor correlation between immune status
and immunological parameters5. If uncontrolled, recurrent pulmonary infections can contribute
to the development of chronic lung disease and bronchiectasis. Preventative management
includes continuous prophylactic antibiotic treatment in some with the need for replacement
immunoglobulin therapy in only a small proportion of cases. Antibiotics have been reasonably
effective in this situation but the emergence of resistance amongst community acquired
pneumococcal isolates is a cause for concern. Appropriate immunisation strategies may also
have a role.
This study is designed to look at antibody responses in a one versus two dose regimen in a
cohort of AT patients recruited through the AT Society a national charitable organisation
involved in providing support to families with this condition and in fostering education and
research in the field.
Phase:
Phase 3
Details
Lead Sponsor:
Institute of Child Health
Collaborator:
Great Ormond Street Hospital for Children NHS Foundation Trust