Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)

This study has been completed.
Great Ormond Street Hospital for Children NHS Foundation Trust
Information provided by:
Institute of Child Health Identifier:
First received: April 7, 2008
Last updated: April 10, 2008
Last verified: April 2008

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.

Condition Intervention Phase
Ataxia Telangiectasia (AT)
Drug: Conjugated pneumococcal vaccine (Prevenar)
Phase 3

Study Type: Interventional
Study Design: Primary Purpose: Basic Science
Official Title: Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)

Resource links provided by NLM:

Further study details as provided by Institute of Child Health:

Primary Outcome Measures:
  • Immunogenicity of vaccine

Secondary Outcome Measures:
  • Incidence of adverse reactions to vaccine

Enrollment: 30
Study Start Date: June 2006
Study Completion Date: March 2008
Intervention Details:
    Drug: Conjugated pneumococcal vaccine (Prevenar)
    Other Name: Prevenar

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Genetically confirmed diagnosis of AT
  Contacts and Locations
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Please refer to this study by its identifier: NCT00656409

United Kingdom
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Institute of Child Health
Great Ormond Street Hospital for Children NHS Foundation Trust
  More Information Identifier: NCT00656409     History of Changes
Other Study ID Numbers: 04MI07 
Study First Received: April 7, 2008
Last Updated: April 10, 2008
Health Authority: United Kingdom: Research Ethics Committee

Keywords provided by Institute of Child Health:
Ataxia telangiectasia,
Conjugated pneumococcal,
Immunogenicity ,

Additional relevant MeSH terms:
Cerebellar Ataxia
Cerebellar Diseases
Ataxia Telangiectasia
Brain Diseases
Cardiovascular Diseases
Central Nervous System Diseases
DNA Repair-Deficiency Disorders
Genetic Diseases, Inborn
Immune System Diseases
Immunologic Deficiency Syndromes
Metabolic Diseases
Nervous System Diseases
Neurocutaneous Syndromes
Neurologic Manifestations
Signs and Symptoms
Spinocerebellar Ataxias
Vascular Diseases
Heptavalent Pneumococcal Conjugate Vaccine
Immunologic Factors
Physiological Effects of Drugs processed this record on May 26, 2016