Ataxia Telangiectasia (AT)Phase 3NCT00656409
Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)
A Phase 3 study testing Conjugated pneumococcal vaccine (Prevenar) in people with Ataxia Telangiectasia (AT), run by Institute of Child Health at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Ataxia Telangiectasia (AT)
- What is being tested?
- Conjugated pneumococcal vaccine (Prevenar)
- Type of treatment
- Vaccines
- How many people
- 30 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jun 2006to Mar 2008
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Genetically confirmed diagnosis of AT
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
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.
- Conditions
- Ataxia Telangiectasia (AT)
- Being tested
- Conjugated pneumococcal vaccine (Prevenar)
- Keywords
- Ataxia telangiectasia,, Conjugated pneumococcal,, Prevenar,, Immunogenicity ,, Vaccine
- Medical subject headings
- Ataxia Telangiectasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Conjugated pneumococcal vaccine (Prevenar)Drug
Also called Prevenar
What is being measured
The primary outcome is the main question the study is designed to answer.
Immunogenicity of vaccine
Secondary outcomes (1)
Incidence of adverse reactions to vaccine
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2008 (estimated)
- Study startJun 2006
- Study completionMar 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ataxia Telangiectasia (AT)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Basic science
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institute of Child Health
- Sponsor type
- University, hospital or other
- Collaborators
- Great Ormond Street Hospital for Children NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00656409
- Sponsor's protocol ID
- 04MI07
- Status last verified
- Apr 2008
- First submitted
- 7 Apr 2008
- Last updated
- 11 Apr 2008
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)
- ClinicalTrials.gov ID
- NCT00656409
- Acronym / sponsor ID
- 04MI07
- Conditions
- Ataxia Telangiectasia (AT)
- Keywords
- Ataxia telangiectasia,, Conjugated pneumococcal,, Prevenar,, Immunogenicity ,, Vaccine
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Institute of Child HealthUniversity, hospital or other
- Collaborators
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Study start
- Jun 2006
- Study completion
- Mar 2008 (actual)
- First posted
- 11 Apr 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Genetically confirmed diagnosis of AT
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Conjugated pneumococcal vaccine (Prevenar)Also: Prevenar | Drug | – |
Primary outcome
Immunogenicity of vaccine
1 secondary outcome
Incidence of adverse reactions to vaccine
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2008.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Conjugated pneumococcal vaccine (Prevenar) in people with Ataxia Telangiectasia (AT), run by Institute of Child Health at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Apr 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Immunogenicity of vaccine. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Apr 2008.