Biomarkers for Babies and Young Children With Ataxia Telangiectasia
ConditionAtaxia Telangiectasia
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-5
SponsorUniversity of Nottingham
About this trial
The goal of this observational study is to identify progressive changes of quantitative brain and lung imaging, serum and movement-related biomarkers reflecting disease progression in pre-symptomatic infants and very young children (0-5 yo) with a genetic diagnosis of A-T, that could be used in future early-life intervention trials.
Eligibility criteria
Qualifiers
Participants with A-T
* Genetic diagnosis of Ataxia Telangiectasia
Aged under two years old at the time of first recruitment
Parents/ guardians able to give informed consent
Disqualifiers
Contraindication to MRI
Diagnosis of any other neurogenetic disease
On approved treatment targeting neurodegeneration in A-T at the time of first recruitment
Participating in the trial of novel therapy targeting neurodegeneration in A-T at the time of first recruitment
Trial design
Treatments tested in this trial
- Not listed
Trial groups
56 Participants
are grouped into 2 trial groupsLocations
Sponsors and collaborators
Source ClinicalTrials.gov