ClinicalTrials.gov record NCT07709728
Biomarkers for Babies and Young Children With Ataxia Telangiectasia
Biomarkers for Babies and Young Children With Ataxia Telangiectasia (The BOBCAT Study)
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This observational cohort follows infants and children aged 0–5 with a genetic diagnosis of Ataxia Telangiectasia and age-matched controls. The study collects brain and lung imaging, blood tests, movement recordings, and developmental assessments over up to four years to look for early measurable changes. Controls typically have a single visit with a blood test; children with A-T may have multiple visits including MRI, movement assessment, and blood sampling.
What it is studying. The study is trying to find and track quantitative brain and lung imaging measures, blood markers, and movement-related measurements that change over time in pre-symptomatic infants and very young children with a genetic diagnosis of Ataxia Telangiectasia. It also tests whether repeated MRI and other biomarker measurements can be collected reliably in this age group. The goal is to identify candidate biomarkers that could be measured in future early-life intervention studies.
What the researchers hope to learn. Identify imaging, blood, and movement measures that reflect early disease progression in infants and young children with A-T. Assess the feasibility of collecting repeated MRI and other biomarker data in infancy and early childhood. Generate candidate biomarkers to inform the design of future early-life intervention trials.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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
Exclusion criteria
- 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
- NB - co-recruitment to other observational studies or trials is permitted. If a family chooses to enrol their child in an interventional study targeting neurodegeneration, provided that the intervention trial allows co-recruitment, we would like to retain the participant in the BOBCAT study until its conclusion. In this circumstance, the child's data would not be considered as part of the natural history dataset but would instead be used to demonstrate the feasibility of collecting longitudinal quantitative imaging and other biomarker data in people with A-T during infancy and early childhood.
- Participants without A-T
- Child undergoing general anaesthesia at Nottingham University Hospitals NHS Trust for minor surgical procedures or diagnostic MRI.
- Aged 0-5 years (to match the age range of participants with A-T throughout the longitudinal study).
- Parents/ guardians able to give informed consent
- Diagnosis of any neurological or neurodevelopmental disease
- Diagnosis of any other significant chronic childhood illness
- On any long-term prescribed treatments
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
A‑T participants may attend up to four study visits over about four years for brain MRI (including natural‑sleep MRI when used), neurological and developmental assessments, quantitative movement recordings, and blood tests for biomarker analysis; lung MRI may be attempted where feasible. Control children have a single visit for a blood test (taken while they are under anaesthesia for their planned procedure). Co‑recruitment to other observational studies or trials is permitted; if a child later joins an interventional trial targeting neurodegeneration in A‑T (and that trial allows co‑recruitment), their BOBCAT data would be retained but not counted in the natural history dataset.
How the study is structured. An observational, longitudinal cohort. Children with A-T may attend up to four visits across a period of up to four years for brain MRI (including structural, spectroscopy, and diffusion scans), lung MRI where feasible, quantitative movement recordings, neurological/developmental assessments, and blood sampling. Age-matched control children attend a single visit for a blood sample. Data are collected but no experimental treatment is given.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Ataxia Telangiectasia. Does it apply to my diagnosis?
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on spinocerebellar ataxia
Important dates
- Study start
- Sep 1, 2026 (estimated)
- Primary completion
- Aug 31, 2029 (estimated)
- Study completion
- Aug 31, 2029 (estimated)
- Target enrollment
- 56 participants (estimated)
- Last updated on registry
- Jul 16, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- University of Nottingham
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins.
Terms used on this page · explanations by MedicalUpdates.ai
- biomarkers
- Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- Contraindication
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- MRI
- A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
- interventional study
- A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
- natural history
- A study that follows how a condition changes over time without assigning an experimental treatment. It may involve study visits and tests, and is often used to plan future trials.
- Inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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