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Could early markers unlock treatments for a rare childhood disease?

NCT ID NCT07709728

Knowledge-focused Sponsor: University of Nottingham Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 16, 2026 · Last updated Jul 17, 2026 · Updated 1 time

Summary

This study follows babies and young children (up to age 5) who have a genetic diagnosis of ataxia telangiectasia (A-T), a rare disease that affects movement and immunity. Researchers use brain and lung scans, blood tests, and movement analysis to spot early signs of disease progression. The goal is to find reliable markers that could be used in future trials of early-life treatments.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this study could identify early signs of disease progression in young children with ataxia telangiectasia, paving the way for future treatments that start before symptoms appear.
What could go wrong
This is an observational study, not a treatment trial. It may not find clear markers, and results might not apply to all children with A-T.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 56 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Aug 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Participants with A-T will be recruited principally via the National Paediatric A-T Clinic based at Nottingham University Hospitals NHS Trust. A second route for recruitment is the A-T Society, a UK charity that provides support and advocacy for people with A-T and their families. Participants without A-T or other conditions described in the exclusion criteria, will be infants and young children undergoing general anaesthesia at Nottingham University Hospitals NHS Trust for minor surgical procedures (e.g. circumcision, orchidopexy) or diagnostic MRI.

Ages

0 to 5 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

1. Participants with A-T * Inclusion criteria * 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. 2. Participants without A-T * Inclusion criteria * 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 * Exclusion criteria * Diagnosis of any neurological or neurodevelopmental disease * Diagnosis of any other significant chronic childhood illness * On any long-term prescribed treatments

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Conditions

The condition(s) this trial relates to.

ataxia telangiectasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Ataxia telangiectasia

How to take part

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  1. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

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