Here are some important studies. Please do take part in them if you fit the criteria.
INSPIRE AT: Longitudinal Study Comparing the Natural History of Ataxia Telangiectasia in Children to Healthy Comparators
New opportunity to join an important new study to help improve the design of future clinical trials. Please sign up!
This study will involve wearable devices to track movement (not dissimilar to a watch or Fitbit), and for some, there will also be the chance to include siblings, cousins or friends who do not have AT too.
Location: Nottingham, UK; Baltimore, Boston, Houston and Los Angeles, USA
Investigator: Insmed Incorporated, a global biopharmaceutical company with a focus of patients with serious and rare diseases.
Purpose: The aim of this non-promotional ‘natural history’ and ‘exploratory biomarker’ study is to help improve our understanding of classic ataxia telangiectasia (AT) so we can find out whether there are better ways to monitor and treat children with this condition.
Eligibility: The study will be carried out in about 45 children aged 1 to 10 years with a diagnosis of classic AT and about 24 children of the same age without AT.
Assessments: Children will be assessed 5 times over a period of up to 24 months to identify changes in health-related day-to-day life (daily activities and quality of life), growth and development, physical activity, and hand function/co-ordination. The study will also look for biological markers (biomarkers) in the blood that could be used to monitor disease progression. Participants will not be asked to take any new medicines during the study.
Benefits: Results from this study could potentially help us design new clinical trials and develop new treatments for children with classic AT. For more information about the study please go to: INSPIRE AT study
If you would like to join this study, please email [email protected]
The AT team at Nottingham University Hospital will check your child’s eligibility against the study criteria. Stefanie, at NUH, will then contact you directly.
Engage AT Study
Engage AT is a study being undertaken by Dr Rupinder Bajwa at Nottingham University to help make research fairer and easier for people with AT and their families,
especially those from diverse backgrounds. Right now, there’s no regular cancer screening for people with AT, and some communities face extra challenges joining research. Rupinder will be running workshops to listen, learn, and co-create better ways to make research fairer and more inclusive. If you or a loved one with AT, would live to be involved please contact Rupinder Bajwa via email: 📧 [email protected] or 📞 0115 748 561. Find out more.
Trial Readiness in Ataxia Telangiectasia (TREAT-AT)
If you are aged 16 years and over, and have AT, with a specific gene ‘mutation’ of c.5673-1050A>G, c.8418+681A>G; p.Glu2807 ValfsTer4, and c.6807G>A, then neurologists Professor Rita Horvath and Dr Anke Hensiek would love to hear from you.They are conducting a study to assess the neurological progression of AT to
select the best outcome measure for a future clinical trial. As a participant in this study, you will be asked to attend three study visits, over two years, in Cambridge. You will be asked to give blood/tissue samples, complete physiological and cognitive assessments and fill out questionnaires about your quality of life. All travel expenses will be reimbursed. If you think you might be eligible and are interested in taking part, or would like any further information, please contact the study team ASAP, using the contact details here: [email protected]; or 📞 01223 331506. Recruitment for this study will close on the 31st December 2026.
Study on Culturally Sensitive Communication
King’s College London is conducting a study focused on improving communication between researchers, healthcare professionals, and children and young people living with complex or chronic health conditions, as well as their families. The project aims to develop guidance on the use of culturally sensitive and appropriate language, with particular emphasis on improving the inclusion of children, young people and families from ethnic minority backgrounds, who remain underrepresented in research and patient involvement activities. As part of this work, the research team will hold two online co‑production workshops: one for parent/carers and one for children and young people (including siblings). These sessions will
provide a structured space for families to share their perspectives on discussing sensitive health topics and on making research more inclusive and accessible.
The study is seeking families who identify as belonging to an ethnic minority group (defined as non‑White British) and who have experience of caring for, or living with, a serious illness or chronic condition. The workshops are scheduled for mid‑February. Families interested in participating or wishing to receive further information are invited to contact Victoria at [email protected]
