The Aplastic Anaemia Trust have been asked by the team at King's to let our community know that they are currently looking for people to participate in this clinical trial. 

This is an early phase (1) clinical trial for patients with Aplastic Anaemia. The trial aims to investigate the safety of using patient’s own (‘autologous’) T regulatory cells (a type of immune cell) which are ‘expanded’ in a laboratory setting and then given back to the patient through an IV infusion. The trial is called TIARA and is a UK based trial at King’s College Hospital in London. The trial will recruit up to 12 patients over a 2 year period.

The primary purpose of the trial is to assess the safety and optimal dose of this new type of treatment and is available to AA patients who have failed, or are considered ineligible for immunosuppressive therapy or other treatments, and who meet the eligibility criteria (listed below). A secondary aim of the study will also be to assess the clinical response over time.

What's involved?

Participants in the trial will receive up to two doses of the experimental treatment over a two-week period (there is no placebo). The new treatment is manufactured from patients’ own cells and these cells are collected through a procedure called leukapheresis.

This trial launched in Autumn 2022, and participants are being treated one at a time. Each group of three patients will receive up to two infusions at a set dose level and are closely monitored through follow up visits at the hospital. If it is deemed safe to do so, the next group of patients will receive a slightly higher dose.

Patients will need to travel to King’s College Hospital in London for all trial related visits. There are approximately 11 visits in total plus a two-night stay, but the doctor may ask for patients to attend on other occasions too. Travel expenses can be covered.

Can I take part?

This trial is designed for adult patients (age 18 years or older) with Aplastic Anaemia who have either not responded to or are not eligible for standard treatments including stem cell transplant and ATG. Some of the main criteria for taking part in this trial are listed below, you can speak to your clinician about whether you meet these inclusion criteria. There will be other, additional criteria which you will be assessed against when you express an interest in the trial.

To be considered for inclusion in this trial you will need to meet these criteria:

  1. Acquired idiopathic AA
  2. No evidence of constitutional/inherited AA
  3. Very severe, severe or non-severe AA
  4. Lack a suitable donor (or ineligible) for a stem cell transplant
  5. Transfusion dependent
  6. Failed or ineligible for a course of ATG and CSA

How to take part

If you are interested in participating in the study and would like to know more about whether you might be a suitable candidate for inclusion, you need to contact your clinical team and ask them to contact Dr Shreyans Gandhi at Kings College Hospital. 

If your clinical team need Dr Gandhi’s contact details, they can email Ellie, our Interim CEO, at [email protected]

Patients who express an interest will be screened for suitability and provided with more detailed information. 

With thanks to our supporters.

The TIARA clinical trial is funded by The Aplastic Anaemia Trust and LifeArc.

The AAT are able to fund exciting research projects like this because of our community's generous donations and fundraising.
Thank you so much to all our supporters.