The Aplastic Anaemia Trust would like to inform our community about a new clinical trial which is currently recruiting patients.

This is a early (Phase 1/2) trial to investigate the safety of a new antibody called REGN7257 (Anti-Interleukin 2 Receptor Subunit Gamma [IL2RG] Monoclonal Antibody). This is a global study which will recruit up to 48 patients to the potential new treatment.

The primary purpose of the trial is to assess the safety and appropriate dosing level of this new antibody, and as a secondary aim the study will also assess the clinical response over time.

What is involved?

Participants in the trial will all receive a dose of the new antibody (there is no placebo). 

This trial is set to launch on March/April 2021, patients will be treated in staggered groups. Each group of volunteers will receive a small dose of REGN7257, and be closely monitored through follow up sessions, before the next group are administered a slightly increased dose.

In the UK, patients will need to travel to King’s College Hospital, London to receive a single dose of the antibody, and to attend follow up sessions. Travel and accommodation expenses will be covered.

Can I take part?

This trial is designed for adult patients with severe aplastic anaemia whose disease has not responded to immunosuppressive therapy, or who have relapsed after receiving immunosuppressive therapy. Some of the main criteria for taking part in this trial are listed below, there will be other, additional criteria which you will be assessed against when you express an interest in the trial.

To take part you will need to meet these criteria:

• Severe aplastic anaemia that is refractory to or has relapsed while on standard of care immunosuppressive therapy
• Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient
• Adequate hepatic and renal function as defined in the protocol

Exclusion criteria for this study include:

• Diagnosis of Fanconi anaemia as defined in the protocol
• Evidence of myelodysplastic syndrome as defined in the protocol
• Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of significant hemolysis or history of PNH-associated thrombosis
• Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior to dosing
• Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to dosing
• Treatment with eltrombopag or investigational thrombopoietin receptor agonist, Granulocyte Colony-Stimulating Factor (G-CSF), or an androgen (eg, danazol), within 2 weeks prior to dosing
• HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit
• Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI

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 Austin Kulasekararaj at Kings Hospital. If your clinical team need Austin's contact details, they can email Stevie, our CEO, at [email protected].

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