UAE: Two Phase 3 thalassemia clinical trials to begin in Abu Dhabi, aim to develop new treatment

In another milestone in the health sector, the UAE Capital is set to begin two global clinical trials to transform the way thalassemia is being treated.

Thalassemia is a group of inherited blood disorders that affect the body’s production of haemoglobin and red blood cells. It is among the world’s most common genetic diseases and is highly prevalent in the region.

Under the supervision and support of the Department of Health – Abu Dhabi (DoH), Energise and Energise-T – two Phase 3 trials for adult patients with thalassemia, aim to test the effectiveness and safety of a new drug called Mitapivat.

Conducted at Burjeel Medical City in Abu Dhabi, the clinical trials offer new hope for thalassemia patients in the country. They will provide an opportunity for eligible patients in the region to get screened and be part of studies aimed at developing new thalassemia treatments.

The trials will be conducted in collaboration with several centres and multiple sites across the US, the EU, and Asia, enabling access to state-of-the-art therapies from the outset of clinical development.

Mitapivat is approved in the US and the EU for managing haemolytic anaemia in adults with a pyruvate kinase (PK) deficiency. Recently, it has demonstrated potential in earlier trials for the treatment of thalassemia.

Energise, Energise-T studies

In the upcoming stage, the Phase 3 programme would evaluate the drug in two types of thalassemia in adults, with relatively different clinical needs. The Energise study aims to evaluate whether Mitapivat can effectively and safely improve haemoglobin levels in non-transfusion-dependent thalassemia patients who do not require regular transfusions. Previous studies from Burjeel Medical City researchers have shown that low haemoglobin levels can lead to significant morbidity and may lead to death. Furthermore, Energise-T aims to evaluate whether the same drug can decrease transfusion requirements in transfusion-dependent thalassemia patients.

“As the Emirate’s healthcare regulator, we are relentlessly working on cementing Abu Dhabi’s position as a pioneering healthcare destination globally and the region’s hub for research and innovation in life science,” said Dr Asma Al Mannaei, executive director, Research Centre and Innovation at DoH – Abu Dhabi.

“Abu Dhabi was able to play a pivotal role in developing medicines and conducting clinical trials with several international partners. Today, we are witnessing the launch of new clinical trials, so that we continue our efforts as we aim to further improve healthcare services in Abu Dhabi, and help all members of our community, and across the globe.”

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