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

The blood disorders are among the world’s most common genetic diseases, and are highly prevalent in the region


Ashwani Kumar

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Published: Mon 13 Feb 2023, 1:02 PM

Last updated: Mon 13 Feb 2023, 2:56 PM

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.”

71 clinical trials in 2022

Last year, clinical trials in Abu Dhabi saw an increase of 27 per cent, reaching 71 clinical trials in 2022, compared to 56 trials in 2021. The UAE was one of the first countries to participate in clinical trials of Covid-19 vaccines.

Dr Khaled Musallam, group chief research officer, Burjeel Holdings and principal investigator on the trials said: “With DoH’s full support and encouragement, Abu Dhabi is now at the forefront of leading-edge innovations. Burjeel Medical City is committed to improving the health and well-being of patients by prioritising the latest developments in treatment, research and development, and actively participating in projects with some of the world’s leading institutions.”

According to Dr Musallam, this milestone comes only after stringent and thorough regulatory approvals to ensure the facility meets the necessary global standards for clinical trials.

“If these studies meet their endpoints, this could be a game changer for how the disease is being treated today. A disease-modifying therapy can address persisting unmet needs of patients in the UAE and around the world,” Dr Musallam noted.

The Energise trials will start with a typical screening period and run for 24 weeks, while Energise-T will run for 48 weeks, followed by core treatment periods for primary results, and then five-year extensions. The trials will include clinical, radiologic, and laboratory assessments, which will be conducted at Burjeel Medical City using state-of-the-art technology.


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