Abu Dhabi to start trials for groundbreaking thalassemia treatment

Abu Dhabi is set to begin clinical trials to transform the way thalassemia is being treated.

Under the Department of Health - Abu Dhabi’s (DoH) supervision and support, the Phase 3 trials for adult patients with thalassemia, ENERGIZE and ENERGIZE-T, aim to test the effectiveness and safety of a new drug called Mitapivat.

The trials will be conducted at Burjeel Medical City.

Mitapivat is already approved in the U.S. and in the European Union for its ability to manage pyruvate kinase deficiency, however, recently, it has demonstrated potential in earlier trials for transforming the treatment of thalassemia.

In the upcoming stage, the Phase 3 programme will evaluate the drug in two types of thalassemia with relatively different clinical needs.

The ENERGIZE 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.

Meanwhile, ENERGIZE-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.

"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. Furthermore, the UAE was one of the first countries to participate in clinical trials of Covid-19 vaccines, witnessing the largest clinical trial on the Chinese Sinopharm vaccine, covering more than 32,000 volunteers," she added.

Dr. Khaled Musallam, Group Chief Research Officer, Burjeel Holdings, said that Abu Dhabi is now at the forefront of leading-edge innovations.

"If these studies meet their endpoints, this could be a game changer to 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," he explained.

The ENERGIZE trials will start with a typical screening period and run for 24 weeks, while ENERGIZE-T will run for 48 weeks, core treatment periods for primary results, followed by 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.

With support of @DoHSocial, the third phase of global clinical trials will be conducted in #AbuDhabi to assess the effectiveness and safety of new thalassemia treatment Mitapivat, enhancing the emirate’s position as an incubator of innovation and a hub for life-science research. pic.twitter.com/ZxIHxhgZXV

— مكتب أبوظبي الإعلامي (@ADMediaOffice) February 13, 2023