Ground-breaking Thalassemia Clinical Trials to Begin in Abu Dhabi

Strengthening the emirate’s positioning as a life science hub

Trials will provide an opportunity for eligible patients in the region to get screened and be part of studies aimed at developing new thalassemia treatment

Abu Dhabi, UAE – 13 January 2023: As an incubator and hub for life-science research and innovation, Abu Dhabi is set to begin clinical trials to transform the way thalassemia is being treated, kicking it off with two global clinical trials. 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, Mitapivat. Conducted at Abu Dhabi-based Burjeel Medical City, the clinical trials offer new hope for thalassemia patients in the country. The trials will be conducted in collaboration with several centres and multiple sites across the United States of America (USA), the European Union (EU), and Asia, enabling access to state-of-the-art therapies from the outset of clinical development.

Mitapivat (Agios Pharmaceuticals; MA, USA) is already approved in the USA and the EU for managing hemolytic anemia in adults with pyruvate kinase deficiency, however, recently, it has also demonstrated potential in earlier trials for the treatment of thalassemia.

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

Commenting on this breakthrough, Dr. Asma Al Mannaei, Executive Director, Research Centre and Innovation at Department of Health – Abu Dhabi, said: “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. 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 helping all members of our community, and across the globe.”

Last year, clinical trials in Abu Dhabi saw an increase of 27%, 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.

Dr. Khaled Musallam, Group Chief Research Officer, Burjeel Holdings and principal investigator on the trials stated: “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 prioritizing the latest developments in treatment, research and development and actively participating in projects with some of the world’s leading institutions. 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.”

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. 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 5-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.

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

For further information on the trials and eligibility, please contact 0564104317.