Abu Dhabi to begin clinical trials to develop new thalassemia treatment

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

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

The Phase 3 trials for adult patients with thalassemia aim to test the effectiveness and safety of a new drug, Mitapivat.

Mitapivat is a US- and EU-approved drug to manage pyruvate kinase deficiency. It recently demonstrated potential in earlier trials for transforming thalassemia treatment as well.

The Phase 3 programme would 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. The Energize trial will start with a typical screening period and run for 24 weeks.

The Energize-T study aims to evaluate whether the same drug can decrease transfusion requirements in transfusion-dependent thalassemia patients. This trial will run for 48 weeks, core treatment periods for primary results, followed by five-year extensions.

The trials will be conducted at Burjeel Medical City, and include clinical, radiologic, and laboratory assessments,  Abu Dhabi Media Office reported on Monday.

“Abu Dhabi was able to play a pivotal role in developing medicines and conducting clinical trials with several international partners,” said Dr Asma Al Mannaei, executive director, research centre and innovation at Department of Health – Abu Dhabi.

“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.

Clinical trials in Abu Dhabi last year increased 27 per cent, totalling 71 compared to 56 trials in 2021, she added.

“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 Al Mannaei said.

Read: UAE to begin Covid-19 vaccine trials with 15,000 volunteers

Dr Khaled Musallam, group chief research officer at Burjeel Holdings noted that 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 added.