Abu Dhabi hospital to conduct Phase 3 trials of novel thalassemia treatment

A private hospital in Abu Dhabi will join Phase 3 global clinical trials of a novel first-in-class thalassemia treatment.

Thalassemia is an inherited blood disorder which affects the body’s ability to produce haemoglobin and red blood cells. It is among the most common genetic diseases worldwide and highly prevalent in the region.

Abu Dhabi’s Burjeel Medical City is preparing to begin two Phase 3 clinical trials of a new, first-in-class enzyme-activating drug: Mitapivat (AG-348). The new drug directly affects the survival of red blood cells and has already demonstrated potential in earlier trials for transforming the treatment of thalassemia by improving the hallmark of the disease – chronic haemolytic anaemia.

“The Phase 3 programme will be done in partnership with several health centres in the US and Europe,” Dr Khaled Musallam, group chief research officer at VPS Healthcare and principal investigator on the trials, told Khaleej Times.

He noted the Phase 3 programme would evaluate Mitapivat in two types of thalassemia with relatively different clinical needs.

“One trial will be conducted in transfusion-independent patients with the aim of increasing haemoglobin level and improving quality of life, and the other trial will be conducted on transfusion-dependent patients with the aim of decreasing transfusion requirement and thus decreasing the burden of the disease on the patient and healthcare system. Advances in the management of thalassemia would have significant benefits for the public health sector overall, as well as for patients and their families.”

The global trials will involve more than 400 volunteers: 240 transfusion-dependent and 171 non-transfusion-dependent thalassemia adult patients.

“In the UAE, we will rely on standard recruitment channels through our large healthcare network and referrals from colleagues taking care of thalassemia patients.”

The two trials, Dr Musallam added, will have 24- and 48-months core treatment periods, respectively, and extend up to five years.

John Sunil, CEO of Burjeel Hospitals in Abu Dhabi, underlined: “We are always ready to bring on cutting-edge technologies and attract talented physicians and scientists to our medical facilities to offer state of the art treatment options for patients. This becomes more imperative for rare diseases, which are often left with poor access to optimal management and chronic holistic care.”

Meanwhile, last month the hospital and HH Sheikh Sultan Bin Khalifa Al Nahyan Humanitarian and Scientific Foundation entered a collaboration to advance the care for thalassemia patients.

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