Sidra Medicine pioneering rare and complex disease clinical trials for children

Sidra Medicine, a key player in Qatar's healthcare landscape, is pioneering clinical trials to provide new hope for children with rare and complex diseases. As a member of the Qatar Foundation, Sidra Medicine's Clinical Trials Program is poised to become a leader in regional clinical research, focusing on offering personalized treatments for rare pediatric conditions.

This initiative aims to fill the long-existing gap in medical research among the Arab population, bringing innovative solutions to the forefront of pediatric healthcare in the region.

How does it work?

Clinical trials are the final stage in medical research, where new treatments are carefully tested for safety and effectiveness before they are made available to the wider population. At Sidra Medicine, these trials focus on children with rare diseases, offering them access to cutting-edge treatments, from new drugs to medical devices and therapies.

Every trial is voluntary, and participants may receive life-changing treatments that are otherwise unavailable.

As Prof. Khalid A. Fakhro, Chief Research Officer at Sidra Medicine, highlights: "We are building towards undertaking complex research, including critical Phase 1 through Phase 4 studies, which will establish us as a regional leader in clinical trials."

Sidra Medicine's Clinical Trials Program is unique in its approach to targeting Middle Eastern pediatric populations from the outset.

Dr. Chiara Cugno, Director of Advanced Cell Therapy Core at Sidra Medicine, explains: "Many current treatment options are ineffective because they are not designed with Arab genetics in mind. Our clinical trials program allows us to offer personalized experimental solutions for families with these genetic ancestries."

Why does it matter?

Sidra Medicine's work is more than just a medical breakthrough; it addresses a historical gap in clinical research within the Arab world. A lack of localized research has limited the development of effective treatments tailored to the population's unique needs. By leading clinical trials that focus on children of Arab, African-Arab, and Persian-Arab ancestry, Sidra Medicine is creating new treatment pathways and building a vital genetic knowledge database.

Dr. Iyabo Tinubu-Karch, Chief Executive Officer at Sidra Medicine, emphasizes this point: "Our clinical services in partnership with our research division have identified critical areas in pediatric health related to rare and genetic diseases. This will help us expand treatment options and redefine what is possible in pediatric medicine."

The significance of these efforts cannot be overstated, as the results of these trials could impact the future of healthcare for generations of children in Qatar and beyond. The program ensures that children no longer need to travel abroad to access experimental treatments, providing relief and hope for families across the region.

The context

Sidra Medicine operates within a network of global healthcare institutions and has already been selected for several pharmaceutical-sponsored clinical trials. These trials span various conditions, including endocrine disorders like Congenital Hyperinsulinism and Type 1 Diabetes, neuromuscular conditions like Spinal Muscular Atrophy, metabolic disorders, and pediatric cancers such as Low Grade Glioma.

One of the key strengths of Sidra Medicine's clinical trials is its strict adherence to international ethical standards. Every trial undergoes extensive review by an independent Institutional Review Board and Qatar's Ministry of Public Health to ensure the safety and well-being of the children involved.

Dr. Ahmed Al Hammadi, Acting Chief Medical Officer, states: "Sidra Medicine's commitment to personalized medicine thrives on the support of families. The outcome of one child's clinical trial has the potential to positively impact the care and well-being of many other children."