Clinical trials at Sidra Medicine offers hope to children with rare diseases

Doha, Qatar: Sidra Medicine, a member of Qatar Foundation, aims to transform precision healthcare through clinical trials, offering a new lease of hope to young patients with rare and complex diseases.

The Clinical Trials Program at Sidra Medicine will focus on clinical trials that will benefit children in the Arab region, particularly those withrare diseases that lack effective treatment.This initiative will position Sidra Medicine as a regional leader in clinical research, offering personalized treatment options to children with rare diseases.

Dr. Iyabo Tinubu-Karch, Chief Executive Officer at Sidra Medicine said: “At Sidra Medicine, we are entrusted with the care of Qatar’s most precious resource – its children. Our clinical services in partnership with our research division, has identified a number of 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. Having a clinical trials program, embodies our commitment towards advancing pediatric healthcare and pioneering new medications and therapies for the benefit of the people in Qatar and the region.”

Clinical trials are the final step of medical research, where new treatments undergo careful evaluation for safety and effectiveness before being made widely available. Everyone who participates in a clinical trial is a volunteer. The trials could offer children the chance to receive beneficial treatment whether it is a new drug, therapy, device or procedure—before it becomes widely available

Prof. Khalid A. Fakhro, Chief Research Officer at Sidra Medicine said: “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.1.This ambition is built upon years of excellence at diagnostic research where our teams made many fundamental discoveries in the cause and origin of diseases in our patients. We were able to achieve this in collaborationwith global academic and health partners, pharmaceutical companies and with the support of stakeholders such as Qatar Foundation (QF) and Qatar Precision Health Institute (QPHI). By becoming a regional leader in clinical trials,we will be able to bring innovative therapies to our patients, offering access to potentially life-changing outcomesto children with rare diseases."

A historical lack of research within the Arab population has led to a significant data gap, limiting the development of effective and personalized treatments. The clinical trials at Sidra Medicine will play a crucial role in addressing this gap, building a robust database of knowledge that will enable more effective and personalized treatment approaches tailored to the unique needs of the population.

Dr. Chiara Cugno, Director of Advanced Cell Therapy Core at Sidra Medicine said: "The Clinical Trial Program at Sidra Medicine will focus on representing Middle Eastern pediatric populations from the very beginning of the drug testing process, not as an afterthought. Many of the current treatment options are often ineffective because they have not been designed for those with Arabancestry, African-Arab or Persian-Arabgenetics in mind. Our clinical trials programwill allow us to change that, offering personalized experimental solutions and new hope, particularly for families with these genetic ancestries.”

Sidra Medicine’sclinical trials will be based on medicines, medical devices and medical procedures that have either been approved for adults but not yet studied for children or are not yet government approved. Initially, it willfocus on disorders and medical conditions like endocrine disorders such as Congenital Hyperinsulinism (CHI), Diabetes Type 1 (TD1); neuromuscular conditions like Spinal Muscular Atrophy (SMA); metabolic disorders like Homocystinuria (HCU) and pediatric cancers like as Low Grade Glioma (pLGG).

Sidra Medicine is part of a network of global healthcare institutions and has been selected as a site for several pharma sponsored industry clinical trials to date, which are at various stages of feasibility assessment.

Dr. Cugno continued: “Being chosen by international healthcare and pharmaceutical companies, also speaks to the credibility of our research, expertise and technologies. Most importantly, we are one of the few hospitals in the Middle East and Qatar where research is closely embedded with clinical care. We are also leading on research on theArab population, with several of our studies published in some of the world’s top-tier high-impact journals.”

Every clinical trial at Sidra Medicine will undergo intense examination, including review by an independentInstitutional Review board and Qatar’s Ministry of Public Health—to ensure the well-being of every patient enrolled, particularly children. The privacy and confidentiality of each family will be strictly maintained throughout the journey. This multi-layered approach, with multiple checkpoints throughout the process, will guarantee adherence to the highest international ethical and scientific standards.

Dr. Ahmed Al Hammadi, Acting Chief Medical Officer at Sidra Medicine said: “Sidra Medicine's commitment to personalized medicine thrives on the support of families. By voluntarily enrolling their children in research studies andclinical trials, families become partners in helping us develop the treatments of the future.The outcome of one child’s clinical trial has the potential to positively impact the care, treatment and wellbeing of not only that child but many other children - not only in the same family but for families everywhere. As a volunteer, your child is playing an important role in helping us find better ways to prevent, detect and treat diseases. It is our hope, that in the future many families will no longer need to relocate abroad to participate in clinical trials, as these opportunities will be available in Qatar.”