Tribune News Network
Doha
Sidra Medicine, a member of Qatar Foundation, has successfully conducted Qatar’s first industry-sponsored pharmaceutical trial for paediatric congenital hyperinsulinism on a young patient.
The trial follows Sidra Medicine’s launch of its Clinical Trials Programme aimed at offering a new lease of hope to children with rare and complex diseases. It primarily focuses on clinical trials that will benefit children in the Arab region, particularly those with rare diseases that lack effective treatment.
Sidra Medicine was selected as one of 22 leading centres worldwide to participate in the sunRIZE Phase 3 clinical trial. Led by Rezolute, a biopharmaceutical company specialising in rare diseases, the trial underscores Sidra Medicine’s growing prominence in paediatric endocrinology and its commitment to advancing innovative, patient-centred care.
Congenital hyperinsulinism is a complex condition that causes life-threatening hypoglycemia in children due to excessive insulin production. Many patients are unresponsive to existing therapies and often require invasive pancreatic surgery, which can lead to lifelong complications such as diabetes and pancreatic insufficiency.
Prof Khalid Hussain, Division Chief of Endocrinology at Sidra Medicine, said: "This clinical trial represents a pivotal step in advancing care for congenital hyperinsulinism. Phase 3 trials compare new treatments to existing options in a diverse and larger patient population, providing evidence for regulatory approval based on effectiveness and safety. Our collaboration with Rezolute and international experts aims to develop a safer, non-invasive treatment option that could transform the lives of affected children and significantly reduce the need for surgery.”
The clinical trial was conducted on a three-month-old Qatari girl, who was admitted to Sidra Medicine upon being diagnosed with congenital hyperinsulinism. After previous therapies to treat her condition were not successful, the child’s parents enrolled her for the SunRIZE trial as a last possible option to find an effective treatment.
"We are extremely pleased with the results of the trial as the young patient has responded favourably to the new medication. After careful monitoring to ensure she was in good health and was not experiencing any post-medication side-effects or risks, she has been discharged. Her blood sugar levels are now in the normal range, and she is feeding normally. We will continue to monitor her progress in the clinic every three months. We would like to thank the girl’s parents for putting their trust in us to find the best possible care and cure for their child,” added Prof Hussain.
The Congenital Hyperinsulinism trial, approved by Qatar’s Ministry of Public Health, was conducted by Sidra Medicine’s Endocrinology and Clinical Trials Programme in collaboration with several internal departments, including Nursing, Pharmacy, IT, and the Institutional Review Board. Sidra Medicine is currently recruiting for more patients for the Phase 3 SunRIZE trial, which is open to children aged between three months until 18 years of age.
Dr Antonella Cioce, Clinical Trials Office Manager at Sidra Medicine, said: "This clinical trial highlights the exceptional teamwork and dedication at Sidra Medicine in advancing therapies for rare paediatric conditions.”
In addition to endocrine disorders such as Congenital Hyperinsulinism (CHI), Diabetes Type 1 (TD1), Sidra Medicine is also focusing on disorders and medical conditions such as neuromuscular conditions like Spinal Muscular Atrophy (SMA); Metabolic disorders like Homocystinuria (HCU) and paediatric cancers like Low Grade Glioma (pLGG).
Earlier in 2024, Sidra Medicine announced a groundbreaking protocol for treating congenital hyperinsulinism in a young Kuwaiti patient, marking another milestone in its mission to pioneer innovative treatments for rare diseases.