Sidra Medicine pioneers paediatric care in Qatar

QATAR— Sidra Medicine, a member of the Qatar Foundation, has conducted Qatar’s first industry-sponsored pharmaceutical trial for pediatric congenital hyperinsulinism.

This pioneering trial involved a three-month-old Qatari girl diagnosed with the condition who had not responded to previous therapies.

The child’s parents enrolled her in the SunRIZE Phase 3 clinical trial as a last resort to find an effective treatment.

This achievement comes after the launch of Sidra Medicine’s Clinical Trials Program, which focuses on bringing hope to children with rare and complex diseases.

The program prioritizes clinical trials for conditions affecting children in the Arab region, particularly those for which effective treatments are lacking.

Sidra Medicine is among 22 leading centers worldwide chosen to participate in the SunRIZE Phase 3 trial.

This initiative is led by Rezolute, a biopharmaceutical company specializing in rare diseases.

The trial highlights Sidra Medicine’s growing reputation in pediatric endocrinology and its dedication to advancing innovative, patient-focused care.

Congenital hyperinsulinism, the focus of this trial, is a rare and life-threatening condition in children.

It occurs due to excessive insulin production, leading to dangerously low blood sugar levels.

Current treatments are often ineffective, and many patients require invasive pancreatic surgery, which can result in lifelong complications such as diabetes and pancreatic insufficiency. 

According to Prof. Khalid Hussain, Division Chief of Endocrinology at Sidra Medicine, this trial represents a pivotal step in transforming care for children with congenital hyperinsulinism.

He explained that phase 3 trials are crucial for comparing new treatments with existing options, using larger and more diverse patient populations to gather evidence on safety and effectiveness.

He further noted that the collaboration with Rezolute and international experts aims to develop a safer, non-invasive treatment option, reducing the need for surgical intervention and improving outcomes for affected children.

In addition to its work on congenital hyperinsulinism, Sidra Medicine is actively conducting research and clinical trials for other rare and complex conditions.

These include endocrine disorders such as Type 1 diabetes, neuromuscular diseases like Spinal Muscular Atrophy (SMA), metabolic disorders such as Homocystinuria (HCU), and pediatric cancers like Low-Grade Glioma (pLGG).

Earlier in 2024, the hospital also introduced a pioneering protocol for treating congenital hyperinsulinism, further highlighting its commitment to pioneering innovative care for rare diseases.

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