Clinical Trials Program

Clinical trials are the final step of medical research, where new treatments undergo careful evaluation for safety and effectiveness before being made widely available.

The trials could offer your child the chance to receive beneficial treatment whether it is a new drug therapy, device or procedure—before it becomes widely available. Your child’s participation could result in scientists discovering a more effective treatment for diseases that affect children all over the globe—and it could treat their disease more effectively than standard protocols.

Clinical trials serve as the backbone of medical advancements, offering a structured approach to evaluate the efficacy of new treatments. While not all clinical trials are successful in addressing or curing a disease, by enrolling your child in a clinical trial, will allow our team to find information that could help yours and other children.

Everyone who participates in clinical trials is a volunteer.

The outcome of your child’s clinical trial has the potential to positively impact the care, treatment and wellbeing of not only your child but that of many other children, not only in the same family but for families everywhere. It will also be a chance to help future patients. As a volunteer, your child is playing an important role in helping us find better ways to prevent, detect and treat diseases.

We work closely with the parents to empower them throughout the clinical trial process. Involving families in decision-making promotes shared responsibility, enhancing treatment adherence and outcomes. We become partners to ensure the best outcomes for your child.

By enrolling your children in research studies and clinical trials, families become partners in helping us develop the treatments of the future. It could benefit your family because it offers an opportunity to try a new medicine or treatment protocol or one that’s used in a new way.

We also recommend talking to your child’s doctor, your child, your family, and others in your support system. Having as much information as possible will help you make the right decision for your child.

Sidra Medicine’s clinical and Child Life teams have resources in place to help you explain to your child about the clinical trial process and how their care journey would look like.

For young children, they may even use pictures, props or videos to explain the process.

Each child’s legal guardian must give permission before the child is enrolled in a clinical trial. His or Her doctor and the clinical trials team may meet with the child to make sure they understand what the trial is about.

If the child is mature and shows adequate understanding, the team will ask for their written consent.

At Sidra Medicine, patient safety and well-being are the foremost concern.
Every clinical trial undergoes intense scrutiny, including review by an independent Institutional Ethical Board (IRB) and Qatar’s Ministry of Public Health (MOPH)—to ensure the protection of every patient enrolled, particularly vulnerable children.
The privacy and confidentiality of each family is strictly maintained throughout the journey. We have an informed consent process which adopts clear communication and ensures understanding of risks and benefits for both the patients and their guardians.
Your child’s involvement in a clinical trial is protected. Their name and family information will never be made public without your consent. This multi-layered approach, with multiple checkpoints throughout the process, guarantees adherence to the highest international ethical and scientific standards in clinical research.

Sidra Medicine is currently focusing on the following disorders and medical conditions:

• Endocrine disorders such as Congenital Hyperinsulinism (CHI), Diabetes Type 1 (TD1)
• Neuromuscular conditions like Spinal Muscular Atrophy (SMA)
• Metabolic disorders like Homocystinuria (HCU)
• Pediatric cancers like as Low Grade Glioma (pLGG)

Sidra Medicine will be conducting clinical trials that have either been approved for adults but not yet studied for children or have not yet been government approved. The trials will focus on either of the following:

• Medicines
• Medical devices
• Medical procedures

The capabilities of our clinical trials allow us to undertake complex research, including critical Phase 1 trials right up until Phase 4 studies. This follows years of excellence at diagnostic research where our teams made many fundamental discoveries in the cause and origin of diseases in our patients.