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Pediatric Clinical Trials

Pediatric clinical trials are important to understand how children respond to different medicines and vaccines.  

Why are Pediatric Clinical Trials Important?

Pediatric clinical trials are those involving children and young people, and are very important for developing safe and effective medications and vaccines appropriate for this age group.1 

Children are not simply 'small adults’—they have bodies that are still growing and changing, which means medications can affect them differently from adults.1,2 Relying on clinical trial data from adults may therefore result in ineffective treatments and unexpected side effects in children.1,2 

In addition to efficacy and safety, pediatric trials help in determining the most appropriate dosage and administration methods for children.2  The taste, smell, and texture of medications are also particularly important to understand, as these factors can influence a child's willingness to take medications.3

Finally, some diseases are unique to children in their frequency or severity, making it essential to study their treatment or prevention specifically in pediatric clinical trials.1,4 

Commonly Used Terms in Pediatric Clinical Studies

There may be different terms used to refer to the individuals involved in pediatric clinical trials. These include: 

  • Pediatric/child/children: These terms refer to individuals under the age of 18, who are not legally classified as adults, although the exact age of adulthood may vary by country. 
  • Young people: Some individuals under 18, such as teenagers, may not identify as children, so this term may be more appropriate to describe these people. 

Other terms may also be used to refer to pediatric populations, such as infant, teenager, neonate, baby, toddler, kid, and adolescent. 

Challenges in Pediatric Clinical Trials

Children are a unique population, so there are also unique challenges that may accompany pediatric clinical trials.1,2 

Ethical Considerations for Childhood Enrollment in Clinical Trials

As stated, it is ethically important to study medications specifically for children.1 However, they are considered a vulnerable population, which raises concerns about exposing them to the potential risks associated with using untested medications or with clinical research in general.1–3 As such, the benefits of a trial need to be weighed against the risks to justify the research effort.2 

For this reason, the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have established ethical guidelines for clinical trials involving children, which incorporate additional safeguards to ensure their protection.5,6  

Additionally, the ethical principles of respect for persons, beneficence, and justice guide all clinical trials, including those that involve children.7 

Informed Consent

Another challenge that may be encountered in pediatric clinical trials is informed consent. Informed consent is required for participation in all clinical trials, but the process may becomes more complex in pediatric trials.  

For example, in the US, children under the age of 18 cannot legally provide consent, as they may not fully understand the risks and benefits of the trial.1,2 Instead, consent must be obtained from a parent or legal guardian. However, some parents or legal guardians may be uncomfortable with the responsibility of making this decision on their child's behalf.1 

Although children in the US cannot provide consent, those who are seven years or older typically need to provide ‘assent’. Assent involves the child’s agreement to participate in the clinical trial, ensuring they are given an age-appropriate explanation of the study. This procedure helps in addressing the ethical factor of respecting a child's autonomy.1,2

Limited Sample Collection

Many clinical trials require the collection of blood, however, an additional challenge in pediatric trials is limited blood sample collection. Ethically, this is done to minimize any pain or discomfort children may experience from needles, as well as to prevent a certain type of anemia (lack of healthy red blood cells) triggered by excessive blood collection.2,3,8 

This can be particularly restrictive in clinical trials involving smaller children, where the maximum allowable blood volume for collection may be less than 5ml.3 Pediatric clinical trials must therefore be designed to consider this. 

Sanofi’s Commitment to Pediatric Clinical Research

Here at Sanofi, we adhere to the principles outlined in the ICH guidelines for good clinical practice (GCP) which are upheld in all clinical trials. We also follow the advice of regulatory bodies such as the Institutional Review Boards (IRBs), who are responsible for regulating any potential risk to the child in all clinical trials.9 

Learn more about Sanofi's bioethics in clinical trials. 

Explore Sanofi’s Pediatric Clinical Trials and Studies

Sanofi has ongoing pediatric clinical trials in a number of different diseases: 

Study on the Safety and Efficacy of an Investigational Medication for Type 1 Diabetes

FrexalimAB in Preservation of Endogenous insULIN Secretion Compared to Placebo in adUlts and Adolescents on Top of inSulin Therapy (FABULINUS)

View trial details

Investigating the Effect of an Investigational Medication on Type 1 Diabetes

Treatment of Type 1 Diabetes With Anti-OX40L Bispecific With Anti-TNF Activity In a Single Nanobody® Molecule

View trial details

Study on Glycemic Control in Type 1 Diabetes

A Study to Investigate Efficacy and Safety of Teplizumab Compared With Placebo in Participants 1 to 25 Years of Age With Stage 3 Type 1 Diabetes

View trial details

Investigational Vaccine Study for Infant Immunogenicity (the ability to provoke an immune response) and Safety

Study of a 21-valent Pneumococcal Conjugate Vaccine in Healthy Infants From Approximately 2 Months of Age

View trial details

Investigating the Safety and Immunogenicity (Ability to Produce a Strong Immune Response) of an Investigational Pneumococcal Vaccine in Infants

Study of a 4-Dose Regimen of a 21-valent Pneumococcal Conjugate Vaccine in Healthy Infants From Approximately 2 Months of Age

View trial details

Research Study on an Investigational Medication for Systemic Juvenile Idiopathic Arthritis in Children and Adolescents

A Repeated Dose-finding Study of Sarilumab in Children and Adolescents With Systemic Juvenile Idiopathic Arthritis (SKYPS)

View trial details

Study on Pneumococcal (related to the bacteria Streptococcus pneumoniae) Vaccines for Infants, Toddlers, and Children

Study of the Safety and Immunogenicity of Catch-up Vaccination With a 21-valent Pneumococcal Conjugate Vaccine (PCV21) in Healthy Infants, Toddlers, Children, and Adolescents

View trial details
Pediatric Trials

Abbreviations

FDA, Food and Drug Administration; GCP, Good Clinical Practice; ICH, The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use; IRB, Institutional Review Boards; EMA, European Medicines Agency. 

References

  1. Patel T. The Ethics of Pediatric Clinical Trials. Intersect: The Stanford Journal of Science, Technology, and Society. 2024;17(2). Accessed May 27, 2025.

https://ojs.stanford.edu/ojs/index.php/intersect/article/view/2732

  1. Raschke E. Conducting Pediatric Studies: Addressing Ethical and Clinical Challenges. Precisionformedicine.com. Published October 23, 2023. Accessed May 27, 2025. https://www.precisionformedicine.com/blog/pediatric-clinical-trials-challenges/#_ftn1
  2. Shakhnovich V, Hornik CP, Kearns GL, Weigel J, Abdel‐Rahman SM. How to Conduct Clinical Trials in Children: A Tutorial. Clinical and Translational Science. 2019;12(3):218-230. doi:https://doi.org/10.1111/cts.12615
  3. Subramanian D, Cruz CV, Facundo Garcia-Bournissen. Systematic Review of Early Phase Pediatric Clinical Pharmacology Trials. The journal of pediatric pharmacology and therapeutics. 2022;27(7):609-617. doi:https://doi.org/10.5863/1551-6776-27.7.609
  4. FDA. Pediatric Ethics. Available from:  https://www.fda.gov/science-research/pediatrics/pediatric-ethics [Accessed: May 2025]
  5. European Medicines Agency. Paediatric medicines: Overview. Available from: https://www.ema.europa.eu/en/human-regulatory-overview/paediatric-medicines-overview [Accessed: May 2025]
  6. HHS.gov. The Belmont Report. Available from: https://www.hhs.gov/ohrp/regulations-and-policy/belmont-report/read-the-belmont-report/index.html#xbasic [Accessed: May 2025]
  7. Baysan M, Arbous MS, van der Bom JG. Iatrogenic anemia: an underestimated and solvable problem in the intensive care unit. Annals of Blood. 2020;5:19-19. doi:https://doi.org/10.21037/aob-20-29
  8. European Medicines Agency (EMA). ICH E6 good clinical practice - Scientific guideline. Available from: https://www.ema.europa.eu/en/ich-e6-good-clinical-practice-scientific-guideline  [Accessed: May 2025]
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