Rare diseases
Investigational molecules
Locations
A study testing fitusiran to prevent bleeding in boys aged 1 to 11 years with hemophilia A or B with or without inhibitors
BelgiumA study of mirdametinib for infants and toddlers under 24 months of age with neurofibromatosis type 1 associated plexiform neurofibromas
GermanyA study testing intranasal esketamine compared to midazolam for rapidly reducing symptoms of major depressive disorder in adolescents with suicidal thoughts or behavior
Hungary
FranceA study on azithromycin for preventing airway infections in children aged 0-72 months with structural lung damage or congenital lung and airway malformations
DenmarkLong-Term Study of Vosoritide Safety and Effectiveness in Children with Hypochondroplasia Until Adult Height is Reached
FranceA Study of Gene Replacement Therapy for Children with CTNNB1 Syndrome Using Adeno-Associated Viral Vector Serotype 9 Containing the Human CTNNB1 Gene
SloveniaStudy of axatilimab compared to standard therapy in children with chronic graft-versus-host disease who have previously received at least 2 treatments
BelgiumStudy of Apitegromab Treatment in Children Under 2 Years Old with Spinal Muscular Atrophy (SMA)
BelgiumStudy of Vosoritide Safety and Effectiveness in Infants and Young Children (0 to 36 months) with Hypochondroplasia
FranceStudy of inhaled corticosteroids (beclometasone, fluticasone, budesonide) and salbutamol in children aged 1-6 years with episodic wheezing
The NetherlandsLong-term safety and effectiveness study of ritlecitinib in children with severe alopecia areata who completed previous trials B7981027 or B7981031
CzechiaClinical research plays a vital role in developing safe and effective treatments for younger patients. Pediatric clinical trials are trials designed specifically for infants, children, and adolescents. They help researchers understand how new therapies, vaccines, or medical procedures work in younger populations and ensure that treatments are safe and appropriate for their age group.
Children are not simply smaller versions of adults. Their bodies grow and develop, which means medications may work differently than they do in adults. For this reason, therapies must be carefully studied in pediatric populations.
Another important reason for pediatric clinical trials is that some diseases occur primarily or exclusively in children. Certain genetic conditions, developmental disorders, and rare pediatric diseases affect only young patients. Without dedicated research involving children and adolescents, it would be difficult to develop treatments specifically designed for these conditions.
Pediatric research also helps doctors determine:
Participants in pediatric clinical trials may include infants, children, and teenagers depending on the trial design. Each trial has specific eligibility criteria based on age, health condition, and medical history.
Because participants are minors, the process always involves:
This ensures that families fully understand the purpose of the research, the procedures involved, and any potential risks or benefits.
Safety is the highest priority in pediatric clinical trials. These trials follow strict ethical and regulatory standards in Europe and internationally. Research protocols are reviewed by ethics committees and regulatory authorities to ensure that the risks are minimized and justified by the potential benefits of the research.
Children participating in clinical trials are monitored closely by qualified healthcare professionals throughout the entire process. Families receive detailed information about trial procedures, medical monitoring, and the right to withdraw from the trial at any time.
Our search platform allows parents and guardians to explore pediatric clinical trials available in different regions. Using the search tool, families can find trials based on factors such as age group, medical condition, and location.
Clinical trials for children and adolescents are essential for advancing pediatric medicine. By participating in research, families contribute to the development of safer and more effective treatments for young patients today and in the future.
Tell us about your condition – we search every trial in Europe and connect you with the right site.
