Similarly to older adults, children are a unique population with different physiological and developmental needs than adults. Children are effected by diseases and medicines in unique ways, which in turn means that the effectiveness of specific drugs should be measured independently.
It is important to perform clinical trials in children because scientists need to determine how different treatments, medications, and devices work on these populations.
Clinical research studies in children are what have helped develop child-appropriate medications and figure out which doses are the safest for each age group.
Sometimes when a child is suffering from a medical condition the doctor might ask the parents to consider enrolling him or her in a clinical trial to become part of a group of patients that are testing of a new drug or treatment.
What is a Clinical Trial?
Clinical trials are a type of research study designed to test the effectiveness of a new drug or treatment. Clinical trials are meticulously designed, planned, and carried out by scientists and other experts. These trials are carefully monitored by internal and external agencies in order to maintain the highest standards possible and to ensure the safety and comfort of its participants. Before a clinical trial even begins, it needs to be approved by the FDA and by an Internal Review Board that makes sure no part of the study poses a more than minimal risk for its participants and that the proposed research is clinically necessary and relevant.
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All research studies are completely voluntary, meaning that the choice of participating in a clinical trial is completely up to the individual or legal guardian of the individual. This also means that any person has the right and freedom to withdraw their consent at any point in time. Withdrawing consent during the research study will not yield any physical or monetary consequences, nor will it affect their ability to receive adequate medical care.
If a parent agrees to enroll their child in a research study they must sign an informed consent document, which will outline all relevant information about the study as well as all the potential risks and benefits this trial might pose to the participant.
It is important to note that by the time a drug has reached advanced stages of the clinical research, it has generally been extensively tested on animals and sometimes even on healthy individuals to assess its potential toxicity, bioavailability, and general safety.
- Preventing Vulnerable Child Syndrome in the NICU With Cognitive Behavioral Therapy (PreVNT Trial)
on July 9, 2026 at 4:00 amConditions: Premature Infant; Mental Health Issue (E.G., Depression, Psychosis, Personality Disorder, Substance Abuse); Development, Child; Parent-Child Relations Interventions: Behavioral: Cognitive Behavioral Therapy Sponsors: University of Texas Southwestern Medical Center; Stanford University Recruiting
- Live Parent-Child Video Interaction and Cartoon Watching for Emergence Agitation in Childrenon July 8, 2026 at 4:00 am
Conditions: Emergence Delirium; Preoperative Anxiety Interventions: Behavioral: Live Parent-Child Video Interaction; Behavioral: Cartoon Watching Sponsors: Izmir City Hospital Recruiting
- LOVING (Learning About Our Behavior is Valuable for Increasing Nurturing Relationships and Healthy Growth) RCTon July 7, 2026 at 4:00 am
Conditions: Child Development; Child Behavioral Problems; Parent Child Relationship Interventions: Behavioral: VIPP-SD-SingH, "Video-Feedback Intervention to Promote Positive Parenting and Sensitive Discipline Adapted for the Singapore Context with a Health Component"; Behavioral: CARE-SD, "Caregiver Awareness of Relationships through E-learning for Sensitivity and Discipline"; Behavioral: NeuroEducation for Parents Sponsors: National University of Singapore; Nanyang Technological University; Institute for Human Development and Potential (IHDP), Singapore; National Institute of Education, Singapore; KK Women's and Children's Hospital Recruiting
- Digitally Delivered Child Obesity Prevention for Parents in Home Visiting Programson July 6, 2026 at 4:00 am
Conditions: Childhood Obesity Prevention Interventions: Behavioral: digital intervention + home visiting curriculum; Behavioral: Home visiting curriculum Sponsors: University of Florida; National Heart, Lung, and Blood Institute (NHLBI) Recruiting
- Neural Prediction to Enhance Languageon July 2, 2026 at 4:00 am
Conditions: Bilateral Sensorineural Hearing Loss; Speech and Language Development Delay Due to Hearing Loss; Social Communication Interventions: Behavioral: Parent-Implemented Communication Treatment Sponsors: Ann & Robert H Lurie Children's Hospital of Chicago; Chinese University of Hong Kong; Northwestern University; University of North Carolina, Chapel Hill; University of Southern California; University of Miami; The University of Texas at Dallas Recruiting
- Evaluation of the Invest in Play Program - a New Parent Intervention for Children With Problem Behavioron June 29, 2026 at 4:00 am
Conditions: Problem Behavior; Attention Deficit and Disruptive Behavior Disorders Interventions: Behavioral: Intervention Sponsors: University of Tromso; NORCE Norwegian Research Centre AS; Norwegian University of Science and Technology Recruiting
- Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.on June 24, 2026 at 4:00 am
Conditions: Phenylketonuria Interventions: Behavioral: socio-psychological questionnaire; Behavioral: revised event impact scale (IER-S); Other: Non directive interview; Other: Stern interview; Other: semi-directive interview Sponsors: Assistance Publique - Hôpitaux de Paris; URC-CIC Paris Descartes Necker Cochin Recruiting
- ACT Together: Implementing a Web-Based Program With Brief Coaching for Parents of Children With Disabilities in Pediatric Outpatient Clinicson June 23, 2026 at 4:00 am
Conditions: Parents; Children With Disabilities; Stress; Depression Interventions: Behavioral: ACT Together Sponsors: University of South Florida; American Occupational Therapy Foundation Recruiting
- Socio-Cultural Level, Health Literacy, and Parental CT Demand in Paediatric Minor Head Traumaon June 22, 2026 at 4:00 am
Conditions: Craniocerebral Trauma; Brain Injuries, Traumatic; Head Injuries, Closed Sponsors: Bursa Yuksek Ihtisas Training and Research Hospital Recruiting
- Parent-Mediated Telehealth Intervention for Insomnia in Young Autistic Childrenon June 18, 2026 at 4:00 am
Conditions: Insomnia Interventions: Behavioral: Sleep Parent Training (SPT); Behavioral: Sleep Parent Education (SPE) Sponsors: Emory University; Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); The Cleveland Clinic; Ohio State University Recruiting
- K-ORCA: Testing a Decision Support Tool and Group Process for Selecting Interventionson June 18, 2026 at 4:00 am
Conditions: Child Abuse; Decision Making; Child Welfare; Decision Making, Shared; Decision Support Technique; Social Facilitation; Implementation Science; Policy; Organizations; Consensus; Mental Health; Family Interventions: Behavioral: Optimizing Responses with Collaborative Assessments-Automated (ORCA-A); Behavioral: Optimizing Responses with Collaborative Assessments-Live (ORCA-L) Sponsors: Chestnut Health Systems; National Institute of Mental Health (NIMH) Recruiting
- Sensory Optimization of the Hospital Environmenton June 17, 2026 at 4:00 am
Conditions: Preterm; Parent-Child Relations; Parents; Development, Infant; Sensory Integration Dysfunction Interventions: Behavioral: SENSE multisensory program; Behavioral: Monitored standard of care Sponsors: University of Southern California; St. Louis University; Washington University School of Medicine; University of North Carolina; Harvard University; Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; St. Louis Children's Hospital Recruiting
What Happens During a Clinical Trial?
All clinical trials are different and they depend on what the researchers are investigating. Most clinical trials are carried out in a hospital setting and all of them are performed under strict supervision.
One of the most common designs for a research study is called a randomized controlled trial (RCT). During an RCT the child will be randomly assigned into a treatment group or a placebo group, and participants will not know which group they were assigned to until the end of the study. This type of study is only carried out when participants are healthy enough to withstand a short amount of time without treatment. Regardless of the group they were assigned to, all participants will receive the necessary medical treatment after the study ends.
External Link: Is a Clinical Trial Right for Your Child?
What Are the Benefits of Enrolling My Child in a Clinical Trial?
Children clinical trials are very important to determine the safety and effectiveness of different medications and treatments for kids. Without clinical trials, doctors can only guess what is the appropriate dose for a child, so research studies are extremely important for taking the guesswork out of the clinical practice.
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Scientist and pharmaceutical companies only carry out a research study when they have sufficient scientific knowledge to believe that a medication might actually be beneficial, so although it is never a guarantee, it is possible that the child has the opportunity to receive a potentially beneficial treatment even before it’s widely available.
What Are the Risks of Enrolling My Child in a Clinical Trial?
It is important to remember that a clinical trial is, after all, a trial; so parents should be prepared for the possibility of the treatment not working at all. Another potential risk is side effects; when a medication is being tested it is possible for some side effects to come up that were not present in previous phases of the study. To ensure that participants are safe, doctors and investigators monitor the effects of the treatment very closely by performing constant tests and assessments.