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.
External Link: For Parents and Children
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.
- Generation Victoria Cohort 2020s: A Statewide Longitudinal Cohort Study of Victorian Children and Their Parents
on September 17, 2026 at 4:00 amConditions: Mental Health; Child Wellbeing; Infant Health; Child Health; Reproductive Health; Healthy Aging; Health Equity; Quality of Life; Social Determinants of Health; Environmental Exposure; Genetics; Cognition; Learning; Child Development; Congenital Abnormalities; Disability; Injuries; Communicable Diseases; Noncommunicable Diseases; Physical Fitness; Diet; Obesity; Allergy and Immunology; Inflammation; Intergenerational Relations Sponsors: Murdoch Childrens Research Institute; The Paul Ramsay Foundation; Royal Children's Hospital; Victoria State Government; Royal Children's Hospital Foundation; University of Melbourne; National Health and Medical Research Council, Australia; Medical Research Future Fund; Angliss Health Service; Bairnsdale Regional Health Service; Grampians Health - Ballarat Base Hospital; Bass Coast Regional Health; The Bays Private Hospital; Benalla & District Memorial Hospital; Bendigo Hospital; Box Hill Hospital; Cabrini Private Hospital; Casey Hospital; Castlemaine Hospital; Central Gippsland Health Service; Colac Area Health; Dandenong Hospital; Bacchus Marsh - Western Health (formerly Djerriwarrh Health Service); East Grampians Health Service; Echuca Regional Health; Epworth Freemason's Private Hospital; Frances Perry Private Hospital; Peninsula Health; Epworth Geelong; Geelong University Hospital; Goulburn Valley Health; Hamilton Base Hospital; Jessie McPherson Private Hospital; Joan Kirner Women's and Children's Hospital; The Kilmore & District Hospital; Latrobe Regional Hospital; Leongatha Memorial Hospital; Mansfield District Hospital; Maryborough District Health Service; Mercy Hospital for Women; Mercy Werribee Public Hospital; Mildura Base Hospital; Mitcham Private Hospital; Monash Medical Centre; Northeast Health Wangaratta; The Northern Hospital; Northpark Private Hospital; Peninsula Private Hospital; Royal Women's Hospital; Sandringham & District Memorial Hospital; South Gippsland Hospital; South West Healthcare Camperdown; South West Healthcare Warnambool; St John of God Hospital Ballarat; St John of God Hospital Bendigo; St John of God Hospital Berwick; St John of God Hospital Geelong; St Vincents Private Hospital; Swan Hill District Hospital; Waverley Private Hospital; West Gippsland Healthcare Group; Grampians Health - Horsham (formerly Wimmera Health Care Group); Albury Wodonga Health; Yarrawonga District Health Service; Portland District Health Recruiting
- Online Emotion Regulation and Parenting Program for Parents of Children Aged 2-6 Yearson September 16, 2026 at 4:00 am
Conditions: Parent Emotion Regulation; Parenting; Child Socioemotional and Behavioral Outcomes Interventions: Behavioral: Online Parent Emotion Regulation and Parenting Program Sponsors: The University of Hong Kong Recruiting
- Assessing the Relationships Between Parent-child Eating in the Absence of Hunger, Appetitive Traits, and Food Parenting.on September 14, 2026 at 4:00 am
Conditions: Eating in Absence of Hunger; Food Parenting; Appetitive Traits Interventions: Behavioral: Eating in Absence of Hunger (EAH) paradigm Sponsors: State University of New York at Buffalo Recruiting
- Parenting STAIR: Adapting a Trauma-Focused Parenting Intervention for Military-Connected Mothers and Their Childrenon September 11, 2026 at 4:00 am
Conditions: PTSD; Depression; Parent-Child Relations Interventions: Behavioral: Parenting-STAIR Modular (PSTAIR-M) Sponsors: New York University; Cohen Veterans Network; University of California, Davis Recruiting
- Pai.ACT - An Artificial Intelligence Driven Chatbot Assisted ACT (Full Scale RCT)on September 10, 2026 at 4:00 am
Conditions: Neurodevelopmental Disorders; Autism Spectrum Disorder (ASD); Attention Deficit Disorder With Hyperactivity (ADHD) Interventions: Behavioral: Pai.ACT; Other: Positive parenting advice Sponsors: Chinese University of Hong Kong; Social Welfare Department, Hong Kong; Hong Kong Christian Service Recruiting
- Efficacy of a Smart Holistic Health Care Platform Intervention on Cancer Children and Parents' Bio-Psycho-Social and Spiritual Healthon September 4, 2026 at 4:00 am
Conditions: Leukemia (Both ALL and AML); Brain Tumors Interventions: Behavioral: Parent Smart Holistic Health Care Platform (P-SHHCP); Behavioral: Child Smart Holistic Health Care Platform (C-SHHCP) Sponsors: National Defense Medical University, Taiwan Recruiting
- Evaluating the Efficacy of Central Executive Training (CET) For Young Children With ADHDon August 25, 2026 at 4:00 am
Conditions: ADHD Interventions: Behavioral: Central executive training; Behavioral: Behavioral parent training Sponsors: Florida State University; National Institute of Mental Health (NIMH) Recruiting
- Perceptions of High and Low Intensity Speech Intervention in Children With a Cleft Palateon August 24, 2026 at 4:00 am
Conditions: Cleft Lip and Palate; Cleft Palate Children; Speech Therapy; Speech Disorders in Children; Intervention Intensity Interventions: Behavioral: High-intensity speech therapy for children with cleft palate; Behavioral: Low-intensity speech therapy for children with cleft palate Sponsors: University Ghent; Research Foundation Flanders Recruiting
- Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trialon August 24, 2026 at 4:00 am
Conditions: Neurofibromatosis 1; Noonan Syndrome; Legius Syndrome; Cardiofaciocutaneous Syndrome; Costello Syndrome Interventions: Behavioral: Waitlist; Behavioral: ACT Intervention Sponsors: National Cancer Institute (NCI) Recruiting
- Longitudinal Ulnar Deficit (ULNEER)on August 24, 2026 at 4:00 am
Conditions: Ulnar Longitudinal Deficiency; Ulnar Hand Deformity Sponsors: Hopitaux de Saint-Maurice Recruiting
- Investigating the Efficacy of a Paediatric Fertility Preservation Decision Aid in Parents and Adolescents and Young Adults (CAYA) Cancer Survivorson August 19, 2026 at 4:00 am
Conditions: Unmet Fertility Information Needs Interventions: Other: Decision-aid Sponsors: Murdoch Childrens Research Institute; University of Melbourne Recruiting
- Online Feeding Education for Fathers of Children With Feeding Problemson August 18, 2026 at 4:00 am
Conditions: Feeding Problems; Parent Training; Parent Child Relationship; Feeding Behaviors; Fathers Interventions: Other: Online Feeding Awareness Training for Fathers Sponsors: Ankara Medipol University 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.
External Link: Why PTN Research Matters and Patient Safeguards in PTN Research
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.