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.
- Feasibility Study of UP-A-AST Parent Group for Adolescents With Autism and Co-occurring Anxiety/Depression
on June 10, 2026 at 4:00 amConditions: Autistic Spectrum Disorder; Anxiety Disorders; Depressive Disorder Interventions: Other: Unified Protocol-Adolescent Autism Parent Group (UP-A-AST) Sponsors: Sahlgrenska University Hospital Recruiting
- Comparing Single Versus Repeat NMT on the Diversity of the Neonatal Nasal Microbiomeon June 8, 2026 at 4:00 am
Conditions: Staphylococcus Aureus; Microbial Colonization; Neonatal Infection Interventions: Biological: Nasal Microbiota Transplant (NMT); Biological: Placebo Sponsors: Johns Hopkins University Recruiting
- Effect of Yoga and Values Clarification on Stress, Comfort, and Emotional Regulation in Parents of Children With Special Needson June 5, 2026 at 4:00 am
Conditions: Stress; Emotional Regulation; Comfort Interventions: Behavioral: Yoga; Behavioral: Values Clarification Sponsors: Ankara Yildirim Beyazıt University Recruiting
- Testing eSCCIP: An eHealth Psychosocial Intervention for English and Spanish Speaking Parents of Children With Canceron June 5, 2026 at 4:00 am
Conditions: Pediatric Cancer Interventions: Behavioral: eSCCIP/eSCCIP-SP; Behavioral: Coping Space Sponsors: Nemours Children's Clinic; M.D. Anderson Cancer Center; Children's Hospital Los Angeles; University of Virginia Recruiting
- FOCUS Bipolar: Families Opening Conversations for Understanding Signson June 3, 2026 at 4:00 am
Conditions: Bipolar Disorder (BD); Bipolar Disorder I or II; Bipolar Disorder Family Members; Screening Tool Interventions: Other: Co-design Workshop (Observational Procedure); Other: Alpha Testing (Observational Procedure); Other: Beta Testing (Observational Procedure); Other: Youth Screening Pathway & Brief Follow-Up Interview (Observational Procedure) Sponsors: University of Texas Southwestern Medical Center; The Texas Child Mental Health Care Consortium (TCMHCC) Recruiting
- Randomized Controlled Trial of a Family-focused Intervention for Caregivers and Young Adolescents (Phase 3 of FLOURISH)on June 2, 2026 at 4:00 am
Conditions: Adolescent - Emotional Problem; Parent Child Relationship; Family Functioning; Well-Being, Psychological Interventions: Behavioral: Optimized Parenting for Lifelong Health (PLH) for Parents and Teens Sponsors: University of Klagenfurt; Health for Youth Association, Moldova; Institute for Marriage, Family and Systemic Practice - ALTERNATIVA; Cardiff University; Medical University of Vienna; Bielefeld University; University Jaume I Castellon; Association of Systemic Therapists Education Centre Recruiting
- Language Development in Children With Cochlear Implants and Speech-language Therapy Interventionson May 28, 2026 at 4:00 am
Conditions: Severe to Profound Prelingual Hearing Loss Interventions: Other: Speech-language therapy (observed standard care) Sponsors: Assistance Publique - Hôpitaux de Paris; URC-CIC Paris Descartes Necker Cochin Recruiting
- Word Learning in Bilingual Typical and Late Talking Children: The Role of Meaning and Inputon May 20, 2026 at 4:00 am
Conditions: Bilingualism; Vocabulary Acquisition; Late Talkers; Network Science Interventions: Behavioral: Large Semantic Category Condition; Behavioral: Small Semantic Category Condition Sponsors: Boston University Charles River Campus; National Institute on Deafness and Other Communication Disorders (NIDCD) Recruiting
- Breaking Silence Through Story: A Narrative Medicine Intervention for Parents of Children With Urogenital Conditionson May 18, 2026 at 4:00 am
Conditions: DSD; Hypospadias Interventions: Behavioral: Narrative Medicine Journaling Intervention Sponsors: Sarah Schlegel; Harvard Catalyst Pilot Grant Recruiting
- K-ORCA: Testing a Decision Support Tool and Group Process for Selecting Interventionson May 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
- Child-Parent Familial Hypercholesterolemia Screeningon May 15, 2026 at 4:00 am
Conditions: Familial Hypercholesterolemia Interventions: Other: no interventions Sponsors: Children's Hospital of Fudan University Recruiting
- Reducing Parental Substance Use and Enhancing Family Resilience Among Rural Families Through Ohio STARTon May 14, 2026 at 4:00 am
Conditions: Substance Use Disorder (SUD); Rural Health; Family Resilience; Child Maltreatment; Child Well-beiing; Substance Use Recovery Interventions: Behavioral: Ohio START (Ohio Sobriety, Treatment, and Reducing Trauma) Sponsors: Ohio State University; Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); University of Michigan 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.