Top Pediatric Clinical Trial Sites Globally Directory and Specializations
Pediatric clinical trials are never one-size-fits-all. Unlike adult studies, they demand distinct ethical standards, dosage formulations, and safety frameworks. Children’s metabolic rates, cognitive development stages, and vulnerability to side effects require tailored protocols—not just scaled-down versions. Fewer eligible patients, higher regulatory oversight, and the need for parental consent all make site selection a strategic decision. Without dedicated pediatric capabilities, even the most promising drug studies risk delay or failure.
Globally, only a select group of research centers specialize in child-centric clinical operations. These sites are trusted for their multidisciplinary pediatric teams, IRB navigation expertise, and long-term sponsor collaborations. From breakthrough gene therapies in London to vaccine innovations in Seoul, these institutions are accelerating child health through excellence in trial execution. This guide breaks down the world’s top pediatric trial sites—by region and specialty—so you can align your study with the most capable and compliant clinical partners.
Why Pediatric Trials Require Specialized Sites
Ethical, pharmacokinetic, and formulation differences
Pediatric patients are a protected population—both legally and biologically. Their organ development, metabolism, and immune response vary dramatically from adults, altering drug absorption and clearance rates. This is why pharmacokinetic modeling in pediatrics must account for age, weight, and maturation, not just body surface area.
In formulation, many drugs require child-friendly versions: liquid syrups, dissolvable tablets, or flavor-masked formats. What works in adults can be unsafe or noncompliant for a 4-year-old. Without site-level formulation expertise and compounding capabilities, studies often face dropout or protocol amendments that compromise data integrity.
IRB/ethics approval challenges with minors
Obtaining informed consent in pediatric trials is far more complex. You’re dealing with both parental consent and minor assent, with added layers of jurisdictional laws, especially in multinational trials. Ethics committees typically demand enhanced safety monitoring, non-invasive procedures, and proof that the study offers minimal risk with potential benefit.
Sites experienced in pediatric trials often maintain pre-approved ethics templates, trained counselors, and robust documentation workflows to streamline IRB cycles. This becomes a strategic advantage—reducing timeline bottlenecks while ensuring regulatory compliance.
Importance of family-centered design and communication
Children rarely make healthcare decisions alone. Trials must be designed with a family-first approach—accommodating parents’ schedules, explaining procedures in age-appropriate language, and ensuring emotional comfort throughout.
Top pediatric trial sites embed this philosophy into daily operations. They staff child life specialists, use visual aids or play therapy, and integrate flexible scheduling to reduce family burden. These factors directly improve enrollment and retention—two of the biggest pain points in pediatric research. Sites that overlook this human element often suffer from incomplete data and high dropout rates, regardless of clinical infrastructure.
Leading Pediatric Sites in North America
Children’s Hospital of Philadelphia (CHOP)
Founded in 1855, CHOP is globally recognized for pediatric translational research. It houses more than 1 million square feet of research space, and leads over 1,000 active studies. Its strengths lie in oncology, rare diseases, gene therapy, and vaccine development.
CHOP’s Center for Pediatric Clinical Effectiveness collaborates with sponsors on early-phase through post-marketing trials, particularly in neuroblastoma, CAR-T cell therapies, and metabolic disorders. It has pioneered adaptive trial models and leads several NIH-funded consortia, including the Pediatric Trials Network. Its recruitment teams are known for navigating hard-to-reach pediatric subgroups—especially in genetic disorders and hematology-oncology.
Focus on oncology, rare diseases, vaccines
CHOP’s Division of Oncology runs over 250 trials per year and is a hub for global phase I/II studies in leukemia, lymphoma, and neuro-oncology. It also supports rare disease research through the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, housing in-house vector manufacturing for AAV-based therapies. In vaccine trials, CHOP partners with major pharma for pediatric COVID-19, RSV, and HPV vaccine development.
Cincinnati Children’s Hospital Medical Center
Consistently ranked among the top three U.S. children’s hospitals, Cincinnati Children’s runs more than 1,600 active trials, with over 400 specifically for pediatric populations. Its success is rooted in longstanding NIH partnerships, advanced informatics, and registry-based trial models.
The James M. Anderson Center for Health Systems Excellence supports real-world data analytics and EHR-integrated trial workflows. Their multidisciplinary model allows for parallel feasibility analysis, cohort identification, and virtual follow-up—all critical for high-volume pediatric trials.
Long-standing NIH partnerships, data registries
Cincinnati Children’s hosts one of the largest pediatric biobanks in the U.S., tied to longitudinal registries in asthma, GI disorders, and rare genetic conditions. Their Cystic Fibrosis Therapeutics Development Network site is among the most productive globally. With embedded NIH grant pipelines and early-phase research facilities, they offer bench-to-bedside continuity for novel pediatric compounds.
Their ability to integrate with national data networks—like PEDSnet and PCORnet—gives sponsors access to real-world pediatric data at scale, ideal for comparative effectiveness studies or pre-market validation.
| Institution | Core Specializations | Notable Infrastructure & Capabilities | Sponsor Advantages |
|---|---|---|---|
| Children’s Hospital of Philadelphia (CHOP) | Pediatric oncology, rare diseases, gene therapy, vaccines | 1,000+ active studies, in-house vector manufacturing (AAV), adaptive trial models, NIH consortia leadership | Access to hard-to-reach subgroups, advanced early-phase capabilities, strong regulatory history |
| CHOP Division of Oncology | Leukemia, lymphoma, neuro-oncology | 250+ trials/year, Perelman Center for Cellular & Molecular Therapeutics | Ideal for early-phase oncology and rare disease studies |
| Cincinnati Children’s Hospital Medical Center | Pediatrics across 400+ dedicated trials: asthma, GI, genetics | James M. Anderson Center, NIH-funded biobank, registry-integrated workflows, EHR integration | Real-world data access, scalable recruitment, seamless NIH collaboration |
| Cincinnati: NIH & Registries | Cystic fibrosis, GI disorders, rare pediatric conditions | PCORnet, PEDSnet, embedded data platforms | Enables large-scale, registry-driven, RWE-focused pediatric trials |
Premier Pediatric Sites in Europe
Great Ormond Street Hospital (UK)
One of the most iconic pediatric hospitals in Europe, Great Ormond Street Hospital (GOSH) leads in early-phase pediatric trials, rare disease therapeutics, and advanced biologics. With its dedicated NIHR-funded Biomedical Research Centre, GOSH runs over 350 active clinical trials annually, many targeting ultra-rare and genetic conditions.
GOSH has pioneered compassionate access protocols for pediatric patients and is closely partnered with the UCL Great Ormond Street Institute of Child Health—integrating research and clinical application under one infrastructure. Their approach focuses on co-developing protocols with sponsors, optimizing timelines while centering on patient safety.
Gene therapy and rare disease focus
GOSH is a global leader in pediatric gene therapy, with landmark work on ADA-SCID, Wiskott-Aldrich Syndrome, and spinal muscular atrophy. Its Center for Cellular Therapy enables in-house vector development and GMP manufacturing—cutting out delays in early-phase studies. Their rare disease program integrates whole-genome sequencing and biomarker discovery to inform inclusion criteria and outcome measurement.
University Hospital of Paris (Necker-Enfants Malades)
Founded in 1802, Necker is Europe’s oldest pediatric hospital and part of the Assistance Publique – Hôpitaux de Paris network. It is known for its strength in multicenter trial coordination and pediatric subspecialties, particularly neurology, immunology, and nephrology.
Necker’s integration with INSERM and AP-HP Research fosters academic–industry collaboration, enabling rapid start-up for EU-wide trials. The hospital’s Clinical Investigation Center (CIC) is GCP-certified and supports over 100 pediatric trials per year, with a focus on CNS disorders, epilepsy, and autoimmune conditions.
Multicenter trial coordination and pediatric neurology
Necker excels in cross-border trial logistics, often acting as lead EU site for EMA-authorized pediatric trials. Their neurogenetics team works on advanced protocols for Dravet syndrome, leukodystrophies, and pediatric MS. Their CIC’s coordination team supports regulatory harmonization across EU member states, ensuring trials avoid bottlenecks due to country-specific IRB discrepancies.
Their use of patient registries and telemedicine platforms allows for real-time data collection and decentralized monitoring, especially valuable in post-COVID trial models where remote participation is crucial.
| Institution | Core Specializations | Key Capabilities | Sponsor Advantages |
|---|---|---|---|
| Great Ormond Street Hospital (UK) | Early-phase trials, rare diseases, gene therapy, advanced biologics | 350+ trials/year, NIHR Biomedical Research Centre, in-house GMP vector manufacturing | Accelerated protocol development, pediatric-focused trial design, gene therapy readiness |
| GOSH – Gene Therapy Focus | ADA-SCID, Wiskott-Aldrich, spinal muscular atrophy | Whole-genome sequencing, biomarker-driven inclusion, compassionate access programs | Reduces early-phase delays, enables rare disease targeting |
| University Hospital of Paris (Necker-Enfants Malades) | Neurology, immunology, nephrology, CNS disorders | GCP-certified CIC, INSERM-APHP collaboration, 100+ pediatric trials/year | Fast EU trial start-up, protocol harmonization across countries |
| Necker – Multicenter Coordination | Dravet syndrome, leukodystrophies, pediatric MS | Regulatory streamlining, decentralized monitoring, patient registries | Ideal for EMA trials, post-COVID remote data workflows |
High-Impact Sites in Asia-Pacific
National Center for Child Health and Development (Japan)
Located in Tokyo, the National Center for Child Health and Development (NCCHD) leads Japan’s pediatric research ecosystem. The center is uniquely structured under Japan’s national medical research system, making it a go-to site for pediatric pharmacology, vaccines, and developmental medicine.
NCCHD integrates its Pediatric Clinical Research Network (J-PRN), which harmonizes protocols across multiple children’s hospitals in Japan. It has conducted major trials in Kawasaki disease, RSV vaccines, and growth hormone therapy. Regulatory collaboration with PMDA has led to shortened review cycles for pediatric INDs, a major advantage for sponsors.
Their facilities include a child-centric Phase I unit with dedicated sedation and resuscitation protocols, and a multidisciplinary ethics committee trained in Japanese and ICH-GCP pediatric standards.
PGIMER Chandigarh & AIIMS Delhi (India)
India’s top public research institutions—PGIMER and AIIMS—serve as regional anchors for pediatric trials in South Asia. Both operate under ICMR guidance and are trusted by UNICEF, WHO, and Gates Foundation for large-scale pediatric trials, particularly vaccines, malnutrition, and infectious disease protocols.
PGIMER focuses on neonatal intensive care, infectious diseases, and pulmonary studies, while AIIMS runs multicenter pediatric oncology and nutrition studies. Both have fully functional Clinical Trial Units (CTUs) with trained CRCs and central labs, plus fast-track IEC processes for pediatric arms.
They also conduct government-backed longitudinal studies on pediatric asthma, typhoid conjugate vaccines, and anemia—positioning them as high-volume trial hubs with statistically significant child cohorts.
Seoul National University Hospital (South Korea)
SNUH is a cornerstone of pediatric trials in East Asia, blending clinical technology, regulatory alignment, and academic leadership. Its Pediatric Clinical Research Center supports GCP and MFDS-compliant trials, often partnering with global pharma on orphan drugs and pediatric oncology.
Their Phase I pediatric unit includes specialized anesthesia, imaging, and telemedicine monitoring—vital for complex pediatric rare disease protocols. SNUH is also active in international pediatric consortiums like ITCC and JAPAN-EMA alignment initiatives, enabling cross-border study acceleration.
They have conducted pivotal trials in pediatric epilepsy, ADHD, and metabolic disorders. By combining high recruitment potential with precision monitoring, SNUH delivers low dropout rates and consistent data integrity—key to successful pediatric studies.
Pediatric Research Strength in Middle East & LATAM
King Faisal Specialist Hospital (Saudi Arabia)
King Faisal Specialist Hospital & Research Centre (KFSHRC) is Saudi Arabia’s most advanced pediatric trial site, offering tertiary care pediatric subspecialties and ICH-GCP-compliant research infrastructure. KFSHRC is known for its work in pediatric hematology-oncology, rare metabolic conditions, and genetic disorders, many of which are regionally prevalent due to consanguinity.
It has a fully operational Clinical Research Unit with pediatric CRCs, a child-dedicated IRB, and multilingual assent materials. The hospital’s genetics lab supports on-site molecular diagnostics, speeding inclusion criteria assessment and pharmacogenomic screening.
Their partnership with King Saud University and SFDA streamlines regulatory clearance, often reducing site activation timelines for pediatric trials by 30–40% compared to regional peers.
São Paulo State University Hospital (Brazil)
As one of Brazil’s largest public academic hospitals, São Paulo State University Hospital (UNESP) is central to Latin America’s pediatric research ecosystem. It coordinates vaccine trials for diseases like dengue and yellow fever, and specializes in pediatric pulmonology, GI disorders, and congenital syndromes.
With access to vast public health databases and strong collaboration with ANVISA, the site supports rapid ethics submission and bilingual documentation workflows. Its pediatric trial unit integrates with Brazil’s Unified Health System (SUS), allowing access to diverse and underrepresented child populations for epidemiologically significant results.
UAE: Al Jalila Children’s Specialty Hospital
Al Jalila Children’s, the UAE’s only standalone pediatric hospital, is rapidly becoming a regional hub for pediatric clinical trials. Located in Dubai, it operates under the Dubai Health Authority and maintains collaborations with international CROs, pharma sponsors, and academic partners.
The site features a GCP-certified Pediatric Clinical Research Center, with a specialty in neurodevelopmental, behavioral, and rare genetic disorders. Al Jalila also implements caregiver-inclusive protocols, telehealth trial visits, and mobile sample collection—enhancing family participation and minimizing dropout rates.
Its alignment with MOHAP and Emirates IRB allows for accelerated approvals and multi-Emirate feasibility, a unique capability in the Gulf region. With rising regional demand for pediatric studies in endocrinology, autism, and metabolic diseases, Al Jalila is a site to watch.
| Institution | Core Specializations | Key Capabilities | Sponsor Advantages |
|---|---|---|---|
| King Faisal Specialist Hospital (Saudi Arabia) | Pediatric hematology-oncology, genetic disorders, rare metabolic diseases | Child-focused IRB, multilingual assent, in-house genetics lab, SFDA-aligned regulatory pathway | Faster site activation (30–40% reduction), localized diagnostics, ICH-GCP infrastructure |
| São Paulo State University Hospital (Brazil) | Vaccine trials (dengue, yellow fever), pulmonology, GI disorders, congenital syndromes | Ties to ANVISA, bilingual workflows, SUS integration, public health data access | Diverse population access, rapid ethics submission, epidemiologically significant sampling |
| Al Jalila Children’s Specialty Hospital (UAE) | Neurodevelopmental, behavioral, metabolic, and genetic pediatric conditions | GCP-certified center, caregiver-inclusive protocols, telehealth & mobile visits | Multi-Emirate feasibility, high retention rates, Gulf region CRO and sponsor alignment |
How CCRPS’s Clinical Research Coordinator Certification Supports Pediatric Trials
Pediatric trial design, ethics, and safety coordination
Pediatric trials demand more than just scientific rigor—they require specialized coordination, regulatory fluency, and family-centered communication. The Advanced Clinical Research Coordinator Certification (ACRCC) by CCRPS equips professionals with the exact tools needed to manage these complexities.
The course trains CRCs on minor assent, parental consent documentation, and protocol adaptations for vulnerable populations. Participants learn how to coordinate pediatric-specific procedures like sedation monitoring, guardian engagement, and adverse event reporting for younger cohorts. These aren’t just theoretical modules—they’re built around real-world case scenarios from multi-phase clinical environments.
ACRCC-certified professionals gain confidence managing pediatric trials across hospital, outpatient, and decentralized settings. The curriculum also emphasizes IRB navigation, SAE documentation, and flexible scheduling practices, all of which are crucial for reducing dropout in pediatric arms.
Link to CCRPS Clinical Research Coordinator Certification
Whether you’re overseeing a vaccine trial at a children’s hospital or managing data for a rare disease registry, your ability to handle child-focused workflows will define your site’s success. The CCRPS Clinical Research Coordinator Certification prepares you to deliver exactly that. It’s CPD-accredited, 100% online, and tailored for coordinators involved in global pediatric and adult research. By completing the certification, you'll be ready to align with trial sponsors prioritizing safe, ethical, and efficient pediatric study execution.
Frequently Asked Questions
-
Pediatric trials require completely different design parameters. Children have unique pharmacokinetics, meaning they metabolize drugs differently depending on age, weight, and organ development. Ethical considerations are more stringent due to vulnerable population status, requiring both parental consent and child assent. Trials must be non-invasive wherever possible and structured to minimize emotional distress. Dosing often needs formulation adjustments like syrups or dissolvables. Most importantly, retention relies on family-centered scheduling and communication. Sites that specialize in pediatric research understand how to integrate child psychology, clinical safety, and family logistics to create effective studies that would otherwise be impossible under adult trial frameworks.
-
Pediatric trials face higher regulatory scrutiny, tougher recruitment, and increased dropout risk. That’s why choosing the right clinical trial site isn’t just helpful—it’s mission-critical. Top-performing pediatric sites have dedicated ethics committees, trained CRCs, child-specific infrastructure, and strong sponsor relationships. They understand the nuanced documentation needed for minors, and often maintain pre-approved templates for faster IRB approvals. Most importantly, these sites have the ability to engage families, not just patients, through flexible scheduling, clear communication, and emotionally sensitive care. Choosing the wrong site means slower enrollment, incomplete data, and even trial failure.
-
Coordinators in pediatric trials need more than standard GCP knowledge. They should have training in informed consent for minors, adverse event reporting for children, and managing pediatric patient flow. Familiarity with child-specific trial tools, like assent forms and play-based communication aids, is essential. Many sponsors prefer CRCs who’ve completed the Advanced Clinical Research Coordinator Certification (ACRCC) or a similar program that includes pediatric case handling. These coordinators must also be emotionally intelligent, quick to de-escalate parent concerns, and meticulous in documentation—since compliance and family trust are the pillars of pediatric research success.
-
Yes. Pediatric trial regulations vary widely across regions. In the U.S., the FDA mandates additional protections for children under Subpart D, and requires separate safety review committees. The EU enforces the Paediatric Regulation (EC) No 1901/2006, which mandates a Pediatric Investigation Plan (PIP) for all new drug applications. Japan and South Korea require country-specific IRB and Ministry approvals, often with stricter consent documentation. In regions like the Middle East, bilingual materials and guardian-specific consent are required. Top pediatric sites navigate these hurdles through localized IRB templates, in-country regulatory liaisons, and sponsor-aligned protocol development.
-
Enrollment in pediatric trials is complex due to multiple barriers. Parents often hesitate to enroll their children due to safety concerns, especially in early-phase studies. The patient pool is also much smaller and more stratified—by age, weight, condition severity, and developmental stage. Trials need to account for school schedules, family logistics, and transportation limitations. Additionally, if protocols are too invasive or scheduling too rigid, dropout rates spike dramatically. That’s why successful enrollment often hinges on trusted pediatric sites with community ties, experienced CRCs, and patient-friendly protocols designed with families in mind.
-
Top sites reduce dropout by making trials child- and family-friendly. This includes flexible visit scheduling, educational support during absences, and compassionate, transparent communication throughout the study. Some sites integrate play therapy or use child life specialists to ease stress during procedures. Others offer telemedicine visits and mobile sample collection to minimize disruption. Sites that retain participants also train CRCs in age-appropriate assent conversations, helping children feel involved rather than coerced. Most importantly, retention improves when families feel heard, respected, and part of the care team—not just a data point.
-
Yes, but only with careful design. Decentralized trials (DCTs) can improve access for families in remote areas or those with demanding schedules. However, pediatric DCTs require telemedicine compliance tools, courier-supported sample handling, and user-friendly remote monitoring systems. Consent and assent must still be done thoroughly, often via secure video calls. Sites must also account for guardian education and tech literacy, since caregivers play a central role in executing remote tasks. Pediatric DCTs work best when hybrid—balancing in-person exams with remote data collection to reduce visits while maintaining clinical safety and parent confidence.
-
Several key trends are redefining pediatric research. First is the rise of gene therapy and rare disease trials, where pediatric cohorts represent the primary population. Second is the growth of patient registries and real-world evidence (RWE) to support smaller trials with long-term data. Third, more sponsors are implementing caregiver-informed protocol design, ensuring studies are feasible for real families. Telehealth, mobile monitoring, and digital consent tools are also expanding. Lastly, training and certification—such as the ACRCC from CCRPS—are becoming essential as regulators demand proof of site-level pediatric expertise before greenlighting studies.
Summing Up: Choosing the Right Pediatric Site
Choosing the right pediatric clinical trial site is never just about infrastructure—it’s about capability, compliance, and child-centered care. The best sites don’t just meet regulatory standards; they anticipate family needs, customize trial delivery, and deliver high-quality data from difficult-to-enroll populations.
Whether you’re running a global rare disease study or a regional vaccine trial, site selection will directly impact enrollment speed, retention rates, and ethical compliance. The institutions featured in this directory have proven their ability to lead, innovate, and collaborate across the pediatric research ecosystem.
For professionals managing or supporting pediatric trials, investing in proper site evaluation and team training—such as through the Advanced Clinical Research Coordinator Certification (ACRCC)—isn’t optional. It’s the clearest path to trial success, regulatory trust, and meaningful outcomes for children worldwide.
Poll: Which region do you believe offers the most advanced pediatric clinical trial infrastructure?
