Unique Solutions to the Challenges of Pediatric Clinical Trials

Conducting successful pediatric clinical trials often requires overcoming challenges that adult trials may not face, including patient recruitment and retention, managing potentially difficult biostatistics from small trials, performing analyses on low sample draw volume (especially in infants), distinct regulatory requirements, and logistical challenges that may arise due to many geographically diverse study sites.

These challenges require demonstrated expertise and unique solutions. Precision can help.

Unique Solutions to the Challenges of Pediatric Clinical Trials

Conducting successful pediatric clinical trials often requires overcoming challenges that adult trials may not face, including patient recruitment and retention, managing potentially difficult biostatistics from small trials, performing analyses on low sample draw volume (especially in infants), distinct regulatory requirements, and logistical challenges that may arise due to many geographically diverse study sites.

These challenges require demonstrated expertise and unique solutions. Precision can help.

Recruitment & retention, biostatistics, and regulatory

Our clinical team has 25+ years of experience executing small trials with our focus on rare and orphan diseases. Through a patient-centric approach that reduces the burden of the trial to the patient, we facilitate patient recruitment and retention. And with our highly experienced biostatistics and regulatory team, we can create a study design and regulatory approach that is specific to the needs of a pediatric trial.

Learn more about our rare and orphan disease expertise >

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Logistics that support multi-site studies

Due to limitations of the patient pool, pediatric clinical trials often have more sites and investigators than trials in adults – and are often conducted globally. Precision’s specialty lab services and clinical sample management operations support multi-site studies with global transport and storage as well as robust technologies, like Epiontis ID, that works on frozen samples or dried blood and can be shipped in batches.

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Biomarker technologies that work with small sample volumes

Pediatric clinical trials can be challenged by the need to run biomarker assays on extremely limited sample volumes. Our specialty labs are equipped to overcome this hurdle with a range of technologies that deliver accurate data on very small volumes. One example is our proprietary immune monitoring and immune cell profiling technology, Epiontis ID. With much smaller sample volume requirements than traditionally used technologies such as flow cytometry, Epiontis ID can monitor up to 4 different cell types from 75uL of blood. Additionally, Epiontis ID can analyze “leftover” samples such as pellets or blood clots that may otherwise be discarded.

We also employ highly sensitive analyte detection platforms including MesoScale Discovery (MSD), Luminex, and Quanterix® SIMOA™.

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Epigenetic immune cell counting in human blood samples for immunodiagnostics

Precision for Medicine’s epigenetic immune cell diagnostic tool Epiontis ID shows promise in detecting severe immune diseases not currently identified in newborn screenings

Case Study: Enrolling a global natural history study: How to find 200 rare patients for a global natural history study

A sponsor selected Precision to manage a global natural history study in a rare, X-linked kidney disorder. The study had been recommended by the FDA because there was extremely limited published data on this rare disease-the goal was to characterize renal function and decline in patients with the disease prior to initiating a phase 2/3 clinical study. The study, to be conducted in the United States, Canada, Australia and in Europe, would follow 200+ patients for approximately 2 years.

Enrollment was by far the greatest study challenge, combining a low disease prevalence and a primary target population-young males-with life commitments such as school and work that left little time for participation in a trial that offered no therapeutic benefit for participants and would require traveling to a clinic every 3 to 6 months.

Find out how Precision overcame the enrollment challenge and generated the data needed to design a phase II/III study by reading the complete Case Study.

Discover how our pediatric clinical trial expertise can advance your clinical development program

Discover how our pediatric clinical trial expertise can advance your clinical development program

Services supporting clinical development of pediatric therapeutics

Clinical Trial Design

Advanced trial-design approaches-including basket, umbrella, and adaptive trials-deliver biomarker driven clinical research. Deep experience in these highly complex trial designs maximizes both insights and efficiency.

Biostatistics

Seasoned biostatisticians and statistical programmers deliver insight into every trial phase, from study design to regulatory submissions, all backed by meticulous documentation and data monitoring.

Immune Monitoring

Leverage sophisticated immune monitoring approaches, including flow, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

Explore other areas of expertise