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POP02 Includes Children with Down Syndrome

Posted on by Meagan.daly@duke.edu

A small child with down syndrome is enjoying with his father at a public park on a sunny day.

The POP02 study continues to enroll participants at an impressive rate. Nearly 15 participants enrolled in POP02 are children with Down Syndrome (DS). Enrolling children with DS in clinical trials helps achieve the goals put forth by INCLUDE.

The purpose of the INCLUDE project is to enhance clinical trial support for medication usage in children with DS and to promote training for clinical trials in those with DS for PTN and DS clinicians.

Specifically, the PTN DS will (1) Characterize the pharmacokinetics (PK), pharmacodynamics (PD), and pharmacogenomics (PGx) of understudied off-patent drugs administered to children and young adults with DS. (2) Develop a stand-alone protocol specifically for those with DS after convening thought leaders and experts. (3) Develop a training program for clinical researchers, both within the PTN and with external DS experts, to provide insight and guidance in trial design, recruitment, and engagement (including assent/consent) issues specifically in this population.

The National Institute of Child Health and Human Development (NICHD) supports this work through the Best Pharmaceuticals for Children Act (BPCA). Dr. Daniel Benjamin, Principal Investigator and Chair of PTN, will partner with Dr. Mara Becker of the Duke Department of Pediatrics as the INCLUDE Principal Investigators. For more information on the NIH’s efforts to support children with Down syndrome and their families, visit DSConnect.

The ABC Science Collaborative Shares “The Year in Review and a Path Forward”

Posted on by Meagan.daly@duke.edu

With support from the Pediatric Trials Network (PTN), The ABC Science Collaborative recently produced “The Year in Review and a Path Forward.”

This summary of the current science reflects feedback from leading scientists from 13 states across the nation. The science highlights proven best practices to safely return to school in the wake of COVID-19. Schools will have many decisions to make in the 2021 school year. This “Year in Review” offers a road map for school leaders and policymakers to use the latest science as they plan the path forward for K-12 in-person instruction.

About ABC Science Collaborative

The ABC Science Collaborative is an initiative that extends across 13 states, connecting scientists and physicians with school and community leaders to help understand the most current and relevant information about COVID-19. The program helps school leaders and state policymakers arrive at informed decisions about returning to school using data from their own communities. Our shared goal is to keep students, teachers, and their local communities healthy and safe. The ABC Science Collaborative is coordinated by the Duke Clinical Research Institute at the Duke University School of Medicine and is funded through grants from the National Center for Advancing Translational Sciences, the National Institutes of Health, and the Food and Drug Administration.

PTN Digoxin Study Reaches Enrollment Goal

Posted on by Meagan.daly@duke.edu

The Pediatric Trial Network’s (PTN) Digoxin study recently reached the targeted enrollment goal for its study population of infants with Congenital Heart Disease (CHD). Thanks to the hard work and diligence of the clinical care team part of this study, including the PTN team, the target enrollment goal was timely and successfully met to move forward with collecting data from study participants.

The Pharmacokinetics and Safety Profile of Digoxin in Infants with Single Ventricle Congenital Heart Disease study is collecting safety and efficacy data on infants with single ventricle CHD to determine the appropriate dosing and safety measures of the study drug digoxin.  Digoxin is approved by the U.S. Food and Drug Administration (FDA) to treat heart failure in patients with congenital heart disease. There has not been enough safety and efficacy data to determine proper dosing of this drug in pediatric patients with single ventricle CHD, which is what the PTN aims to change with this research effort.

Fifty study participants have been recruited for this study, where the drug digoxin is a standard of care protocol to treat heart failure by controlling the functions of the cardiovascular system. Study participants are infants <6 months of age who have single ventricle CHD and who have received an initial surgery to correct it, and whose healthcare team is planning on providing digoxin treatment. Each year about 40,000 infants born in the U.S. are diagnosed with CHD, with single ventricle CHD being the most serious and complex form of the disease.

To review more about the digoxin study, visit ClinicalTrials.gov.

 

 

Enrollment Goal Rapidly Approaches for PTN Digoxin Study

Posted on by Meagan.daly@duke.edu

Toddler keeps red plush heart. Closeup

The Pediatric Trial Network’s (PTN) Digoxin study is rapidly approaching an enrollment milestone for its unique study participant population.

Digoxin’s original study timeline projected enrollment to be completed by the end of May 2021. However, due to the onset of the COVID-19 pandemic and related shutdowns and delays, the study team adjusted its target to finish enrollment by June or July of 2021. Through, teamwork, diligence, and determination the study team was able to meet the original target enrollment date, much quicker than anticipated after COVID-19 delays. Investigators and clinical coordinators on this study team worked together to quickly identify and enroll prospective participants, and with the engagement of two new study sites, the team anticipates to soon reach the target population of 48 enrollees.

About 40,000 infants born in the U.S. are diagnosed with single ventricle congenital heart disease (CHD). Digoxin is approved by the U.S. Food and Drug Administration (FDA) for the treatment of heart failure in congenital heart disease and heart failure patients, yet more information is needed for dosing this drug in children with single ventricle CHD. According to study Principal Investigator, Dr. Christoph Hornik of the Duke Department of Pediatrics, “Although many different providers that treat infants with CHD have made significant efforts, the mortality rate is still high.”

Infant participants enrolled in this study have been diagnosed with single ventricle CHD, the most complex and fatal form of the disease. Researchers of the Digoxin study aim to find appropriate dosing and determine safety measures of the drug, as it is promising in reducing mortality rate in this population.

To review more about the digoxin study, visit ClinicalTrials.gov.

POP02 Enrolls Participants in Record Speed

Posted on by Meagan.daly@duke.edu

Family doctor examining throat of a small black boy while visiting him at home during coronavirus pandemic.

The Pediatric Trials Network (PTN), NICHD Funded, Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children per Standard of Care (POP02) Study has activated new sites in record speed.

In less than one year, the team selected approximately 60 sites and worked to activate >35 sites across the US and Canada. Site teams have overcome local challenges and enrolled almost 400 participants in the study, including those with acute COVID-19 and multisystem inflammatory syndrome in children (MIS-C). Over 70% of these children are underrepresented minority (African Americans, Hispanic/Latinos, and American Indians/Alaska Natives) who are disproportionately impacted by the COVID-19 pandemic.

The study structure previously put in place by PTN POPS served as the supporting framework and was helpful in PTN’s ability to establish a COVID-19-focused trial (POP02-COVID sub-study) more rapidly. The fast support of sites to expedite the activation process was paramount in getting the project off the ground.

“This past year was indescribably difficult for everyone, but our study team, site staff and investigators worked tirelessly and unified efforts to help close knowledge gaps during this public health crisis. Central to these efforts are our participants. We are indebted to the families and patients who willingly participate in research to help other children,” said Chi Hornik, POP02 Principal Investigator.

In addition to evaluating the dosing and safety of drugs of interest (Anakinra, Aspirin, Azithromycin, Canakinumab, Colchicine, Interferon, Lopinavir/Ritonavir, Remdesivir, Ribavirin, Ruxolitinib, Sarilumab, Tocilizumab), the POP02-COVID study aims to evaluate combinations of therapies, risk factors, and genetic factors that influence the susceptibility, severity, and outcomes of children affected by acute COVID-19 or MIS-C.

First Child with Down syndrome to Enroll in PTN Study

Posted on by Meagan.daly@duke.edu

Portrait of man and girl with down syndrome swingingThe Pediatric Trials Network (PTN) has enrolled the first child participant with Down syndrome into the Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children per Standard of Care (POP02) study.

This marks an exciting and significant milestone for the network. As part of the INCLUDE initiative, a directive from the National Institute of Health (NIH), PTN is committed to supporting clinical trials on conditions and diseases that affect people with Down syndrome, both to accelerate the development of new therapies for individuals with Down syndrome and to include them in ongoing clinical trials.

The POP02 study aims to expand upon the work done as part of PTN POPS, with hopes to shrink the gap between pediatric and adult dosing information available to prescribing physicians for their pediatric patients. In the initial phases, POP02 will focus on six drugs that have the potential to be involved in the treatment of COVID-19. The completed list of drugs that will be studied are: Anakinra, Aspirin, Azithromycin, Colchicine, Canakinumab, Interferon alpha & beta, Lopinavir/Ritonavir, Remdesivir, Ribavirin , Ruxolitinib, Sarilumab, and Tocilizumab.

The National Institute of Child Health and Human Development (NICHD) supports this work through the Best Pharmaceuticals for Children Act (BPCA). Dr. Daniel Benjamin, Principal Investigator and Chair of PTN, will partner with Dr. Mara Becker of the Duke Department of Pediatrics as the INCLUDE Principal Investigators. For more information on the NIH’s efforts to support children with Down syndrome and their families, visit DSConnect.

Anesthesia and Analgesics in Children (ANA) Hydromorphone Cohort Reaches Study Milestone

Posted on by Meagan.daly@duke.edu

Cheerful young boyThe Pediatric Trials Network (PTN) Anesthesia and Analgesics in Children (ANA) study has recently closed enrollment for its hydromorphone drug of interest (DOI) cohort (or group of study participants), a major study milestone. This means the goal for participant enrollment for this DOI cohort has been reached for this pharmacokinetic (PK) study, and researchers can study hydromorphone, an analgesic drug, in children.

The study’s principal investigator, Dr. Kanecia Zimmerman, and researchers are seeking to determine the appropriate dosing for anesthesia and analgesia drugs, which are administered to children for inpatient and outpatient surgical procedures to treat pain and anxiety. Many commonly used analgesia and anesthesia drugs are not prescribed to children due to their lack of safety and efficacy dosing guidelines in pediatric populations. The goal of this study is to determine how children’s bodies break down these drugs, and establish pediatric dosing guidelines for prescribers.

This study will enroll participants aged 2 to 17 who are already receiving an anesthetic or analgesic drug as part of their routine care. The recruited participants will not be randomized for this study due to their needed drug therapy. The following DOI cohorts were established for this study, with approximately 60 participants assigned to each cohort:  hydromorphone (analgesic), ketorolac (analgesic), ketamine (anesthetic), oxycodone (analgesic), and morphine (analgesic).  The drugs being studied are administered to children during their regular care. Once the study is completed, the results will be submitted to the U.S. Food and Drug Administration (FDA) to change the information and dosing guidelines for pediatric dosing.

INCLUDE Project Gains Perspective

Posted on by Meagan.daly@duke.edu

The INCLUDE directive calls for a trans-NIH research initiative to address critical health and quality-of-life needs for individuals with Down syndrome across the lifespan.

One of the three primary components of the INCLUDE initiative is to support clinical trials on conditions and diseases that affect people with Down syndrome, both to accelerate the development of new therapies for individuals with Down syndrome and to include them in ongoing clinical trials.

The National Institute of Child Health and Human Development (NICHD) supports this work through the Best Pharmaceuticals for Children Act (BPCA). Dr. Daniel Benjamin, Principal Investigator and Chair of PTN, will partner with Dr. Mara Becker of the Duke Department of Pediatrics as the INCLUDE Principal Investigators.

The following testimonials and videos help illustrate the importance of the INCLUDE initiative from a variety of perspectives: 

A Parent’s Perspective: Michelle Pfeiffer (PDF)
https://youtu.be/ZfQ9zSMf0S8

A Researcher and Sibling Perspective: Dr. Brian Skotko (PDF)
https://youtu.be/hNvcCKoZVjA 

A Pediatrician Perspective: Dr. Maria Stanley (PDF) 
https://youtu.be/GASDldUshJw

 

 

 

 

 

PTN Studying Multi-System Inflammatory Syndrome in Children

Posted on by Meagan.daly@duke.edu

While there have been relatively few confirmed cases and deaths from COVID-19 in children, researchers have been reporting an increased incidence of multi-system inflammatory syndrome in children (MIS-C) that is associated with prior or concurrent SARS-CoV-2 infections in children. Between March and October 1, 2020, over 1,000 MIS-C patients were reported from 44 state health departments, the District of Columbia, and New York City. The cases seem to be disproportionately affecting Black and Latino children. According to the CDC,

  • 41% were Hispanic or Latino
  • 35% were Black
  • 14% were non-Hispanic White

The average age for these patients was 8 years old, and obesity was the most commonly reported underlying medical condition, occurring in 30.5% of Hispanic, 27.5% of Black, and 6.6% of White MIS-C patients according to CDC data published in August.

The Pediatric Trials Network (PTN) is poised to prospectively gather data from children with COVID-19 and MIS-C and how best to treat them. The NICHD-funded Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children per Standard of Care (POP02) Study continues to evolve in an effort to support the treatment of younger COVID-19 patients. POP02 is evaluating several therapeutics to potentially treat COVID-19 in patients age 0 – <21 years old. About 60 different sites across the United States and Canada are being selected to participate in this important research.

Dr. Chi Hornik, POP02 study principal investigator, said that this platform and the opportunistic design of the study helps researchers find urgent answers on how these therapeutics work in children by enrolling a wide and diverse cohort of children.  “Some asymptomatic patients are presenting weeks later with MIS-C. We need more information, as quickly as possible, to help clinicians and families make the best decisions on treatment for children affected by acute COVID-19 as well as MIS-C,” she said adding. “We need to find the right dose and combination of treatments to ensure safety with optimal outcomes in all affected children.”

Of the affected children who have been enrolled in POP02, approximately 75% of them are from minority populations. “This pandemic is hitting communities of color disproportionately hard which is why a broad, national platform like POP02 is so important,” said Hornik.