First participant enrolled in digoxin study

The PTN digoxin study achieved a major study milestone by enrolling its first participant. The study team aims to enroll up to 48 infants to determine the pharmacokinetics (how a drug travels through the body) and safety of digoxin prescribed to infants with single ventricle congenital heart disease (CHD).

While digoxin has been approved by the U.S. Food and Drug Administration for the treatment of heart failure in children and adults, more information is needed, particularly in children with single ventricle CHD.

Each year 40,000 infants born in the U.S. are diagnosed with CHD. Single ventricle CHD is the most serious and complex form of the disease. Currently, there are no drugs proven to reduce death during this period.

According to study Principal Investigator, Dr. Christoph Hornik of the Duke Department of Pediatrics, “Despite significant efforts by a number of different providers that treat infants with CHD, the mortality rate remains quite high and there are no drugs that have been shown to decrease mortality.”

This study will try to answer questions about the side effects of digoxin and learn more about how it is processed in the bodies of children with single ventricle CHD.

“Digoxin is an old drug, but there is some evidence to suggest it may be helpful, so studying it in this population is essential,” said Hornik.

To learn more about the digoxin study visit ClinicalTrials.gov.

Sildenafil safety study achieves major milestone

The Pediatric Trials Network (PTN) recently enrolled the fortieth infant in the Safety of Sildenafil in Premature Infants study. This is a major study milestone, as it rounds out the first cohort (or group of study participants).

The study aims to assess the safety of sildenafil in premature infants at risk of bronchopulmonary dysplasia (BPD) and determine preliminary effectiveness and pharmacokinetics (how a drug travels through the body). The study aims to enroll a total of up to 120 participants.

BPD is a common chronic lung disease that can affect premature newborns, often leading to life-long medical problems, prolonged hospitalization, and even death. Approximately 17,500 U.S. infants develop BPD each year.

Sildenafil, which is approved for the treatment of pulmonary hypertension in adults, may help improve lung development and is increasingly being used off-label in premature infants with BPD. However, the efficacy and safety of sildenafil in premature infants at risk for BPD is currently unknown.

“Our hope is that this study benefits premature infants and their families by providing more information on the safest and most effective dose of sildenafil to treat this life-threatening condition,” said Dr. Matthew Laughon, principal investigator and neonatologist at the University of North Carolina Hospitals in Chapel Hill, N.C.

To learn more about Safety of Sildenafil in Premature Infants visit ClinicalTrials.gov.

PTN LAPS Trial enrolls more than 100 patients

The PTN Long-term Antipsychotic Pediatric Safety (LAPS) Trial, which aims to assess the long-term health risks of risperidone and aripiprazole in children, has now enrolled more than 100 children. Risperidone and aripiprazole have been shown to be effective for the treatment of conditions such as schizophrenia and bipolar disorder in adults, but little is known about the long-term health risks and quality-of-life benefits for their use in children.

While several antipsychotics are FDA-approved in children, it is common for these drugs to be prescribed without FDA approval for conditions such as attention-deficit disorder, obsessive-compulsive disorder, and major depression.

The ultimate goal of the study is to provide long-term safety data to the FDA to update the risperidone and aripiprazole labels to include correct safety and dosing information. This information will allow doctors to provide the safest, most effective dose to children who require treatment with antipsychotics.

The enrollment of more than 100 children helps achieve nearly 30 percent of the overall enrollment goal of the LAPS Trial and is a key accomplishment for the two-year observational study.

Future Research

The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) follows a prioritization process to identify pediatric therapeutics and indications in need of study. Groups of experts assist with the prioritization, and there is an opportunity for public comment.

Criteria include

  • Current gaps in available evidence
  • Potential impact on children, society, and delivery of care

2018-19 Priority List

From the priority list, PTN investigators can submit a proposal to PTN. The PTN evaluates proposals, coordinates protocol development, and submits proposals to the NIH. If awarded, the PTN works with its sites to conduct the research.

PTN investigators:

Submit a study concept proposal via email.

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PTN Studies

The PTN conducts Phase 1-4 trials and data-based studies across a broad range of conditions affecting pediatric patients.

ONGOING STUDIES

Study Name Status Population Condition Study Type ClinicalTrials.gov ID
Anesthetics and Analgesics in Children (ANA) Enrolling Children (<18 y) Anesthetic and analgesic drugs of interest (DOIs) Prospective, multi-center, open-label PK and safety profile study NCT03427736
Antibiotic safety (SCAMP) Analysis ongoing Premature infants Intra-abdominal infections Interventional NCT01994993
Anti-epileptic obesity Enrolling Obese children Epilepsy Interventional NCT02993861
Commonly used drugs during lactation and infant exposure (CUDDLE) Enrolling Breastfeeding mothers and infants Various (drugs administered regularly by physicians) Interventional NCT03511118
Digoxin Enrolling Children (<6 m) Single ventricle congenital heart disease Phase I NCT03877965
Furosemide Analysis ongoing Premature infants Bronchopulmonary dysplasia Interventional NCT02527798
Long-term Antipsychotic Pediatric Safety Trial (LAPS) Enrolling Children Mono-antipsychotic therapies Observational NCT03522168
POPS Enrolling Children (<21 y) Various (drugs administered regularly by physicians) Observational NCT01431326
Sildenafil II Enrolling Premature infants Bronchopulmonary dysplasia Phase 2 NCT03142568
Timolol Enrolling Infants Infantile hemangioma Phase 2 NCT02913612

COMPLETED STUDIES

Study Name Status Population Condition Study Type ClinicalTrials.gov ID
Acyclovir Study published on DASH; label changed Premature infants Systemic infection Phase 1 NCT00942084
Acyclovir retrospective Study published on DASH Infants Herpes simplex virus Retrospective data collection
Ampicillin Study published on DASH Infants Bacterial infections including sepsis and meningitis Secondary data analysis
Anti-staph trio Completed Premature infants Staphylococcal infections Interventional  NCT01728363
Baby TAPE Study published on DASH Newborns and young infants NA (Weight estimation tool) Interventional  NCT01507090
Baby TAPE III Study published on DASH Newborns and young infants NA (Weight estimation tool) Interventional
Caffeine Study published on DASH Premature infants and infants Bronchopulmonary dysplasia Retrospective data collection
Clindamycin obesity Clinical study report submitted to FDA Obese children Staphylococcal infections Phase 1 NCT01744730
Diuretic safety Clinical study report submitted to FDA Infants Bronchopulmonary dysplasia Retrospective observational  NA
Fluconazole safety Study published on DASH Infants Candidemia Meta-analysis NCT00734539
Hydroxyurea Clinical study report submitted to FDA Children (2-4 y) Sickle cell anemia or sickle beta-zero thalassemia Phase 2 NCT01506544
Lisinopril PK Completed; label changed Children and adolescents (2-17 y) Kidney transplant; high blood pressure Interventional NCT01491919
Lorazepam Completed; label changed Children and adolescents (3 mo-18 y) Status epilepticus (Status 1) Interventional NCT00114569
Lorazepam or diazepam Clinical study report submitted to FDA Children and adolescents (3 mo-18 y) Pediatric status epilepticus (Status 2) Interventional NCT00621478
Meropenem Completed; label changed Infants Intra-abdominal infections Interventional NCT00621192
Methadone Study published on DASH Children Opiate withdrawal Observational NCT01945736
Metronidazole Completed Premature infants Serious infection caused by anaerobic bacteria Interventional NCT01222585
Midazolam Completed Children (≥2 y) Seizures Retrospective data analysis NA
Obesity informatics Completed Children Various Systematic literature review  NA
Pantoprazole Completed Obese children GERD Phase 1 NCT02186652
Pediatrix meta-analysis Published Infants Various Meta-analysis  NA
Personal protective equipment (PPE) Completed Research doctors and nurses Evaluating impact of various PPE Interventional NA
Sildenafil Completed Premature infants Bronchopulmonary dysplasia Phase 1 NCT01670136
TAPE Clinical study report submitted to FDA Children (2-16 y) NA (Mercy Method™ for weight estimation) Observational NCT01507090
Trimethoprim-sulfamethoxazole and clindamycin Study published on DASH Children Skin and soft tissue infections Data from 3 protocols

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