PTN Pantoprazole Study Closes Enrollment

Congratulations to the PTN Pantoprazole Study Team for closing enrollment with 41 subjects. The study goal was to enroll 40 subjects in two age groups. The dedicated team worked hard to keep the study on track and accomplish this goal.

The Pantoprazole Study will evaluate the pharmacokinetics of pantoprazole in obese children and adolescents with gastroesophageal reflux disease (GERD) following administration of an oral dose of pantoprazole.

2015 Best Pharmaceuticals for Children Act Update

Dear Colleagues:

We would like to thank you for your continued interest and past input into the Best Pharmaceuticals for Children Act (BPCA) program at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Institutes of Health (NIH). As stakeholders in this important program, we would like to keep you abreast of current developments:

1. To date, all of our BPCA legacy studies—trials conducted prior to award of the Pediatric Trials Network (PTN) in 2010—have been completed. In September 2014, we submitted the Clinical Study Report (CSR) for the Lorazepam for Status Epilepticus study. A meeting with the FDA division was held in October, and the final CSR incorporating FDA comments was submitted in November 2014. Per FDA request, we recently submitted additional analyses and reports to the agency for a potential label change of this drug.

2. In 2014, we announced one official label change—the use of sodium nitroprusside in children for blood pressure control. This year, we have the privilege to announce the label change for meropenem for intra-abdominal infections. We anticipate that data from at least three additional studies (lithium, acyclovir, and diazepam) will be submitted to the agency by the end of 2015.

3. Between 2014 and to date in 2015, the program has submitted six additional CSRs to the agency. Five of these are PTN-led studies and include: a hydroxyurea bioequivalence study that complements the BABY HUG study; ampicillin for use in preterm neonates to treat infection; lisinopril for use in patients with renal transplant to lower blood pressure; clindamycin-dosing study in patients who are obese; and fluconazole for use in preterm neonates to treat infection. In October 2014, we also submitted a draft CSR for isotretinoin for use in treating neuroblastoma. Our hope is that these submissions will result in label changes in the near future.

4. In addition to the drug studies, the PTN and the BPCA Data Coordinating Center worked closely together to submit our first device study to the agency—the TAPE study, which was approved as of May 2015.

5. The PTN is currently conducting 7 new studies, including an innovative opportunistic study of commonly used medications that more than 1,300 children are receiving for their medical conditions. These new studies on diuretics, caffeine, pediatric opportunistic PK studies (POPs), antibiotics safety, Baby TAPE, sildenafil, and pantoprazole will improve our knowledge of how drugs are used in children.

6. Through the Pediatric Clinical Pharmacology T32 fellowship program, we continue to train new pediatric clinical pharmacologists while stimulating interdisciplinary collaboration among clinical, translational, and basic researchers in pediatric therapeutics.

We would also like for you to Save the date December 9, 2015, for our Annual BPCA Stakeholders Meeting. Held on the NIH campus in Bethesda, MD, the meeting will be conducted via webinar for all non-local attendees. You will be hearing from our logistics contractor soon with further details. We hope you can participate.

Finally, we would like to express our sincere appreciation to each of you for your interest in this important program. We look forward to working with you in the 2016 calendar year on our continuing areas of interest. Please feel free to contact me with any questions or concerns.

Best regards,

Perdita Taylor-Zapata, M.D.
Pediatric Medical Officer
Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB)
NICHD/NIH
Bethesda, MD
Phone: 301-496-9584
Email: taylorpe@mail.nih.gov