Center for Pediatric Research
Clinical Trials
A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) with Acute Leukemia or Myelodysplastic Syndrome (MDS)
This study is currently enrolling.
You are being asked to take part in this research study because you have been diagnosed with acute leukemia or myelodysplastic syndrome (MDS).
A Phase 2, Two-Stage, Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of RLS-0071 in Newborns with Moderate or Severe Hypoxic Ischemic Encephalopathy Undergoing Therapeutic Hypothermia with Long-Term Follow-Up (STAR)
This study is currently enrolling.
This study is being conducted to learn whether the investigational medication, called RLS-0071, may help babies, such as your own, who are affected by hypoxic-ischemic encephalopathy (HIE). HIE is a disease process where the ba ...
A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients with Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) that are Progressing or Causing Significant Morbidity
This study is currently enrolling.
This research is conducted to study an investigational drug referred to as PD-0325901 (also known as mirdametinib) that has not been approved by the US Food and Drug Administration (FDA) or any other Regulatory Authority for th ...
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study: 5 Independent Sub-studies of Setmelanotide in Patients with POMC, PCSK1, LEPR, SRC1, SH2B1, and PCSK1 N221D Gene Defects in the Melanocortin-4 Receptor Pathway
This study is currently enrolling.
Appetite (feeling of hunger and feeling of being full after eating) and weight can be controlled by certain genes located in the brain. These genes are part of the Melanocortin-4-Receptor pathway (MC4R). Sometimes there is a ge ...
AL46 Growth and Tolerance of Preterm Infants Fed an Experimental Human Milk Fortifier
This study is currently enrolling.
You are being asked to allow your baby to take part in this research study because he/she was born prematurely. Because your baby was born early, he/she will have a human milk fortifier (HMF) added to your breast milk or donor ...
An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)
This study is currently enrolling.
The primary objective of the study is to evaluate the long-term safety and tolerability of maralixibat.
Anova Surgical System – Registry Study
This study is currently enrolling.
You are invited to take part in a patient registry research study. A patient registry is an organized way to collect observational data from a large group of patients undergoing the same procedure. Observational data means that ...
Donor Breast Milk and Serum Ferritin Levels in Very Preterm Infants: A Prospective, Observational Study
This study is currently enrolling.
The purpose of the study is to compare the iron levels in very preterm infants receiving donor breast milk (DBM) to very preterm infants receiving maternal breast milks (MBM).
INTERMITTENT HYPOXIA AND CAFFEINE IN INFANTS BORN PRETERM
This study is currently enrolling.
You are being asked to allow your baby to be in this research study because your premature baby was given the medication caffeine as treatment for his/her immature breathing in the neonatal intensive care unit (NICU). Particip ...