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 Partially-Blind, Randomized, Controlled, Parallel-Group Dose Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFact (SF-RI 1 surfactant for inhalation combined with a dedicated drug delivery system) in Preterm Infant at Risk of Worsening Respiratory Distress Syndrome
This study is currently enrolling.
The goal of this study is to see if giving surfactant as an aerosol to babies while they are on nCPAP or nIMV will decrease their need to be placed on a ventilator and receive surfactant through a breathing tube or through a ca ...
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 ...
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Investigating the Efficacy and Safety of PF-04965842 Co-Administered with Background Medicated Topical Therapy in Adolescent Participants 12 to <18 Years of Age with Moderate-to-Severe Atopic Dermatitis
This study is currently enrolling.
The purpose of this research study is to compare the efficacy and safety of two doses of the study drug (PF-04965842) with a placebo when taken together with medicated and non-medicated therapy applied to the skin. Researchers ...
AHEP1531 Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT) - A Phase 2/3 Study
This study is currently enrolling.
Because hepatoblastomas are rare tumors, it has been difficult to progress research in this area. However, in this study, Children's Oncology Group will partner with other research groups in Europe and Japan to combine ef ...
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 ...