The largest CAR-T trial for pediatric solid tumors has opened in the US and UK, enrolling up to 60 young patients across four sites. The NexTGen study targets relapsed or refractory cancers and focuses first on safety and feasibility.
New treatments for rare pediatric neurological conditions show promise, with zorevunersen reducing seizures in Dravet syndrome patients and an SMA therapy under FDA review following stem cell research breakthroughs.
March of Dimes awarded the 2026 Richard B Johnston Jr, MD Prize to hematologist Leonard Zon for pioneering zebrafish research that illuminated blood development in embryos and led to breakthrough treatments for genetic blood disorders in children.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07601581 |
A Trial to Determine People's Explicit vs. Implicit Preferences for Gender-inclusive Sexual and Reproductive Health Content |
COMPLETED | NA |
| NCT07571811 |
Pharmacokinetic and Pharmacodynamic Investigations of Fetal Anesthesia During Maternal Fetal Surgery |
NOT_YET_RECRUITING | |
| NCT07549646 |
24VA021; VATCH Trametinib for Ras/MAPK Pathway VAs |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT07545005 |
Impact of a Produce Prescription Program and Nutrition Education Intervention on Cardiovascular Health in Youth |
NOT_YET_RECRUITING | NA |
| NCT07544979 |
Creation of a Decision Aid for Coronary Anomalies |
NOT_YET_RECRUITING | NA |
| NCT07543822 |
24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT07532109 |
Validation of the Vestibular Migraine Patient Assessment Tool and Handicap Inventory (VM-PATHI) for Adolescents |
NOT_YET_RECRUITING | |
| NCT07513038 |
Use of Indocyanine Green (ICG) for the Diagnosis of Biliary Atresia |
RECRUITING | PHASE1/PHASE2 |
| NCT07476430 |
Readiness Outcomes Affecting Return to Sport 3.0 |
NOT_YET_RECRUITING | NA |
| NCT07474675 |
Evaluation of a Point-of-care Lateral Flow Assay in Glial Fibrillary Acidic Protein (GFAP) and D-dimer in Diagnosis of Large Vessel Occlusion Acute Ischemic Stroke in a Pediatric Hospital |
NOT_YET_RECRUITING |
