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Observational Study of Children with Hypochondroplasia

The purpose of this study is to learn more about how children with hypochondroplasia grow. Hypochondroplasia is a genetic condition that causes short stature and short limbs. Study procedures include a body check-up (measurements) and ...


Other

Dupilumab in Adult and Adolescent Patients With Eosinophilic Gastritis With or Without Eosinophilic Duodenitis

The purpose of this study is to evaluate an experimental drug called dupilumab in people with active eosinophilic gastritis (EoG) with or without eosinophilic duodenitis (EoD). EoG and EoD are persistent allergic/immune diseases in which ...


Lymphoid Leukemia

AZD0486 in Adolescents and Adults With Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia

The purpose of this study is to learn more about an experimental drug called AZD0486 and determine whether it will be safe, tolerable, and effective in treating B-cell acute lymphoblastic leukemia (B-ALL). AZD0486 is ...


Brain and Nervous System, Melanoma, Other Hematopoietic, Soft Tissue

Cobolimab in Combination with Dostarlimab in Pediatric and Young Adult Participants With Newly Diagnosed and Relapsed/Refractory Tumors

The purpose of this study is to evaluate an experimental drug called cobolimab, given with dostarlimab (an anti-cancer medication used in adults) in children, teens, and young adults whose cancer has spread to other parts ...


Other

Ultragenyx_GTX-102-CL301

GTX-102 is a human ubiquitin-protein ligase E3A antisense (UBE3A-AS) transcript-targeted antisense oligonucleotide (ASO) intended for the treatment of Angelman syndrome (AS). Findings from the Phase 1/2 dose-finding study (GTX-102-001) suggest a positive efficacy signal and ...