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Genome Transplant Dynamics
Study Description: Heart and lung transplants can save lives, but long-term success is often limited by organ rejection that is hard to detect early. This stu…
A Multi-centre, Randomised, Placebo-controlled, Double-blind, Parallel-group Trial to Evaluate Safety and Efficacy of Spesolimab (BI 655130) in Adult Patients With Ulcerative Pyoderma Gangrenosum (PG) Who Require Systemic Therapy
The purpose of this study is to find out whether a medicine called spesolimab helps people with pyoderma gangrenosum (PG). The main aim is to see whether speso…
A Clinical and Genetic Investigation of Pituitary and Hypothalamic Tumors and Related Disorders
There is a variety of tumors affecting the pituitary gland in childhood; some of these tumors (eg craniopharyngioma) are included among the most common central…
A Single-arm, Multicenter, Phase III Study to Assess Efficacy, Pharmacokinetics, Safety and Tolerability of Atrasentan in Pediatric Patients of 2 to <18 Years of Age With Primary Immunoglobulin A Nephropathy (IgAN)
A Phase III, single-arm, multicenter pediatric clinical study evaluating atrasentan in children and adolescents aged 2 to \<18 years with primary immunoglobuli…
An Open-label, Multicenter Study to Assess the Effect of Zigakibart Treatment on Histologic, Circulating, and Excreted Markers of Kidney Disease and Dysfunction in Adult Patients With IgA Nephropathy.
The purpose of the study is to assess the effect of zigakibart on IgA nephropathy (IgAN) disease progression.
211At-MABG in Adults With Advanced Neuroendocrine Cancers
Phase I dose escalation study of 211At-MABG in adults with advanced pheochromocytoma / paraganglioma (PPGL) or other NET-overexpressing cancers (as evidenced b…
Emapalumab for the Treatment of Anti-MDA5 Antibody Positive Rapidly Progressive Interstitial Lung Disease
This is a proof of concept study to determine if Emapalumab appears effective for the treatment of anti-MDA5 antibody positive rapidly progressive interstitial…
Phase I Study of Aerosol Gemcitabine in Patients With Solid Tumors and Pulmonary Metastases
Any time the words "you," "your," "I," or "me" appear, it is meant to apply to the potential participant. The goal of this clinical research study is to find …
18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in lat…
18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in lat…
Refining and Pilot Testing a Decision Support Intervention to Facilitate Adoption of Evidence-Based Programs to Improve Parent and Child Mental Health
This proposal responds to NIMH Objective 4.2.c to develop "decision-support tools and technologies that increase the effectiveness and continuous improvement o…
PNOC021: A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients With Recurrent Low-Grade Gliomas and High Grade Gliomas
This phase I trial studies the side effects and best dose of trametinib and everolimus in treating pediatric and young adult patients with gliomas that have co…