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Health-Related Quality of Life and Clinical Utilization in Pediatric and Young Adult Patients With Hereditary Hemorrhagic Telangiectasia
This observational study evaluates health-related quality of life (HR-QoL) in pediatric and young adult patients aged 2-25 years with Hereditary Hemorrhagic Te…
A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
This is a Phase 3, multicenter, long-term, open-label study to evaluate the safety and efficacy of once-daily orally administered deucrictibant extended-releas…
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Peroral Doses of Inno8 in People With Haemophilia A
This study will test how different doses of study medicine (Inno8) work in the bodies of people with haemophilia A. The purpose of the study is to see if Inno8…
The "Savvy for Stroke Survivors" Study: Testing a Novel Tool to Help Stroke Survivors Take Their Medication
The goal of this study is to test if Savvy, a multimodal intervention (consisting of psychological exercises, a weekly pill organizer, and a text message remin…
A Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial of Descartes-08 in Patients With Generalized Myasthenia Gravis (MG)
The AURORA Study is evaluating the safety, tolerability, and efficacy of an investigational mRNA CAR T-cell therapy known as Descartes-08 in adults with acetyl…
A Single-centre, Open-label, Randomised, Single-dose Study to Evaluate the Effect of Pre- and Post-dose Meal Timings on the Pharmacokinetic Properties of Inno8 in Healthy Participants
The purpose of this study is to test a new medicine called Inno8. The study will test how eating and drinking before and after taking Inno8 affects how well it…
Hemorrhoidal Artery Embolization: Longitudinal Impact On Symptoms (HELIOS)
Hemorrhoidal artery embolization (HAE) is a novel treatment for symptomatic internal hemorrhoids. HAE involves the deliberate blockage (embolization) of enlarg…
Nutrition OUtReach In Systems of Healthcare
Many children and adults receiving medical treatments have higher costs, which can make it harder for them to afford groceries. When someone can't afford enoug…
Oral Deucrictibant for the Prophylactic and Acute Treatment in Patients With Bradykinin Mediated Angioedema With Normal C1 Inhibitor (BK-AE-nC1INH)
To assess the efficacy of prophylactic treatment with deucrictibant extended release (XR) tablet versus placebo in preventing angioedema attacks, and to also a…
Genetics of Neonatal Encephalopathy and Related Disorders
Investigators at Boston Children's Hospital are conducting research in order to better understand the genetic factors which may contribute to neonatal encephal…
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study With an Open-label Period and Long-term Extension to Assess the Efficacy and Safety of Rilzabrutinib in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)
This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb respo…
Recovery After Cerebral Hemorrhage--Improving Outcomes for Patients With Life-Threatening Neurologic Illness
Background: While the intensive care of patients with life-threatening brain illnesses has advanced tremendously, a large number of therapies are still withou…