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Matched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-Thalassemia
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The int…
A Phase III, Randomised, Double-blind, Placebo-controlled, Parallel-group Study to Assess the Effect of AZD0780 on Major Adverse Cardiovascular Events in Patients With Established Atherosclerotic Cardiovascular Disease (ASCVD) or at High Risk for a First ASCVD Event
The purpose of this phase 3, randomized, placebo controlled, event-driven study is to assess the effect of AZD0780, an oral PCSK9 inhibitor, compared with plac…
A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease…
A Phase 1 Study to Evaluate the Safety and Tolerability of Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen. Research has shown t…
A Phase III Open-Label Extension Study to Evaluate the Long-Term Safety of Astegolimab in Patients With Chronic Obstructive Pulmonary Disease
The purpose of this study is to assess the long-term safety and to explore the efficacy of astegolimab in participants with chronic obstructive pulmonary disea…
A Pilot Study of Lenvatinib in Combination With Pembrolizumab in HPV-associated Recurrent Respiratory Papillomatosis Patients
This research study is studying Lenvatinib in combination with Pembrolizumab in people with human papillomavirus (HPV)-associated recurrent respiratory papillo…
Host Response to Infection and Treatment in Leishmania Infection of Humans
This study will examine the natural history of Leishmanial infections and their treatments. It will provide an opportunity for NIAID staff to learn more about …
Probiotic Supplementation Safety and Feasibility Study in Preclinical Alzheimer's Disease
The purpose of this study is to assess the safety and feasibility of an oral probiotic supplement (PS) intervention in individuals with dementia or mild-cognit…
MOMENTOUS Study (iMpact Of an Ecg ai ModEl oN The Diagnosis Of pUlmonary hypertenSion)
MOMENTOUS is a multi-center, randomized study to prospectively evaluate the performance of an ECG-based AI device to predict whether participants with intersti…
A Phase 1B Double Blinded Randomized Trial Evaluating Safety, Tolerability, and Immune Response of OSU-2131 a BZLF1 Peptide Vaccine With Stimulon™ QS-21 Solution Adjuvant in Healthy Volunteers and Patients Awaiting Solid Organ Transplantation
This phase 1B trial tests the safety, side effects and best dose of rh-Hsc70- BZLF1 peptide complex (OSU-2131) with Stimulon (Trademark) QS-21 and evaluates ho…
Natural History Study of CADASIL
Background: CADASIL (cerebral autosomal dominant arteriopathy with subcortical infarct and leukoencephalopathy) is a genetic disorder. It causes narrowing of …
Genetic Regulators of Metabolism and Development in Children
This is a prospective, non-randomized, non-blinded observational study. The overarching goal is to discover new disease-associated genes in children, while est…