We've found 50 total result s for "Syndrome".
This is a single-center, open-label, basket phase 2b trial that will enroll Down Syndrome (DS) participants with at least one inflammatory skin condition (Atopic Dermatitis (AD) and/or Alopecia Areata (AA)). Patients will receive Abrocitinib 100 mg daily for 12 weeks...
Age: 12 years - 66+
Gender: All
A Phase 3, randomized, double-blinded, placebo-controlled study to evaluate the efficacy and safety of paltusotine treatment vs placebo as well as the long-term safety of paltusotine in adults with carcinoid syndrome due to well-differentiated neuroendocrine tumors. ...
Age: 18 years - 66+
Gender: All
The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed study medication treatment in the lead-in study TIS6463-203. TIS6463-204 is evaluating zagociguat in an open-label extension study ...
Age: Birth - 66+
Gender: All
The study is conducted in New York, New York at The Cohen Center for Recovery from Complex Chronic Illness at Mount Sinai. This is an IND-exempt, off-label, multi-ascending, randomized, placebo-controlled clinical trial of sirolimus (also known as rapamycin) in adu...
Age: 18 years - 66+
Gender: All
The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.
Age: 2 - 50 years
Gender: All
This will be a pilot multi-arm clinical trial investigating the feasibility of 6 weeks of Lumbrokinase (LK) as an intervention in three clinical cohorts: Long Covid (LC) Post-treatment Lyme disease syndrome (PTLDS) Myalgic encephalomyelitis/chronic fatigue syndrome (ME...
This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing...
Age: 12 years - 66+
Gender: All
This is a phase I multicenter clinical trial that aims to find the optimal dose for conducting a novel low-dose, multi-day oral food challenge (OFC) protocol for diagnosing food protein-induced enterocolitis syndrome (FPIES). Individuals ages 1-60 years with a histor...
Age: 1 year - 60 years
Gender: All
The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the...
Age: 3 - 11 years
Gender: All
This is a Phase 1/2, first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of JAG201 administered via intracerebroventricular (ICV) injection in pediatric and adult participants with SHANK3 haploin...
Age: 2 - 9 years
Gender: All
The purpose of this study is to assess Tacrolimus/Methotrexate/Ruxolitinib versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation
Age: 18 years - 66+
Gender: All
PRISM is a prospective, pilot research study that aims to systematically characterize the usefulness of CardiAQ MCG, a bedside magnetocardiography device, in the evaluation of myocardial ischemia and infarction status.
Age: 18 years - 66+
Gender: All
Chronic lymphocytic leukemia (CLL) is the most common leukemia (cancer of blood cells). The purpose of this study is to assess the safety of venetoclax in combination with obinutuzumab or acalabrutinib in the treatment of CLL. Adverse events and change in disease act...
Age: 18 years - 66+
Gender: All
The platform protocol is designed to be flexible so that it is suitable for a range of study settings and intervention types. Therefore, the platform protocol provides a general protocol structure that can be shared by multiple interventions and allows comparative an...
Age: 18 years - 66+
Gender: All
This study is researching an experimental drug called linvoseltamab combined with another drug called dupilumab. The study is looking at patients who have severe IgE-mediated food allergy. If the patient has an allergy, the body's defense system (immune system) overr...
Age: 18 - 50 years
Gender: All
This is an inpatient four-arm randomized control trial comparing single drug clonazepam (S arm), a two-drug combination clonazepam/olanzapine (D arm), and a three-drug combination clonazepam/olanzapine/buprenorphine (T arm) with treatment as usual (TAU arm) in the treat...
Age: 18 years - 66+
Gender: All
This prospective discovery study is designed to demonstrate the feasibility of using an unshielded, mobile MCG device in an Emergency Department (ED) setting. The main question it aims to answer is whether interpretable data can be reliably collected as part of an ED...
Age: 18 years - 66+
Gender: All
The purpose of this study is to evaluate the efficacy and safety of Luspatercept when administered at the maximum approved dose in low-risk Myelodysplastic Syndrome participants who require red blood cell transfusions.
Age: 18 years - 66+
Gender: All
A major impediment to emergency department (ED)-based HIV/HCV screening success is that often ED patients at risk for, or later diagnosed with, HIV and HCV decline testing. In this R01 project, the research team will assess how well a promising, easy-to-use, one-time...
Age: 18 years - 66+
Gender: All
This phase II trial tests how well zanubrutinib and lisocabtagene maraleucel (liso-cel) work together in treating patients with Richter's syndrome that has come back (recurrent) or does not respond to treatment (refractory). Richter's syndrome occurs when chronic lym...