This Phase 3 placebo-controlled study is planned to further investigate the efficacy, safety, and tolerability of apraglutide in the overall SBS-IF (short bowel syndrome associated with intestinal failure) population during 24 weeks of study treatment. It is expected...

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...

This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.

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. ...

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...

The purpose of this study is to evaluate the efficacy and safety of obudanersen in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.

This is a phase 2 randomized placebo-controlled crossover trial to determine the safety and efficacy of atomoxetine for treating obesity caused by loss-of-function variants in the melanocortin-4 receptor (MC4R), the most common cause of genetic obesity disorders. Ato...

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...

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...

The purpose of this study in children with 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 therapeutic dose will be evaluated up to FAH...

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...

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

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.

Open label, multi-centre, Phase Ia/b adaptive design study with an initial 2-stage Dose Escalation Phase followed by a Dose Expansion Phase.

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...

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...

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...

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.

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...