The DELIVER-MS study seeks to answer the important question: Does early treatment with highly effective DMT improve the prognosis for people with MS? This is an area of significant controversy and no data currently exist to guide treatment choices for patients and clinicians. The study results will help guide overall treatment philosophy and will be applicable not only to a wide range of existing therapies but also to new therapies, meeting a significant unmet need in patient decision making and aiding the decision for medication approval by third parties.

Are you planning to receive the yellow fever vaccine for upcoming travel? This study may be for you! How well a vaccine works sometimes varies by geography, with good protection in one part of the world and poor protection in others. In this research study, the research team is investigating if this is due to different infections in communities, which could affect parts of the immune system that are needed for a good response to a vaccine. Healthy participants who receive the yellow fever vaccine in Uganda and Minnesota will have their levels of infections (from viruses, bacteria, fungi, parasites, and helminths [a type of parasitic worm]) compared. This will help us learn more about relationships between these infections, how they affect immune systems, and how that affects the body’s response to a vaccine.

The purpose of this study is to find out if using a computer program (called iDose) to guide infliximab dosing is more effective and safer than using standard infliximab dosing over 52 weeks. All patients in this study will be receiving infliximab as part of their medical care, this study is only looking at two different methods of determining the dose and timing of administration.

The clinical investigation objective is to study the safety and efficacy of an anti-platelet-free antithrombotic regimen in patients with advanced heart failure treated with the HM3 LVAS.

To compare the outcomes of patients with complex lesions treated with imaging guidance with IVUS versus angiography only.

This is an open-label single arm trial of letermovir (LTV, Prevymis) for prevention of recurrent CMV infection in allogeneic hematopoietic cell transplantation (HCT) recipients with history of CMV infection. The study is being conducted at MSKCC and at the University of Minnesota. Thirty-six HCT recipients who are ≥12 years of age, had clinically significant CMV infection treated with CMV antivirals and are at high risk for recurrent CMV infection, defined as receiving a transplant from an HLA mismatched donor (including cord blood), acute or chronic graft versus host disease (GVHD) requiring either topical and/or systemic steroid treatment within 14 days prior to enrollment, or T cell-depleted (CD34+-selected) allograft, will be eligible to participate in the study.

Feasibility Study to Evaluate the Safety and Efficacy of Cellular Matrix BCT-HA Kit as a New Treatment Option for Dehydrated and Wrinkled Skin

The purpose of this study is to better understand how tobacco and nicotine products affect our bodies. In this observational study smokers, vapers, smokeless tobacco users, nicotine replacement product users, non-users, and ex-users will be asked to provide biological samples. We will look for biological “markers” (biomarkers), or chemical changes in the body, that occur due to tobacco or nicotine exposure. Collected samples will be used for the development of biomarkers of toxicant exposure and for assessing exposure between the different groups. The intent is to eventually use these biomarkers to improve detection, prevention, and treatment strategies for tobacco-related diseases. This study will allow us to test currently used biomarkers, and to establish a biorepository (sample bank) to identify and develop new biomarkers associated with tobacco exposure and cessation. The type of samples and amount collected will depend on the specific biomarker(s) being developed or tested. Potential samples include saliva, cheek (buccal) & oral cells, blood, urine, hair, and/or nail clippings.

The purpose of this trial is to evaluate the efficacy, safety, and tolerability of BI 1015550 9 mg bid and 18 mg bid compared to placebo in patients with IPF in addition to patient’s standard of care over the course of at least 52 weeks. New treatments with better tolerability are needed for patients with IPF to further reduce the decline in lung function and improve quality of life. Based on its anti-inflammatory and antifibrotic properties and the preliminary clinical evidence described, BI 1015550 may provide an additional treatment option to patients with pulmonary fibrosis irrespective of concomitant treatment with standard of care.

Specific Aims: Specific Aim 1: Determine whether a three-month course of daily brivaracetam reduces below-level neuropathic pain in SCI. The objective of this aim is to assess efficacy of a three-month course of brivaracetam to reduce neuropathic pain in men and women with SCI. We will assess change in pain intensity and related outcomes, including mood, satisfaction with life, and community integration. We will monitor drug adverse events and tolerability. Specific Aim 2: Determine whether a three-month course of daily brivaracetam increases parietal operculum brain connectivity. The objective of this exploratory aim is to assess the effect of a three-month course of brivaracetam treatment on parietal operculum activation and connectivity in SCI. We will assess changes in cortical activity of related pain perception regions and networks in the brain in response to brivaracetam treatment compared to placebo using rsfMRI and pain-related task-based fMRI. To achieve this aim, we will test the working hypothesis that brivaracetam increases parietal operculum activation and connectivity compared to placebo, using rsfMRI, task-based fMRI, and a validated image processing protocol. We will also examine changes in network connectivity and brain activity in the insula, because of its concurrent reported importance for neuropathic pain in SCI. We will assess the association between functional connectivity of the parietal operculum and the insula and change in pain intensity in response to brivaracetam treatment. Specific Aim 3: Determine whether baseline microRNA-485 levels are associated with response to brivaracetam treatment. The objective of this exploratory aim is to assess microRNA expression as a potential predictive biomarker of response to brivaracetam treatment. To achieve this, we will test the working hypothesis that baseline circulating miR-485 levels predict change in pain intensity in response to brivaracetam treatment. To test this, we will use Next-Generation sequencing. We will assess miR-485 levels at baseline and after a three-month treatment course.

This Phase 3 study is being conducted to assess the long-term safety, tolerability, and efficacy of sotatercept in PAH. Long-term followup of patients receiving sotatercept is important to understand the maintenance and durability of treatment effect (especially in the presence of background PAH therapy) and to provide greater opportunity for pharmacovigilance following sotatercept treatment in the selected patient populations. This LTFU study is supported by data from the PULSAR study (Phase 2, NCT03496207), in which treatment with sotatercept resulted in hemodynamic and functional improvements in the study participants, including those receiving maximal PAH therapy with double/triple drug combinations and intravenous prostacyclin.

This is a Prospective, open-label, non-inferiority, randomized controlled trial (1:1 randomization) with blinded end-point analysis comparing the use of Prednisone versus Prednisone plus Methatrexate in the treatment of Cardiac Sarcoidosis.

This is a multicenter, randomized, double-blind, placebo-controlled, study comparing the efficacy and safety of intravenous (IV) efzofitimod 3 mg/kg and 5 mg/kg versus placebo after 48 weeks of treatment. This study will enroll adults with histologically confirmed pulmonary sarcoidosis receiving stable treatment with oral corticosteroid (OCS), with or without immunosuppressant therapy.

This research trial studies biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglio-neuroblastoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.

Treatment guidelines for high risk or relapsed solid tumors consisting of a busulfan, melphalan, thiotepa conditioning followed by an autologous peripheral blood stem cell transplant and, if appropriate, disease specific radiation therapy at day 60+

This therapy involves the use of consolidation chemotherapy, autologous stem cell rescue, post-transplant radiation therapy and a maintenance phase with Isotretinoin (Accutane, 13-cis-retinoic acid) therapy. If available, patients should also consider post-transplant therapy with cytokines and monoclonal antibody (ch14.18) on a COG or NANT trial. Ideally, patients should begin consolidation chemotherapy no later than 8 weeks after the start of Induction Cycle #6. (It is strongly recommended to begin consolidation within 4-6 weeks after starting Induction Cycle #6).

This is a treatment protocol for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. There is no research element except the collection of routine clinical data.

This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HSCT. While it will primarily be applied for the treatment of non-malignant diseases (NMD), on occasion it may be used to treat patients with malignant disorders as well.

This protocol will characterize the effects of deep brain stimulation (DBS) location (both adverse and beneficial) on motor signs in people with Parkinson’s disease (PD). This information can be used to inform future DBS protocols to tailor stimulation to the specific needs of a patient. If targeted dorsal GP stimulation is shown to significantly improve motor features that are typically resistant to dopamine replacement therapy, these experiments will likely have major impact on clinical practice by providing a potential strategy to these medically intractable symptoms.

The primary objective of the study is to assess the efficacy of patient controlled sedation (Self-management of sedative therapy) using dexmedetomidine to reduce anxiety, delirium incidence and duration of mechanical ventilation compared to usual sedation practices in mechanically ventilated subjects.

Comorbid AUD+AnxD is a significant barrier to successful AUD treatment. Converging evidence implicates overlap in dysregulation of systems governing stress response (HPA, ANS, CNS) for symptom development in AUD and AnxD. However, this must be systematically demonstrated in comorbid AUD+AnxD. We will assess markers of multi-system biological stress regulation (at rest and in response to laboratory challenge) in alcohol use disorder (AUD) inpatients with and without co-occurring anxiety disorder (AnxD), as well as those with AnxD who do versus do not receive a cognitive behavioral treatment that specifically targets comorbid AUD-AnxD. Laboratory measures include 1) cortisol (to assess the hypothalamic–pituitary–adrenal axis system [HPA] function; 2) heart rate variability (to assess autonomic nervous system [ANS] function), and 3) threat-potentiated startle (to assess central nervous system [CNS] function). Laboratory assessments will occur at the following times: 1) shortly after AUD treatment admittance (Visit 2: Pre-Treatment), 2) immediately following the 4-week AUD treatment (Visit 3: Post-Treatment), 3) 1 month following AUD treatment (Visit 4: 1-Month Follow-Up), and 4) 4 months following AUD treatment (Visit 5: 4-Month Follow-Up). Self-reported alcohol intake will be assessed at baseline as well as at the 1- and 4-Month Follow-Ups to determine whether laboratory stress measures predict treatment outcomes. A single laboratory assessment of healthy controls will serve as a normative reference for characterizing patient laboratory responses in terms of dysregulation and re-regulation.

We are doing the RARE study to learn more about Cystic Fibrosis (CF). CF is caused by mutations in a gene that produces a protein called the cystic fibrosis transmembrane conductance regulator (CFTR). In people with CF, the CFTR does not function correctly. Medications are being developed to help the CFTR function better, but those medications mostly benefit people with common CFTR mutations. There are more than 1,900 mutations of the CF gene. Some of these mutations are rare and found only in a few people. The goal of this research study is to collect specimens (blood, nasal cells, rectal cells) from people with rare CFTR mutations. Another purpose of this study is to create induced pluripotent stem cells or iPS cells. “Pluripotent” stem cells are cells that can be changed into almost any cell type of the body (such as lung or intestine). They can be kept alive and stored indefinitely. There are different kinds of pluripotent stem cells. Inducted pluripotent stem cells can be created from many different kinds of specimens (such as blood, nasal cells, rectal cells). This is different from embryonic stem cells, which can only be derived from embryos. The specimens collected during this study and iPS cells created from them will be stored for use in future research to learn more about CF and study the effect of new medications. This could identify new medications that may help people with rare CFTR mutations.

The goal of this study is to provide comprehensive longitudinal assessments of a cohort of PD patients before, during, and after DBS surgery, including neurological, neurophysiological, and neuropsychological data.

The purpose of this study is to better understand how switching from smoking to the use of electronic cigarettes (e-cigarettes) may change users’ exposures to various harmful chemicals. Your participation will also help us to understand how nicotine that is present in e-cigarettes is taken in and modified by your body.