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Displaying 1 - 82 of 82
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Currently Recruiting
This is a global, multicenter, placebo-controlled, phase 3 study evaluating the efficacy and safety of riliprubart in adult participants with CIDP who are refractory or had an insufficient response to standard of care (SOC) therapies, defined as Ig administered IV (IVIg) or SC (SCIg), or corticosteroids. Treatment duration will be 48 weeks. Participant must…
Condition: Lung Disease / Pulmonary Hypertension
Investigator: Usha Krishnan, MD
Status: Currently Recruiting
This study is being done to determine whether early combination therapy with two study drugs called sildenafil (REVATIO) and bosentan (TRACLEER) can improve pulmonary hypertension when compared to using sildenafil alone. Pediatric pulmonary arterial hypertension (PAH) is a severe medical problem that often worsens with time. It is caused by problems with…
Condition: Blood Disorders / Amyloidosis
Investigator: Rajshekhar Chakraborty, MD
Status: Currently Recruiting
The purpose of this study is to see how safe and effective is the study drug, ABBV-383. ABBV-383 works by binding to a specific protein called B-cell maturation antigen, which is found on the surface of cells affected by amyloidosis. By engaging T-cells, a type of white blood cell, ABBV-383 helps activate the immune response to target and remove the…
Condition: Lung Disease
Investigator: Jeanine Marie D'Armiento, MD
Status: Currently Recruiting
Lymphangioleiomyomatosis (LAM) is a rare lung disease primarily affecting young women. Currently, LAM is often diagnosed in patients with radiology imaging (CTs) and/or lung biopsies; however, these methods can be imprecise, invasive, and/or can carry significant risks. The current study aims to devise a less invasive tool for diagnosis of this rare…
Condition: Lung Disease
Investigator: Christine Kim Garcia, MD
Status: Currently Recruiting
The purpose of this study is to identify genetic causes of lung diseases and to develop a research repository so that we can better understand, prevent and treat these conditions. You should consider joining this study if you have been diagnosed with a lung disease that has a genetic cause or if other family members have been diagnosed with a similar lung…
Condition: Blood Disorders / Amyloidosis
Investigator: Rajshekhar Chakraborty, MD
Status: Closed
The purpose of this study is to evaluate whether birtamimab plus standard of care will improve survival in subjects with Mayo Stage IV AL amyloidosis. Birtamimab has not yet been approved by the Food and Drug Administration (FDA). During the first phase of the study, the purpose of this study is to evaluate whether birtamimab plus standard of care will…
Condition: Lung Disease
Investigator: Christine Kim Garcia, MD
Status: Closed
The purpose of this study is to identify genetic causes of lung diseases and to develop a research repository so that we can better understand, prevent and treat these conditions. You should consider joining this study if you have been diagnosed with a lung disease that has a genetic cause or if other family members have been diagnosed with a similar lung…
Condition: Blood Disorders
Investigator: Aaron Viny, MD
Status: Closed
This is a Tissue Repository to collect and store tissue samples from normal donors and patients with hematological disorders for future study research studies. The purpose of the storage bank is to obtain a large number of the bone marrow aspirates, bone marrow biopsies, and/or blood as well as buccal smears (to be used as a non-hematologic control for each…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the…
Condition: Lung Disease
Investigator: Ana Navas-Acien, MD, PHD, MPH
Status: Closed
The use of electronic cigarettes, also known as e-cigs or vaping, has increased among former smokers and young adults who have never smoked. Relatively little is known, however, about their health effects (whether they are adverse, have no effect, or are beneficial). The purpose of this research study is to evaluate (1) the association of e-cig use with…
Condition: Lung Disease
Investigator: Sanja Jelic, MD
Status: Closed
The purpose of this research study is to help us understand whether blood markers of lung injury are worse in adult patients with interstitial lung disease and obstructive sleep apnea compared with patients with interstitial lung disease who do not have obstructive sleep apnea.
Condition: Lung Disease
Investigator: Gebhard Wagener, MD
Status: Closed
We are asking you to participate in a research study to determine if we can detect minor lung injury that we cannot currently measure with available tests. This injury can occur when somebody is smoking for a long time or when somebody has COPD (chronic obstructive pulmonary disease). We want to compare the results of people who have never smoked, people…
Condition: Blood Disorders / Amyloidosis
Investigator: Divaya Bhutani, MD
Status: Closed
The purpose of the study is to determine if the drug, CAEL-101 in combination with plasma cell treatment improves overall survival for patients who are diagnosed with stage 3b of AL amyloidosis. Patients who participate in this study will receive CAEL-101 as well as plasma cell treatment or Placebo as well as plasma cell treatment to compare the effects.
Condition: Lung Disease
Investigator: Monica Goldklang, MD
Status: Closed
We are asking you to participate in a research study to determine if we can detect changes in enzymes in the blood that may influence lung function decline in COPD (chronic obstructive pulmonary disease), in particular after COPD exacerbations. The procedures of the study include a pulmonary function test (breathing test), a short questionnaire, and blood…
Condition: Blood Disorders / Amyloidosis
Investigator: Divaya Bhutani, MD
Status: Closed
The purpose of the study is to determine if the drug, CAEL-101 in combination with plasma cell treatment improves overall survival for patients who are diagnosed with stage 3a of amyloid light chain (AL) amyloidosis. Patients who participate in this study will receive CAEL-101 as well as plasma cell treatment or Placebo as well as plasma cell treatment to…
Condition: Blood Disorders
Investigator: Suzanne Lentzsch, MD
Status: Closed
The purpose of this research study is to see whether the presence or absence of certain genes is associated with the development of AL amyloidosis in subjects 60 years of age or older with the blood disorders smoldering multiple myeloma (SMM) and monoclonal gammopathy of undetermined significance (MGUS). Each person's disease has different genetic…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Andrew Eisenberger, MD
Status: Closed
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
Condition: Lung Disease / Chronic Obstructive Lung Disease
Investigator: Anna Norweg, OTR
Status: Closed
Capnography-Assisted, Learned, Monitored (CALM) Breathing is a mind-body intervention for COPD that uses breathing exercises and biofeedback with the goal of reducing shortness of breath, anxiety symptoms and improving overall quality of life. The intervention is a 4-week (1 hour, twice weekly) program taking place in-person at New York Presbyterian…
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
The purpose of this study is to evaluate the safety and effectiveness of an antibody drug (STI-6129) in patients with AL amyloidosis that has returned or has not responded to treatment (relapsed/refractory). The study drug (STI-6129) will be given in 3 injections through a needle, into a vein as an intravenous (IV), with 21 days between each injection.
Condition: Blood Disorders
Investigator: Markus Mapara, MD
Status: Closed
This study is being done to learn about the safety and efficacy of CTX001 (the "Study Product") to treat -Thalassemia. CTX001 is "investigational", which means that the Study Product is considered experimental and is not approved by the Food and Drug Administration (FDA), European Medicines Agency (EMA) or any other regulatory (…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
This is a Phase 2 study for patients with myelofibrosis previously treated with one or more Janus Kinase(JAK) Inhibitor. A JAK inhibitor targets a certain pathway that may be causing cancer to grow, bystopping or inhibiting the cycle of development of the pathway. There is one JAK inhibitor that has beenapproved for the treatment of patients with…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Claire Keating, MD
Status: Closed
This study is being done to learn more about the safety and effectiveness of VX-440 used in combination with tezacaftor and ivacaftor in patients with cystic fibrosis. VX-440 given in combination with tezacaftor and ivacaftor is an investigational drug; investigational means the drug is not approved by the Food and Drug Administration (FDA), and is still…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Emily DiMango, MD
Status: Closed
The purpose of this study is to find out whether the study drug altaluren can help lung function and can reduce the symptoms associated with cystic fibrosis (CF), and decrease the number of hospitalizations and the use of antibiotics for CF-related lung infections. The study will also investigate whether the drug can help improve overall patient quality of…
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
This research study is being done to determine if an investigational drug, SY-1425 (tamibarotene) works in treating AML (acute myeloid leukemia) and MDS (myelodysplastic syndrome) in patients with a certain biomarker in their blood (a blood test that may indicate the drug target is present in your type of AML or MDS).
Condition: Blood Disorders
Investigator: Ran Reshef, MD
Status: Closed
Itacitinib is an investigational drug that is being developed by Incyte Corporation for use in the treatment of acute graft-versus-host disease (GVHD). At this time, it is not known whether the study drug has an effect on sperm or eggs, whether they are secreted in the semen or whether they have an effect on a fetus. We would like to collect medical…
Condition: Lung Disease
Investigator: Nina Patel, MD
Status: Closed
This study is for patients with systemic sclerosis (SSc) and associated Interstitial Lung Disease. Systemic sclerosis or systemic scleroderma is a disease that causes thickening of the skin and the internal organs which often includes the kidneys, heart, lungs and gastrointestinal tract. Your disease affects the skin but also it affects the lungs. As the…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this study is to collect information on whether idasanutlin is effective in treating polycythemia vera (a slow-growing blood cancer in which your bone marrow makes too many red blood cells) and what effects, good or bad, idasanutlin has on you. Idasanutlin is an experimental drug, which means Health Authorities (such as the US FDA) have not…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to collect information about how patients with FAP respond to the study drug ALN-TTR02 over 18 months. This includes studying any potential improvements in FAP and any side effects that participants may have from the study drug. In addition, the study will examine how the body handles (distributes, break down, eliminates) the…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Nancy Green, MD
Status: Closed
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Emily DiMango, MD
Status: Closed
This study is being done to learn more about the safety and effects of the combination of VX-661 and ivacaftor in subjects with Cystic Fibrosis (CF).
Condition: Blood Disorders
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to learn more about the safety and effects of CTX001 in patients with severe SCD. CTX001 is created by changing (editing) the DNA in your own blood stem cells near a gene called BCL11A which is stored in a solution called dimethyl sulfoxide (DMSO) to try to prevent cells from dying while they are frozen. BCL11A is the area in the…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
You are being considered to participate in this research study because you have type I or II diabetes with current treatment control and, you are experiencing painful diabetic peripheral neuropathy (DPN) in both lower extremities legs.The specific events that result in painful diabetic peripheral neuropathy are not well understood, but high blood sugar,…
Condition: Lung Disease / Pulmonary Hypertension
Investigator: David Lederer, MD
Status: Closed
The purpose of this study is to study the effects of a new study drug known as Riociguat in people diagnosed with pulmonary hypertension (PH) associated with idiopathic interstitial pneumonia (IIP). Patients will be in the study for approximately 26 weeks in which they will take the pill Riogciguat three times a day.
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
The main purpose of this study is to see if people with certain types of anemia due to MDS will experience a decreased need for regular blood transfusions if they take luspatercept plus best supportive care. The safety of luspatercept will be also evaluated in this study.
Condition: Lung Disease
Investigator: Keith Brenner, MD
Status: Closed
The objective of the Block-COPD Study is to determine if beta-blockers are safe and effective at reducing the number of exacerbations or flare-ups that people living with Chronic Obstructive Pulmonary Disease (COPD) experience. A beta-blockers is a drug that is taken by mouth and is currently used to treat a wide range of diseases including high blood…
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to evaluate the safety, pharmacokinetics (how much the study drugs are absorbed in your body at different times), and efficacy (how well the study drugs work) of venetoclax in combination with azacitidine compared to azacitidine alone in subjects with previously untreated higher-risk MDS.
Condition: Lung Disease / Cystic Fibrosis
Investigator: Hossein Sadeghi, MD
Status: Closed
This study is for patients who have been prescribed tobramycin inhalation powder (TOBI Podhaler) or another inhaled medication used to treat cystic fibrosis patients whose lungs contain bacteria called Pseudomonas aeruginosa. The purpose of this research study is to assess how well TOBI Podhaler works on treating Pseudomonas aeruginosa in the lungs of…
Condition: Blood Disorders
Investigator: David Diuguid, MD
Status: Closed
The main purpose of this research study is to determine the effect of the study drug compared to placebo in increasing red blood cell counts and reducing the severity of symptoms of Sickle Cell Disease (SCD). A placebo is a dummy drug: it looks like the study drug but contains no medicine. Subjects being asked to take part in this research study have SCD.…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the efficacy and safety of study drug, fingolimod taken daily compared with a placebo (does not contain active medication) on delaying disability progression in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The study will consist of 3 periods: a Screening Period (lasting for up to 45…
Condition: Lung Disease
Investigator: Emily DiMango, MD
Status: Closed
This research is being done to look at how a medicine called Losartan helps people with Chronic Obstructive Pulmonary Disease (COPD) with emphysema, a disease of the lungs. COPD is often caused by cigarette smoking. It includes the symptoms of emphysema and chronic bronchitis. Although some medications for COPD reduce symptoms and prevent exacerbations, few…
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
The purpose of this study is to find out if MLN9708 plus dexamethasone improves against systemic light chain amyloidosis better than the physicians choice chemotherapy treatment. Physicians choice chemotherapy treatment is a treatment regimen that is commonly used to treat amyloidosis patients. The study will also determine if MLN9708 plus dexamethasone can…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Emily DiMango, MD
Status: Closed
The main purpose of this study is to look at the effectiveness, safety, and tolerability of study drug CTX-4430 when compared to a placebo (sugar pill) taken once-daily for 48 Weeks in adult patients with CysticFibrosis (CF).
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Subjects with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) will be enrolled in the study and Intravenous Immunoglobulin (IVIg) will be given to eligible subjects on the study. CIDP is a chronic inflammatory condition that causes the immune system to attack the nerves. IVIg is one common treatment for CIDP. IVIG is a blood product that is…
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
This study is for patients have been diagnosed with Intermediate or High-risk myelodysplastic syndrome (MDS). MDS is a condition where there is a low count of white blood cells, red blood cells and platelets because of a malfunction of bone marrow that is responsible for producing healthy mature blood cells. evaluate the combination of the investigational…
Condition: Lung Disease
Investigator: Christine Kim Garcia, MD
Status: Closed
The Families At-Risk for Interstitial Lung Disease (FAR-ILD) study is designed to detect and study the earliest stages of ILD in adults with a family history of ILD. FAR-ILD is a non-interventional study. This means that FAR-ILD is not involved in testing any new interventions or therapies. The purpose of FAR-ILD is to gather data on family members of…
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to evaluate the safety, pharmacokinetics (how much of the study drugs is present in your body at different times), and efficacy (how well the study treatment works) of venetoclax alone and of venetoclax in combination with azacitidine in subjects with higher-risk MDS after HMA-failure.
Condition: Lung Disease / Cystic Fibrosis
Investigator: Claire Keating, MD
Status: Closed
This study is being done to learn more about the safety and effectiveness of study drug VX-661 in combination with ivacaftor in patients aged 12 years and older with Cystic Fibrosis (CF). In this Study there are two treatment groups. If the patient meets all the rules for being in the Study, they willbe in one of the two treatment groups. The patient will…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this phase I study is to test the safety and tolerability of ruxolitinib at different dose levels in combination with decitabine in patients with accelerated or blast phase Myeloproliferative Neoplasm (MPN), which is a group of diseases of the bone marrow in which excess cells are produced. You may qualify to take part in this research study…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the efficacy and safety of ISIS TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy (FAP). The purpose of this study is to determine if ISIS 420915 can help people with mild or moderate FAP. To be in this study you must have FAP and be able to walk without help or walk with using only one cane.
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
This is a research study for subjects that have been previously treated for systemic light chain (AL)amyloidosis (a disease in which abnormal protein deposits can damage organs and tissues in your body) and nowrequire further treatment. The main purpose of this study is to evaluate the safety and determine the maximum tolerated dose of aninvestigational new…
Condition: Lung Disease
Investigator: David Lederer, MD
Status: Closed
This study is for subjects with Idiopathic Pulmonary Fibrosis (IPF). IPF is an uncommon long-term disease that causes scar tissue to build up in the lungs, damaging the lung tissue. Idiopathic means that there is no known cause. Subjects with IPF have symptoms such as cough, shortness of breath, and reduced ability to exercise. The purpose of this study is…
Condition: Blood Disorders
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant condition.
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
International GBS Outcome Study (IGOS) is a study of the Inflammatory Neuropathy Consortium (INC) and Peripheral Nerve Society (PNS) on disease course and outcome in Guillain-Barr syndrome (GBS). Guillian Barre syndrome is an autoimmune disorder affecting the peripheral nervous system, leading to paralysis and potential fatality if it progresses to involve…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
This study will test an experimental drug, momelotinib, for the treatment of primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis. Primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis are all myeloproliferative disorders; which are serious bone…
Condition: Lung Disease / Pulmonary Hypertension
Investigator: Elana Bernstein, MD
Status: Closed
This study involves two infusions of either rituximab or placebo, two right heart catheterizations (6 months apart), and a variety of other clinical study procedures and exams.
Condition: Blood Disorders / Anemia
Investigator: Katherine Ender, MD
Status: Closed
The main purpose of the study is to find out whether Triferic, when taken by mouth (orally) with Shohls solution, is safe and effective for the treatment of Iron Refractory Iron-Deficiency Anemia (IRIDA). The study drug, ferric pyrophosphate citrate (FPC), is also called Triferic. It is an iron salt that has been approved by the Food and Drug Administration…
Condition: Lung Disease
Investigator: David Lederer, MD
Status: Closed
This study is for patients who have been diagnosed Idiopathic Pulmonary Fibrosis (IPF). The purpose of the IPF-PRO registry is to collect data and biological samples that will support future research studies. The IPF-PRO Registry is actively enrolling patients to collect information regarding the natural history of IPF and healthcare interactions. The…
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
The study is being done to see if the combination of bendamustine and dexamethasone will help people with amyloidosis that has returned after standard treatment. Bendamustine is currently approved by the Food and Drug Administration (FDA) for treatment of chronic lymphocytic leukemia (CLL) and is under clinical development in the United States for the…
Condition: Lung Disease
Investigator: Angela DiMango, MD
Status: Closed
Our clinic is seeking patients with nontuberculous mycobacterial, or NTM, lung disease caused by Mycobacterium avium Complex (MAC), ages 18 years and older, for possible participation in a clinical trial. The CONVERT Study is evaluating the safety and effectiveness of an inhaled investigational drug called Liposomal Amikacin for Inhalation, or LAI
Condition: Blood Disorders / Amyloidosis
Investigator: Thomas Brannagan, MD
Status: Closed
HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Charcot-Marie-Tooth type 1A (CMT1A) disease is a rare, slowly progressing and debilitating hereditary condition commonly causing weakness of muscles in the feet and lower parts of the legs. There is currently no approved drug to cure CMT1A disease, and the available treatments commonly target the pain and inflammation associated with the disease. In order…
Condition: Blood Disorders
Investigator: Margaret Lee, MD
Status: Closed
This study is for patients with sickle cell disease (SCD). SCD is an inherited genetic disorder in which red blood cells become abnormally shaped (sickle cells). These sickle cells may block blood vessels and result in a vaso occlusive crisis (VOC). This leads to inflammation and tissue damage and causes pain in various parts of the body. The purpose of…
Clearing Lungs With Study Drug VX-371 Solution for Inhalation in Patients Primary Ciliary Dyskinesia
Condition: Lung Disease
Investigator: Angela DiMango, MD
Status: Closed
The purpose of this study is to find out if you can take the prescribed dose of VX-371 without problems, and whether VX-371 is safe and helps subjects with PCD. VX-371 is called an investigational or experimental drug because it has not been approved for use in people by the Food and Drug Administration (FDA), the branch of U.S. government which approves…
Condition: Blood Disorders
Investigator: Markus Mapara, MD
Status: Closed
We are doing a research study to find out whether it is safe to use a virus to change the genes of bone marrow cells that become red blood cells. We hope that this type of therapy may be helpful in people with sickle cell disease, but it has not been tested before. Genes, which we inherit from our parents, are pieces of information which provide the…
Condition: Lung Disease
Investigator: Hossein Sadeghi, MD
Status: Closed
The purpose of this research study is to gather information about how respiratory syncytial virus (RSV) disease affects babies who were born earlier than their full-term due date, and how the hospitalization for RSV disease affects the parents/guardians daily routine activities. Information about the cost of healthcare and other costs related to the RSV…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
We are doing this study to find out if the research drug known as Rozanolixizumab can have additional benefit for people who are already receiving IVIG( Intravenous Immune Globulin) treatment for CIDP, and to have further safety evaluation of the research drug as well. If you choose to be part of this research study, the following procedures will occur:…
Condition: Lung Disease
Investigator: Angela DiMango, MD
Status: Closed
The purpose of this clinical research study is to evaluate the safety and effectiveness of INS1007 as a treatment for non-CF bronchiectasis and to measure the concentration of INS1007 in the blood after treatment. INS1007 is taken as tablets and has been studied in animals and humans. Patients like you, with non CF bronchiectasis, may have problems with…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle cell disease. Patients will receive busulfan…
Condition: Lung Disease
Investigator: David Lederer, MD
Status: Closed
The purpose of this study is to find out if subjects can take the prescribed dose of GBT440 without problems, and whether GBT440 is safe and helps subjects with IPF. GBT440 is called an investigational or experimental drug because it has not been approved for use in people by the Food and Drug Administration (FDA), the branch of U.S. government which…
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
The purpose of this study is to test for any good and bad effects of the study drug called isatuximab. Isatuximab may or may not improve your AL amyloidosis, but it could also cause side effects. Isatuximab is not approved by the Food and Drug Administration (FDA). Isatuximab has been shown to have good effects in some patients with recurring multiple…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Peripheral Neuropathy is a result of nerve damage, which may cause weakness, numbness, tingling, and pain in your hands, feet, and other areas of your body. Over 20 million Americans are affected by peripheral neuropathy, most commonly caused by diabetes. In approximately 25-50% of all people with peripheral neuropathy, the cause is unknown. These cases…
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
The purpose of this study is to evaluate whether study drug NEOD001 will improve survival in subjects with AL amyloidosis and/or increase the interval of time that they can go without requiring hospitalization for problems with their hearts. This study will also evaluate whether NEOD001 improves the function of subjects' organs that have been affected…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Claire Keating, MD
Status: Closed
The purpose of the ELEVATE CF study is to evaluate an oral investigational medication called VX-440 tosee if it is safe, effective, and well-tolerated when taken alone or with two other oral medications(VX-661 tezacaftor and ivacaftor) in people with cystic fibrosis (CF). This triple combination(TC) is being tested to see if it improves chloride transport (…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this research study is to determine the effects of INCB050465 in combination with ruxolitinib treatment on spleen size and/or symptoms and to learn about any of the side effects that might occur during or following dosing with this combination of molecules in patients diagnosed with myelofibrosis.
Condition: Blood Disorders / Amyloidosis
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the efficacy and safety of ION-682884 given for 24 months in patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) also known as Familial Amyloid Polyneuropathy (FAP), and determine if it can help people with mild or moderate polyneuropathy. To be in this study you must have FAP and be…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Emily DiMango, MD
Status: Closed
The purpose of this study is to assess how effective the study drug Pulmaquin is in the management of chronic lung infections with Pseudomonas aeruginosa in subjects with non cystic fibrosis (nonCF) bronchiectasis by evaluating the time to first pulmonary exacerbation. A pulmonary exacerbation is defined as the new appearance or worsening in 4 or more of…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
This study is looking at using topiramate as a potential therapy for patients with cryptogenic sensory peripheral neuropathy. Patients who are randomized into the study will return to the clinic for follow up every 16 weeks. The total length of the study is 96 weeks.
Condition: Lung Disease / Pulmonary Hypertension
Investigator: Erika Berman-Rosenzweig, MD
Status: Closed
This is a clinical research study designed to determine the safety and effectiveness of the TIVUS system, an investigational device used in the treatment of PAH. The TIVUS System is intended to treat patients with pulmonary artery hypertension by injuring the sympathetic nerves surrounding the pulmonary vasculature (denervation) using ultrasonic energy. The…
Condition: Blood Disorders / Amyloidosis
Investigator: Divaya Bhutani, MD
Status: Closed
The purpose of this study is to see if daratumumab when given with three other drugs cyclophosphamide, bortezomib (VELCADE) and dexamethasone is useful for treating patients with Amyloid light chain Amyloidosis (also known as AL Amyloidosis). The study will look at what happens (both good and bad) when daratumumab is given with these three drugs compared to…
Condition: Lung Disease
Investigator: Nina Patel, MD
Status: Closed
The purpose of this study is to investigate the safety of adding nentedanib to treatment with pifenidone in patients with Idiopathic Pulmonary Fibrosis (IPF). Eligible patients must be receiving consistent treatment with pifenidone for at least 16 weeks on a stable dose. Nintedanib will be added as an additional treatment for IPF (combination treatment) for…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Markus Mapara, MD
Status: Closed
This study is being done to learn more about the safety and effects of CTX001 (the Study Product) in patients with severe Sickle Cell Disease (SCD). The Study Product is considered investigational; investigational means the Study Product is not approved by the United States Food and Drug Administration (FDA). The goal of this study is to see if a single…
Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to see how effective two doses of an investigational new drug called IgPro20 is when given subcutaneously (a shot given into the fat layer between the skin and the muscle) to treat CIDP in patients that still require IVIG and also to look at how safe the IgPro20 is for patients.
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