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Displaying 1 - 18 of 18

Kids MoD PAH Trial

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…
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Study of EDIT-301 in Patients with Severe Sickle Cell Disease (SCD)

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…
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Study of PF-07209326 in Patients with Sickle Cell Disease

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…
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A study for patients with Systemic Lupus Erythematosus using study drug ALX-0061

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
The goal of this study is to test a new lupus drug. This drug interrupts the function of a chemical in the body called IL-6, and IL-6 may be overactive in people with lupus. ALX-0061 is a study drug that will be administered subcutaneously to subjects with moderate to severe active, seropositive Systemic Lupus Erythematosus (SLE) compared to placebo.
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Rituximab for Systemic Sclerosis Associated Pulmonary Arterial Hypertension (SSc-PAH)

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.
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A study for patients with Lupus Nephritis using ACTHAR GEL

Condition: Autoimmune Disorders / Lupus
Investigator: Wooin Ahn, MD
Status: Closed
The purpose of this study is to determine if Acthar gel is a safe and effective therapy for patients with membranous (Class V) lupus nephritis. Class V lupus nephritis is associated with the development of chronic kidney disease and end-stage renal (kidney) disease, and there is no clear therapy for treatment. Currently used therapies arent completely…
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Study of CTX001 in Patients with Severe Sickle Cell Disease (SCD)

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…
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A study on subjects with systemic lupus erythematosus using study drug CC-220

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
This study is for patients who have been diagnosed with systemic Lupus erythematosus (SLE). SLE is a disease in which the body mistakenly attacks healthy tissue. It can affect the skin, joints, kidneys, brain and other organs. The purpose of this study is to test the effects of an investigational (being tested) study drug, CC 220, in order to find out if CC…
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A study for patients with lupus using the study drug XmAb5871.

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
The purpose of this clinical trial is to learn about the ability of an experimental treatment XmAb5871 to maintain lupus disease activity improvement achieved by a brief course of disease-suppressing intramuscular (IM) steroid therapy in lupus patients.
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Study of stem cell transplant effects in patients with sickle cell disease

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…
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A study for subjects with Lupus nephritis using an oral study drug MLN9708

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
The purpose of this study is to evaluate the safety and tolerability of an experimental investigational drug called ixazomib citrate (MLN9708) in patient with Lupus Nephritis. Lupus nephritis is a condition when the kidney is affected in the autoimmune disease known as systemic lupus erythematosus (SLE). SLE is a disease in which the immune system (the body…
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A study for patients with Lupus using study drug ustekinumab

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
If you have active Systemic Lupus Erythematosus, also known as SLE or Lupus, you may be interested to learn about a clinical research study that is being conducted to study the safety and effectiveness of an investigational medication called ustekinumab for active Lupus. Eligible study participants will be provided with study medication and study-related…
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A study for patients with pulmonary hypertension using study drug Riociguat

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.
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Study of Experimental Gel Drug in Subjects with Persistently Active Systemic Lupus Erythematosus (SLE)

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
We are studying a medication to see if it is effective in reducing the disease activity of systemic lupus erythematosus (SLE), which is the most common form of lupus. You may qualify for the Achtar study if you have persistently active SLE, are 18 years of age or older, and: Have rash and/or arthritis as part of your SLE symptoms Are currently taking…
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A study for patients with lupus nephritis using the study drug Obinutuzumab.

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
The purpose of this study is to test the safety and effectiveness of the study drug Obinutuzumab and to find out what effects, good or bad, it may have as an additional therapy in treating your lupus nephritis symptoms.
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A study for children with sickle cell using study drug Hydroxyurea

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…
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A study for pediatric patients with Systemic Lupus Erythematosus (SLE) using study drug Belimumab

Condition: Autoimmune Disorders / Lupus
Investigator: Lisa Imundo, MD
Status: Closed
The purpose of the study is to find out if study drug belimumab is safe and effective in treating children with active lupus who are also taking other lupus medicine. Belimumab (also known as BENLYSTA hasbeen approved in the United States, Canada, and Europe for the treatment of adults with active SLEwho are receiving other lupus medicines but not yet…
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Investigation of an ultrasound device for pulmonary artery denervation in patients with pulmonary hypertension.

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…
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