236 Studies Now Enrolling
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Displaying 1 - 14 of 14
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: 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: 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: 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: Pediatrics
Investigator: Vidhu Thaker, MD
Status: Currently Recruiting
We will enroll children with severe early onset obesity (BMI greater than 99th percentile) diagnosed prior to 6 years of age or other endocrine disorders. We will ask questions about their health, eating behavior and perform a brief physical examination. We will collect saliva or blood to perform…
Condition: Pediatrics
Investigator: Olatundun Williams, MD
Status: Closed
The purpose of this study is to understand the life course of PIRD patients and their family members that have the same gene change but do not have the same symptoms. PIRD is mostly due to inherited changes in the genes that normally have immune cells fight infections and regulate the immune system. There are many types of problems that patients with PIRD…
Condition: Pediatrics
Investigator: Wendy Chung, MD, PhD
Status: Closed
The purpose of this research is to determine whether a new investigational drug called TAK-935 is safe and whether it may help treat epilepsy in children with 15q duplication syndrome or CDKL5 deficiency disorder.
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…
Condition: Pediatrics / Cancer
Investigator: Ran Reshef, MD
Status: Closed
Epstein-Barr Virus (EBV) is the virus that causes mononucleosis, also known as mono. Almost all adults have had EBV. For most people, EBV never causes a health problem. However, when their immune system is not working properly (people who have had a transplant and those who have immune disorders), EBV can cause certain…
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: 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: Pediatrics
Investigator: Cindy Neunert, MD
Status: Closed
The purpose of this study is to investigate the safety and effectiveness of eltrombopag (investigational drug) in treating children and adolescents with newly-diagnosed immune thrombocytopenia (ITP), which is a rare blood disorder.
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 / 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…