This study will enroll with children, adolescents and young adults that are less than 22 years old that have returned or 'relapsed' acute myeloid leukemia (AML) diagnoses. Within this group, the study is also targeting those are also unable to receive additional chemotherapy treatments containing anthracycline, which is a type of antibiotic that…
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…
This study is called a screening study and the purpose of this study to find better ways to diagnose and treat leukemia that has come back after treatment or difficult to treat. Bone marrow, blood, and medical information about cancer and treatment will be collected. The results from this screening study may give other information about leukemia that is…
This is a phase 1 study for participants with relapsed/refractory acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or juvenile myelomonocytic leukemia (JMML) diagnosis. This study will observe the safety, side effects, and best dose of Imetelstat as an investigational drug, or drug that is not approved by the Food and Drug Administration (FDA…
This study will be performed to determine if Panzyga (the study drug) can help with preventing infections in patients who have chronic lymphocytic leukemia (CLL). Panzyga is a study drug administered via injection into the vein, which contains human antibodies also called immunoglobulins. It is manufactured by Octapharma. Your participation in this clinical…
The purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with…
The purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs…
You are being asked to participate in this study because you have a myeloproliferative neoplasm (MPN) that has transformed to accelerated or blast phase. MPN is a group of diseases in which the bone marrow makes too many blood cells. The different types of MPN are myelofibrosis (MF), essential thrombocythemia (ET), or polycythemia vera (PV). Over time,…
You are being asked to participate in this study because you have been diagnosed with a blood cancer called Acute Myeloid Leukemia (AML) with or suspected to have a specific gene mutation (TP53 mutation) for which you have not received any prior AML treatment. Tumor protein p53 (TP53) is a gene that codes for a tumor suppressor protein, which is normally…
The purpose of this study is to determine the optimal dose of oral CGT9486 in patients with Advanced Systemic Mastocytosis (AdvSM) and to determine the efficacy of CGT9486 at the selected optimal dose in patients with Advanced Systemic Mastocytosis (AdvSM). This includes Aggressive Systemic Mastocytosis, Advanced Systemic Mastocytosis with an Associated…
The purpose of this study is to determine the effectiveness of the study treatment- bendamustine and rituximab (BR) followed by venetoclax for 12 months. The combination of the drugs bendamustine and rituximab is a commonly used treatment for your disease. Venetoclax is an oral drug that blocks a protein called BCL-2 which is present on CLL cells. With this…
Patients who will be receiving immune effector cell (IEC) therapy for the treatment of their cancer (leukemia) are being asked to participate in this study. IEC can produce a harmful side effect called Cytokine Release Syndrome (CRS). CRS involves a release of a large amount of proteins into the bloodstream. This may cause changes in blood pressure and…
The purpose of this study is to compare the effects, both good and bad, of magrolimab in combination with azacitidine, to those of azacitidine in combination with placebo, to find out which is better for treating patients with MDS (Myelodysplastic Syndrome). Other purposes of this study include determining the quantity of magrolimab in the blood, aspects of…
The purpose of this study is to assess the safety, effectiveness, pharmacokinetic (PK how long the drug is in your body and how it is metabolized) and pharmacodynamics (PD where does the drug go in your body and what is its biological effect) of the study drug, GFH009, in patients with relapsed or refractory hematologic malignancies including acute myeloid…
The purpose of this clinical research study is to evaluate the safety (any good or bad effects) of CYNK-001 and to assess the effects of CYNK-001 on your Acute Myeloid Leukemia (AML). Also, the study plans to determine the maximum tolerated dose (highest safe dose) of CYNK-001 and assess whether CYNK-001 can eliminate your residual (leftover) disease.…
The purpose of this study is to test the good and bad effects of the combined treatment, using the drugs cetuximab and avelumab as well as avelumab by itself. Avelumab is a drug that works by triggering the immune system so it can try to attack the cancer again. Avelumab works in a similar way to cemiplimab, which is the usual drug for treatment of your…
This research study is evaluating an investigational drug, called cirmtuzumab, in combination with astandard drug, called ibrutinib (Imbruvica), as a possible treatment for patients with CLL/SLL or MCL. All cells in the body receive signals to grow and survive, but sometimes these signals get out of control, causing too much cell growth. When the growth of…
The purpose of this study is to find out what dose of pacritinib is safest and works best for participants. Pacritinib is an experimental drug, which is a drug that is being tested and is not currently approved by the Food and Drug Administration (FDA) in the US. The study will be conducted at about 85 study clinics in North America, Europe, Israel and…
The purpose of this study is to: Test if the lifespan of study participants is increased when uproleselan is added to standard chemotherapy medicines compared to standard chemotherapy alone; test if the number of study participants achieving disease remission is increased when uproleselan is added to standard chemotherapy medicines compared to standard…
This study is for patients with Richter's transformation (RT). The purpose of this study is to see if a new type of investigational drug called PNT2258 that may be effective in blocking the "on" signal to cancer cells, eventually causing them to die. We are trying to see if PNT2258 provides evidence of anti-tumor effect by measuring what…
The purpose of this study is to find if PTC299 is safe to treat people with relapsed/refractory acute myeloid leukemia (AML) as well to determine the dose of PTC299 that can be given without causing severe side effects. AML is a cancer of the blood and bone marrow (spongy tissue inside bones where blood cells are made). PTC299 is being developed to…
The purpose of this study is to compare the safety, tolerability, and effectiveness of ruxolitinib, an investigational drug, compared to anagrelide, an FDA approved drug. Investigational means that the study drug has not been approved by the FDA for treatment of ET. Patients will be assigned to one of two groups. Group A will receive ruxolitinib and placebo…
The purpose of this study is to find the dose of imetelstat that best treats myelofibrosis. (A doseis a measured amount of a drug taken at one time.) The effects of imetelstat on myelofibrosis as well as any side effects will be explored.
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…
The main purpose of this research study is to investigate if a drug called Vedolizumab is safe and effective in preventing acute graft-versus-host disease (attack of donor cells on recipient healthy organs after transplant) of the gut in comparison to placebo (dummy drug). This will be studied in patients undergoing Allogeneic Hematopoietic Stem Cell…
The purpose of this study is to evaluate how well the study drug ABT-199 works and safety of ABT-199 in subjects with Chronic Lymphocytic Leukemia (CLL) whose cancer has come back (relapsed) after treatment or no response (refractory) to treatment with B-cell receptor signaling pathway inhibitor therapy, namely Ibrutinib and/or Idelalisib. In addition,…
The overall purpose of this part of the study is to determine the best dose of JCAR017 for treating adult participants with relapsed or refractory B-cell Chronic Lymphocytic Leukemia (CLL), and Small Lymphocytic Lymphoma (SLL) and to understand the side effects that participants with your disease may have when they are treated with JCAR017.
The purpose of this study is to test the safety of the combination of the experimental drugs called atezolizumab and guadecitabine (not yet approved by the U.S. Food and Drug Administration FDA or any other regulatory authority outside the United States) to find out what effects, good or bad, the combination of atezolizumab and guadecitabine has on patients…
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,…
The purpose of the study is to test how effective and safe the study drug SGI-110 is compared with current standard treatment for AML. The study drug, SGI-110, is changed in the body into a compound called decitabine. Decitabine is an approved drug in the US for treatment of patients with myelodysplastic syndrome (MDS) and in Europe for treatment of…
The purpose of the study is to see if a medicine called gilteritinib/ASP2215 given in combination with atezolizumab is both effective and safe as a treatment for AML patients with mutations in the FLT3 gene. Some AML patients have a mutation in the gene called FLT3. When patients have a FLT3 mutation, more of the FLT3 protein is on the leukemic cells, or…
The purpose of the study is to evaluate the safety, tolerability (the effect of the drug on your body), pharmacokinetics (the effect of your body on the drug) and maximum tolerated dose of sertraline in combination with cytosine arabinoside (ara-C, cytarabine) when given to patients with treatment-resistant myeloid leukemia. In addition, the effect of the…
This study is for patients with Acute Myeloid Leukemia (AML). The purpose of this study is to determine if decitabine plus JNJ-56022473 can be safely combined and is active for your disease. The study drugs used in this study are JNJ-56022473, an experimental drug and DACOGEN ( also known generic as decitabine), a drug already used to treat your type of…
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…
The main goal of this study is to find out about how duvelisib acts in patients with CLL/SLL who were previously treated with a BTKi (Bruton Tyrosine Kinase inhibitor - commercially available ibrutinib or another investigational BTKi). Duvelisib is an investigational drug, meaning that it is not FDA approved, designed to try to stop cancer growth by…
This study is being done to see how well tolerated the investigational drug TGR-1202 is in comparison to other similar therapeutics agents in treating patients with chronic lymphocytic leukemia (CLL).
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…
This is a clinical research study for subjects with Myelofibrosis (Primary, Post-Polycythemia Vera, and Post-Essential Thrombocytopenia) and thrombocytopenia. The purpose of this study is to determine if pacritinib is better at reducing spleen size and a reduction in symptoms than the best available therapy. This is a randomized, controlled study.…
The purpose of this research study is to determine the preliminary safety and effectiveness (how well something works) of alvocidib when used after the standard drug decitabine in patients with MDS. The study will also gain information about the PK (the process by which a drug is absorbed, distributed, metabolized and eventually eliminated by the body),…
This study is for adult subjects with head and neck cancer (oral cavity, oropharynx middle part of the throat, hypopharynx bottom part of the throat, or larynx part of throat between the base of the tongue and the windpipe) that has either returned after treatment (recurrent) or is newly diagnosed. If newly diagnosed, the cancer must be metastatic,…
The main purpose of this study is to see how effective an investigational new drug (tipifarnib, the study drug) is to treat myelodysplastic syndrome (MDS). "Investigational" means that tipifarnib has not yet been approved by regulatory authorities such as the United States Food and Drug Administration (FDA) for use outside of clinical trials. You…
The purpose of this study, which involves research, is to determine if an experimental drug: acalabrutinib is safe and effective in the treatment of this patients with High Risk Chronic Lymphocytic Leukemia (CLL). Acalabrutinib is an investigational drug, which means that it is still being tested in people and has not been approved by government agencies in…
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.
The purpose of this research study is to find out if the study drug MBG453 in combination with other drugs (decitabine or azacitidine) already commonly used to treat MDS is safe and has beneficial effects in people who have some types of MDS like you (intermediate, high, or very high risk MDS). Subjects will be administered the study treatment as indicated…
We are doing this study to find out if an investigational drug called CPI-0610 can help with myelofibrosis. An investigational drug is a drug that is not approved for sale in the United States by the Food and Drug Administration (FDA). This study has two groups (arms) one group of patients will be treated with CPI-0610 alone (monotherapy arm) and the other…
Pevonedistat is currently being studied in humans with advanced solid tumors and acute myelogenous leukemia (AML). The current study is designed to evaluate pevonedistat in combination with azacitidine in patients with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and low-blast AML. This study will assess whether taking…
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…
The purpose of this research study is to obtain information on the safety and effectiveness of PBCAR20A to treat certain types of cancers. PBCAR20A is a new investigational treatment for certain types of cancer of the blood, such as leukemia and lymphoma. It is made from a type of blood cells known as T cells. The T cells in PBCAR20A came from healthy…
The purpose of this part of the study is to obtain further information about the safety and effectiveness(how well it works) of azacitidine plus birinapant (the investigational drug), in people with higher-risk myelodysplastic syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL). One of the standard drugs for these diseases is azacitidine which all…
The purpose of this study is to test the safety and effectiveness of avapritinib (also known as BLU-285) in individuals who have been diagnosed with aggressive systemic mastocytosis (ASM), Systemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell Leukemia (MCL). Avapritinib is an experimental drug means that is is not yet known if…
This study is for patients with melanoma, non-small cell lung cancer (NSCLC), or squamous cell cancer of the head and neck (SCCHN) that has a protein called B7-H3 on the tumor cells or blood vessels in thetumor. The purpose of this study is to test the safety, tolerability, and most effective dose ofMGA271 in combination with ipilimumab (also called Yervoy…
The purpose of this study is to determine how well a drug called rigosertib works on people with MDS and to study the safety of rigosertib when it is given to people with MDS. Rigosertib is currently being tested in other clinical research studies.
The purpose of the study is to see if a medicine called ASP2215 is both effective and safe as atreatment for AML patients with mutations in FLT3 gene identified by an investigational FLT3Mutation Assay companion diagnostic (a specific way to test your blood or bone marrow, which is used to see if you have a mutation of the FLT3 gene), compared to salvage…
The purpose of this research study is to determine the effectiveness (how well something works) and safety of an investigational compound called alvocidib when used in a combination chemotherapy therapy regimen in patients with acute myeloid leukemia (AML) who test positive for NOXA priming.
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…
The purpose of the study is to evaluate the efficacy and safety of imetelstat for the treatment of patients with myelodysplastic syndrome (MDS). All patients participating in Part 1 of this study will get 7.5 mg/kg of imetelstat as a 2-hour intravenous infusion once every 4 weeks. The dose of imetelstat may be increased, held or decreased depending on how…
The purpose of this study is to see if a medicine called ASP2215 is both safe and effective as atreatment for patients with Acute Myeloid Leukemia (AML). ASP2215 is an experimental drug that is being studied to treat AML. In this study, ASP2215 will be given in addition to a standard chemotherapy regimen that patients could be receiving even if they were…
This study is for patients with recurrent or metastatic Squamous Cell Carcinoma of the Head and Neck (SCCHN). This study will test the safety of an experimental anti-cancer drug (MEDI4736). MEDI4736 is an antibody (a protein produced by the defense system of the body immune system) that has been made in the laboratory. MEDI4736 boosts the ability of your…
The purpose of this study is to compare two different combinations of treatment plans to a standard transplant procedure in order to see whether one or both of them are better at reducing the occurrence and severity of chronic GVHD. The research portion of this study involves manipulation of your stem cell product by removing T-cells, which cause chronic…
The purpose of the study is to see if a combination of idelalisib and BI 836826 can help control patients with Chronic Lymphocytic Leukemia (CLL) without serious side effects.
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:…
You are being asked to take part in this study because you have been diagnosed with Myeloproliferative neoplasm (MPN) Associated Myelofibrosis (your disease) and have received ruxolitinib. The purpose of this study is to confirm how well this study treatment works to reduce your spleen volume, spleen size, what effect the study treatment has on you and…
The purpose of this study is to test the safety of DCLL9718S at different dose levels, alone or in combination with azacitidine. DCLL9718S is an experimental drug manufactured by Genentech that is designed to inhibit tumor growth. The drug is being tested in clinical trials and has not been approved by the U.S. Food and Drug Administration (FDA) and/or any…
The goal of this research study is to find the safest highest dose of SL-401 that can be given to patients with AML or BPDCN. This study will also look at how SL-401 stops or slows leukemia or BPDCN growth and how SL-401 enters and leaves the body. This study will also look at certain proteins in the blood and bone marrow and how the amounts of them might…
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…
You are being asked to take part in this study because you have acute myeloid leukemia (AML) that has come back after initial treatment (relapsed) with venetoclax combination therapy or because you did not get a complete response from your initial treatment (refractory) with venetoclax combination therapy. The purpose of this research study is to determine…
The purpose of this study is to determine if oral azacitidine (CC-486) is safe and effective as maintenance therapy in continuing the response patients had with their last acute myeloid leukemia (AML) treatment and improving the quality of that response. Currently, there is no standard treatment to maintain or improve patient response and patients are…
This study will see how the treatment you will receive for your chronic myeloid leukemia affects cardiovascular and metabolic risk factors. The treatments that are being studied are called BCR-ABL1 tyrosine kinase inhibitors (TKIs) (i.e., dasatinib, imatinib, nilotinib). Subjects who are eligible for the study will be starting treatment for chronic myeloid…
The purpose of this study is to determine the effectiveness and safety of study drug MEDI4736 monotherapy, tremelimumab monotherapy, and MEDI4736 and tremelimumab combination therapy in the treatment of patients with recurrent or metastatic PD-L1-negative squamous cell carcinoma of the head and neck who have progressed during or after treatment with…
The purpose of this study is to evaluate the efficacy (how well the study drug works) and safety of venetoclax in combination with azacitidine in subjects who have not been treated for their acute myeloid leukemia (AML). The tolerability (the degree to which evident adverse effects can be tolerated by the patient) of venetoclax will also be evaluated.
This is a clinical research study for subjects with Acute Myeloid Leukemia (AML) who have not received any treatment for their disease and who are over the age of 60. These patients will be treated with low-does cytarabine in combination with Lintuzumab-Ac225. Low dose cytarabine has been investigated in elderly AML patients for at least two decades and is…
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.
The main purposes of this study are to: 1) understand the safety of atezolizumab given in combination with Hu5F9-G4 and 2) understand the way the body processes atezolizumab and Hu5F9-G4 (pharmacokinetics). This study will also be testing if the combination therapy is effective at treating relapsed or refractory acute myeloid leukemia (R/R AML).…
The purpose of this study is to measure the effectiveness and safety of an investigational drug called "alovicidib" in combination with cytarabine and daunorubicin in the treatment of AML. Investigational means that this drug has not been approved by the FDA. Cytarabine and daunorubicin are both standard of care treatments for patients with AML.…
The purpose of this study is to test the safety and effectiveness of an experimental medication, FT-2102, in the treatment of acute myeloid leukemia or myelodysplastic syndrome. Patients will receive either FT-2102 alone or in combination with cytarabine or azacitidine, an approved drug commonly used in the treatment of AML and MDS. Subjects will remain on…
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.
The purpose of this study, which involves research, is to determine if giving an experimental drug called acalabrutinib in combination with venetoclax with and without obinutuzumab is safe and effective for treating people with CLL compared to a combination of bendamustine and rituximab (BR) or a combination of Fludarabine, Cyclophosphamide and Rituximab (…
The purpose of this study is to evaluate the safety, and effectiveness of ABT-199 in combination with decitabine and ABT-199 in combination with azacitidine in subjects who have not been treated for their AML. In addition, substances (biomarkers) found in the blood and bone marrow that may indicate the effects or progress of the leukemia and the activity of…
The purpose of this study is to provide samples for biomarker testing for the different MPD-RC treatment studies. As part of the treatment study you currently participating in, this is an additional research study in which you we will ask for you to donate blood and/or bone marrow samples, two nail clippings, and buccal (cheek) swab. In order to…
This study is for children and teens who have been diagnosed with Acute Myeloid Leukemia (AML). AML is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone…
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in young adults with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) that has either come back or has not responded to the standard therapy. Pevonedistat works by blocking some of the enzymes that are needed for cell growth.