Droxia

This study is being conducted to evaluate the safety and tolerability of INCA035784 in participants with myeloproliferative neoplasms.

The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia. The Hemanext ONE device was cleared through the De Novo process in September 2023.

This phase II trial compares the effect of ASTX727 in combination with iadademstat to ASTX727 alone in treating patients with accelerated or blast phase Philadelphia chromosome negative myeloproliferative neoplasms (MPNs). ASTX727 is a combination of two drugs, cedazuridine and decitabine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Iadademstat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with iadademstat may be more effective than ASTX727 alone in treating patients with accelerated or blast phase Philadelphia chromosome negative MPNs.

Low-dose MTX is a widely used, inexpensive, and safe therapy used for decades and is well tolerated by patients with rheumatologic diseases. Recently, it was identified as a type 2 JAK inhibitor. If MTX proves to be safe and tolerable with a signal of clinical activity, this could have a significant benefit to patients with MPNs. Beyond the potential benefit of adding a type 2 JAK inhibitor to current therapy, this could signal the need to study MTX in MPNs further as a monotherapy. Discovering MTX as safe and clinically effective in MPNs could be profound on both a public health and global health scale for patients who are uninsured and cannot afford more expensive novel JAK inhibitors, or for those in countries where JAK inhibitors are not available. Accordingly, the research team deems it reasonable and prudent to assess the safety and efficacy of MTX in addition to current therapy for patients with MPN. The research team will evaluate patients for spleen responses, symptom responses, and cytologic responses. Correlative data will evaluate pharmacokinetic and disease modifying activity of MTX in MPNs to inform future clinical trials.

The primary purpose of the study is to transition participants into an extension study to collect long-term safety and efficacy data. The study will include participants who are safely tolerating bomedemstat, receiving clinical benefit from its use in estimation of the investigator, and have shown the following criteria: * Participants from the IMG-7289-202/MK-3543-005 (NCT05223920) study must have received at least 6 months of treatment with bomedemstat; * Essential thrombocythemia (ET) and polycythemia vera (PV) participants from studies other than IMG-7289-202/MK-3543-005 must have achieved confirmed hematologic remission. No hypothesis testing will be conducted in this study.

This is a prospective phase I dose-escalation study, with the primary objective to access the MTD and find the RP2D of talazoparib, given in combination with standard of care dosing of pacritinib.

This trial tests a new drug, JNJ-88549968, to find the safest and most effective dose for patients. It aims to determine the best dosing schedule and ensure the drug's safety.

This is a study evaluating the safety and efficacy of bomedemstat (MK-3543) compared with the best available therapy (BAT) in participants with essential thrombocythemia (ET) who have an inadequate response to or are intolerant of hydroxyurea. The primary study hypothesis is that bomedemstat is superior to the best available therapy with respect to durable clinicohematologic response (DCHR).

This trial is testing a new drug called INCA033989 in patients with a type of blood cancer. The goal is to find the safest and most effective dose by checking for side effects and how well the drug works.

This trial is testing a new drug called INCA033989, alone or with ruxolitinib, in patients with a type of blood cancer. The goal is to find the safest dose and see how well patients can tolerate it. Ruxolitinib is a medication used to treat myelofibrosis.

This trial is testing a new medication called Ropeginterferon alfa-2b-njft (P1101) for adults with Essential Thrombocythemia, a blood disorder. The medication helps the body control blood cell production to prevent the disease from worsening. Interferons have been studied in similar conditions for over 30 years, but earlier versions had safety and tolerability issues.

Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 36,000 children in the United States, approximately 90% of whom are Black. The disease is characterized by recurrent, severe pain crises which result in high rates of emergency department visits and hospitalizations, and decreased quality of life. The National Heart, Lung and Blood Institute, as well as the American Society of Hematology, have endorsed pain management guidelines regarding the timeliness of care for children presenting with these acute pain crises. These evidence-based guidelines are infrequently followed, resulting in increased pain and hospitalizations. In additional to other barriers to following the guideline, structural racism has been proposed as a significant contributor and the New England Journal of Medicine recently called for the institution of SCD-specific pain management protocols to combat structural racism and reduce time to opioid administration. The investigators' long-term goal is to improve the care and health outcomes of children with acute painful vaso-occlusive crisis treated in the emergency department. The overall aim of the investigators is to test a care pathway using multifaceted implementation strategies to increase guideline adherent care for children in the emergency department with acute painful vaso-occlusive crisis.