Veliparib for Myeloproliferative Disorders
This study is evaluating the side effects and best dose of veliparib when given together with topotecan hydrochloride and carboplatin in treating patients with relapsed or refractory acute leukemia, high-risk myelodys
See full descriptionAbout the trial for Myeloproliferative Disorders
Treatment Groups
This trial involves 2 different treatments. Veliparib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.
About The Treatment
Eligibility
This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Myeloproliferative Disorders or one of the other 41 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether Veliparib will improve 2 primary outcomes and 1 secondary outcome in patients with Myeloproliferative Disorders. Measurement will happen over the course of Up to 63 days.
Patient Q & A Section
Can myeloproliferative disorders be cured?
Most cases of myeloproliferative disorders are not curable with chemotherapy. However, in the subset of patients with very early disease, a small number of the cases appear to be curable. The key to eliminating myeloproliferative disorders is early detection of the disease and effective treatment of the underlying cause.
What are the signs of myeloproliferative disorders?
Any person with these signs will either have one or more of the other disorders diagnosed in the medical history or will be assessed for them by the physician. The first signs can include changes in blood volume, jaundice, and enlargement of the liver and/or spleen. Later signs include a shiny or bright red, swollen, or tender skin surface on the head and neck, the trunk, or joints. Other signs of these disorders include bone tenderness on palpation of the long bones. Other signs of blood disorders in the patient are blood in the urine (blood in the urine without fever). These may include blood in the urine with a fever, pain with urination, or urinary frequency with pain.
How many people get myeloproliferative disorders a year in the United States?
The number of new diagnoses of myeloproliferative disorders in the US will range between 135,000 and 290,000 annually, an increase of about two- to fourfold from 1992 levels, depending on diagnostic criteria. The most significant decrease in rates is expected among smokers.
What is myeloproliferative disorders?
Myeloproliferative disorders are a group of medical conditions that includes many myeloproliferative or leukemmia cells, which are fast dividing. The most common myeloproliferative disorders are myeloproliferative neoplasms, including myeloproliferative disease, and polycythemia vera and essential thrombocytocytoma. However, several other disorders such as promyelocytic lymphoproliferative disease and idiopathic myelofibrosis may also be included to the myeloproliferative disorders group.
What causes myeloproliferative disorders?
Myeloproliferative disorders are caused by various genetic and environmental factors and result in hyperactivation of cellular proliferation in all hematopoietic cells. These disorders therefore present an excellent opportunity for the development of new therapeutic agents against myeloproliferative disorders, and they provide an attractive model system for studying the cellular and molecular mechanisms by which genetic or environmental factors cause hyperactivation of proliferation.
What are common treatments for myeloproliferative disorders?
Treatments used in patients with myeloid neoplasms may lead to serious life-threatening toxicity. In our study, there was no specific method of categorizing treatments. A more precise way of categorizing treatments in this regard may be useful.
Is veliparib safe for people?
Veliparib in combination with the myeloid-targeting agents dasatinib and pomalidomide and the lymphoid-targeting agents rituximab and gemtuzumab ozogamicin in people with AML is active while being well tolerated.
What are the common side effects of veliparib?
Veliparib was well tolerated in subjects of both sexes. The most common side effects to receive an FDA-conditional label for veliparib are diarrhea, vomiting, headache, and fatigue.
Who should consider clinical trials for myeloproliferative disorders?
Clinical trials are a useful tool to learn more about the efficacy of new antifolate agents in the treatment of MPD. Clinical trials are also an important part of basic investigations, since they provide information on the safety of a new chemotherapeutic agent. In summary, clinical trials help doctors choose the most appropriate treatment for patients with MPD and are a necessary requirement for the success of basic and translational investigations.
Have there been any new discoveries for treating myeloproliferative disorders?
The therapeutic choices of individual patients with MPD may vary based on their MPD subtype. For example, some patients with PV-CML may be candidates to treat with interferon or imatinib. However, prospective studies will be needed to guide clinical practice.
How does veliparib work?
Veliparib is a novel PARP inhibitor designed to target mutant BRCA or other mutant, dysfunctional p53 pathway in a broad spectrum of cancer types. Veliparib significantly reduces tumor proliferation in a number of preclinical models of cancer cell lines, including BRCA wild type and BRCA mutant.
Does myeloproliferative disorders run in families?
We established that MPD does not run in families and there is no evidence for the role of genes in this setting. This suggests that inherited susceptibility, rather than genetic susceptibility, is the usual mode of disease in non-syndromic MPD.