CLINICAL TRIAL

Veliparib for Myeloproliferative Disorders

1 Prior Treatment
Refractory
Relapsed
Waitlist Available · 18+ · All Sexes · Baltimore, MD

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

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About the trial for Myeloproliferative Disorders

Eligible Conditions
Thrombocythemia, Essential · Leukemia, Myeloid · Hematopoietic and Lymphoid Cell Neoplasm · Adult Acute Myelomonocytic Leukemia · Adult Acute Myeloid Leukemia With Maturation · Chronic Myelomonocytic Leukemia (CMML) · Leukemia, Megakaryoblastic, Acute · Myeloproliferative Disorders · Leukemia · Precursor Cell Lymphoblastic Leukemia-Lymphoma · Thrombocytosis · Adult Erythroleukemia · Recurrent Adult Acute Lymphoblastic Leukemia (ALL) · De Novo Myelodysplastic Syndromes · Adult Acute Myeloid Leukemia With Minimal Differentiation · Adult Acute Myeloid Leukemia With t(9;11)(p21.3;q23.3); MLLT3-MLL · Adult Acute Megakaryoblastic Leukemia (M7) · Philadelphia Chromosome Negative, BCR-ABL1 Positive Chronic Myelogenous Leukemia · Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 · Preleukemia · Myelodysplastic Syndromes (MDS) · Adult Acute Monocytic Leukemia · Leukemia, Myelomonocytic, Juvenile · Leukemia, Myelomonocytic, Chronic · Adult Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1 · Adult Pure Erythroid Leukemia · Recurrent Acute Myeloid Leukemia, Adult · Leukemia, Myeloid, Acute · Adult Acute Monoblastic Leukemia · Leukemia, Myelomonocytic, Acute · Philadelphia Chromosome · Adult Acute Myeloid Leukemia Without Maturation · Leukemia, Myelogenous, Chronic, BCR-ABL Positive · Essential Thrombocythemia (ET) · Syndrome · Secondary Myelodysplastic Syndromes · Myelodysplastic Syndromes · Leukemia, Erythroblastic, Acute · Polycythemia · Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 · Polycythemia Vera · Leukemia, Monocytic, Acute

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.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Laboratory Biomarker Analysis
OTHER
Carboplatin
DRUG
Veliparib
DRUG
Topotecan Hydrochloride
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Carboplatin
FDA approved
Veliparib
Not yet FDA approved
Topotecan
FDA approved

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.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Pathologically confirmed diagnosis of 1 of aggressive MPD or AML out of MPD
Marrow blasts > 5%
Peripheral blood blasts plus progranulocytes > 10%
New onset or increasing myelofibrosis OR;
New onset or > 25% increase in hepatomegaly or splenomegaly
New onset constitutional symptoms (i.e., fever, weight loss, splenic pain, or bone pain)
Patients who failed primary induction therapy or relapsed after achieving complete remission are eligible
No active CNS leukemia; patients with a history of CNS disease must be stable for > 3 months after treatment and off steroid treatment prior to study enrollment
5-19% bone marrow blasts (aggressive)
At least 20% marrow blasts (transformation)
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 3 years
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 3 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 3 years.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

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.

Maximum tolerated dose of veliparib, determined as the highest dose level where 0/3 or 1/6 experience DLT, measured according to NCI-CTCAE 4.0
UP TO 63 DAYS
UP TO 63 DAYS
Pharmacokinetics and pharmacodynamics of veliparib
DAY 1 AT PRE-TREATMENT, .25, .5, 1, 2, 4, 6, AND 8 HOURS AFTER VELIPARIB AND DAY 4 AT PRE-VELIPARIB, .25, .5, 1, 2, 4, 6, AND 8 HOURS AFTER THE FIRST DOSE OF VELIPARIB
Relevant individual PK parameters will be estimated using non-compartmental PK methods. PK parameters will be compared when administered alone or in combination by a paired student's t-test. Comparison of PK parameters among dose levels will be performed using non-parametric statistical methods for K-independent samples. Associations between exposure parameters (Cmax and AUC) and pharmacodynamic endpoints (cellular PAR levels, mutation and/or altered expression of selected DNA repair genes) will be assessed using the appropriate non-parametric statistical tests.
DAY 1 AT PRE-TREATMENT, .25, .5, 1, 2, 4, 6, AND 8 HOURS AFTER VELIPARIB AND DAY 4 AT PRE-VELIPARIB, .25, .5, 1, 2, 4, 6, AND 8 HOURS AFTER THE FIRST DOSE OF VELIPARIB
Clinical response (CR, CRi, PR)
UP TO 3 YEARS
UP TO 3 YEARS

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer
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