Can a targeted drug boost chemo for hard-to-treat blood cancers?
NCT ID NCT03289910
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase II trial tested whether adding veliparib (a targeted drug) to standard chemotherapy (topotecan and carboplatin) helps people with advanced myeloproliferative disorders, acute myeloid leukemia, or chronic myelomonocytic leukemia. The study enrolled 25 adults whose cancer had spread or returned. Researchers measured how many patients achieved a complete or partial response, and tracked side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- veliparib (a PARP inhibitor) combined with topotecan and carboplatin (chemotherapy drugs)
- What this could lead to
- If it works, this could point toward a more effective chemotherapy combination for advanced blood cancers that are hard to treat.
- What could go wrong
- This is a small, early-phase (phase II) trial with only 25 participants. The added drug may not improve outcomes and could increase side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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25 people
The number who actually took part.
- Started
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Sep 2018
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * PRE-REGISTRATION ELIGIBILITY CRITERIA * Newly diagnosed acute myeloid leukemia (AML) associated with antecedent myeloproliferative disorder (polycythemia vera, essential thrombocythemia, myelofibrosis, atypical chronic myeloid leukemia, chronic myelomonocytic leukemia and related undifferentiated myeloproliferative/myelodysplastic disorders) * Relapsed/refractory AML associated with antecedent myeloproliferative disorder (polycythemia vera, essential thrombocythemia, myelofibrosis, atypical chronic myeloid leukemia, chronic myelomonocytic leukemia and related undifferentiated myeloproliferative/myelodysplastic disorders) who have received two or fewer prior induction chemotherapy courses * Accelerated phase myeloproliferative disorders per Zeider et al with two or fewer prior therapies * For aggressive phase myeloproliferative disorders (MPD) (polycythemia vera, essential thrombocythemia, Philadelphia \[Ph\]-negative chronic myelogenous leukemia), one or more of the following criteria must be met: marrow blasts \> 5%, peripheral blood blasts plus progranulocytes \> 10%, new onset or increasing myelofibrosis, new onset or \> 25% increase in hepatomegaly or splenomegaly, new onset constitutional symptoms (fever, weight loss, splenic pain, bone pain). Zeider et al * For chronic myelomonocytic leukemia (CMML), the following criteria must be met: 5-19% bone marrow blasts (aggressive) or \>= 20% marrow blasts (transformation) * Bone marrow and/or peripheral blood specimens will be submitted for correlative studies; patients with a dry tap will still be eligible * RANDOMIZATION ELIGIBILITY CRITERIA * Bone marrow aspirate and/or peripheral blood specimens were submitted to the central lab and site has confirmation by the local institution that the patient meets one of the criteria specified above * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 or Karnofsky \>= 60% * Total bilirubin less than 2.0 mg/dL unless due to Gilbert's syndrome, then less than 5.0 mg/dL * Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) less than 5 x institutional upper limit of normal * Creatinine clearance glomerular filtration rate (GFR) greater than 30 ml/min per modified Cockcroft-Gault formula * Interval of greater than 4 weeks since allogeneic blood or marrow transplantation (BMT) if performed; and absence of active graft versus host disease (GVHD) * The effects of veliparib on the developing human fetus are unknown; for this reason and because PARP inhibiting agents as well as topoisomerase inhibitors and platinating agents are known to be teratogenic, women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 6 months following the last dose of study drug; should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately; men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of veliparib administration * Ability to understand and the willingness to sign a written informed consent document Exclusion Criteria: * Patients who have had chemotherapy or radiotherapy within 4 weeks prior to entering the study with the exception of hydroxyurea for cytoreduction; therapy with tyrosine kinase inhibitors (TKIs) directed against JAK2, BCR-ABL or FLT3 will be allowed to be continued until 24 hours prior to start of therapy on trial * Patients with active uncontrolled infection; antibiotic therapy for fevers, and continuation of treatment of prior infection are allowed * Patients who have active central nervous system (CNS) disease are excluded; patients with known active CNS leukemia should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events * Patients who are receiving any other investigational agents; patients who have completed therapy with an investigational agent should be off this therapy for at least 5 half-lives or two weeks, whichever is shorter * History of allergic reactions attributed to compounds of similar chemical or biologic composition to veliparib, topotecan or carboplatin * Uncontrolled intercurrent illness including, but not limited to, active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements * Pregnant women are excluded from this study because veliparib is PARP inhibiting agent with the potential for teratogenic or abortifacient effects; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with veliparib, breastfeeding should be discontinued if the mother is treated with veliparib; these potential risks may also apply to topotecan and carboplatin used in this study * Human immunodeficiency virus (HIV)-patients positive patients are not excluded if they have CD4+ cells \>= 250/mm\^3 and negligible viral load and are on a stable combination antiretroviral therapy * History of uncontrolled seizure disorder, including focal or generalized seizure within the past year
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287, United States
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Los Angeles General Medical Center
Los Angeles, California, 90033, United States
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Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903, United States
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UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599, United States
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USC / Norris Comprehensive Cancer Center
Los Angeles, California, 90033, United States
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USC Norris Oncology/Hematology-Newport Beach
Newport Beach, California, 92663, United States
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