ClinicalTrials.gg
Active, not recruitingNCT03289910Updated Jun 23, 2026Results posted

Topotecan Hydrochloride and Carboplatin With or Without Veliparib in Treating Advanced Myeloproliferative Disorders and Acute Myeloid Leukemia or Chronic Myelomonocytic Leukemia

A Phase 2 interventional study of Carboplatin and Topotecan in Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome and Atypical Chronic Myeloid Leukemia, sponsored by National Cancer Institute (NCI). Active, not recruiting at 6 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-06-23.

Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
25
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

This phase II trial studies how well topotecan hydrochloride and carboplatin with or without veliparib work in treating patients with myeloproliferative disorders that have spread to other places in the body and usually cannot be cured or controlled with treatment (advanced), and acute myeloid leukemia or chronic myelomonocytic leukemia. Drugs used in chemotherapy, such as topotecan hydrochloride and carboplatin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Veliparib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving topotecan hydrochloride, carboplatin, and veliparib may work better in treating patients with myeloproliferative disorders and acute myeloid leukemia or chronic myelomonocytic leukemia compared to topotecan hydrochloride and carboplatin alone.

Read the detailed description

PRIMARY OBJECTIVE:

I. To estimate and compare the complete response/complete response with incomplete recovery (CR/CRi) rate of induction therapy with topotecan hydrochloride (topotecan)/carboplatin (T/C) with or without veliparib (V) in myeloproliferative disorder associated leukemias and chronic myelomonocytic leukemia (CMML).

SECONDARY OBJECTIVES:

I. To evaluate and compare the toxicities of T/C/V versus (vs.) T/C. II. To compare the 2-year disease-free survival (DFS) and overall survival (OS) in response to T/C/V vs. T/C.

III. To detect and compare the presence of minimal residual disease (MRD) remaining after T/C/V vs. T/C.

IV. Evaluate predictive biomarkers of response via assessment of pretreatment impaired homologous recombination via assessment of:

IVa. Next generation sequencing (NGS) panel for genes mutated in myeloid malignancies done as standard of care per institution.

IVb. Functional impairment of deoxyribonucleic acid (DNA) damage response via assessment of pretreatment samples for radiation-induced RAD51 foci.

IVc. Topotecan-induced stabilization of topoisomerase I-DNA covalent complexes, which has recently been observed to be a critical predictor of response to combination of a topoisomerase I poison and PARP inhibitor in xenografts.

V. To evaluate veliparib exposure and contribution to response (efficacy and toxicity).

OUTLINE: Patients are randomized to 1 of 2 arms.

ARM A: Patients receive veliparib orally (PO) twice daily (BID) on days 1-21 and topotecan hydrochloride intravenously (IV) continuously over 24 hours and carboplatin IV continuously over 24 hours on days 3-7. Treatment repeats every 28-63 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.

ARM B: Patients receive topotecan hydrochloride IV continuously over 24 hours and carboplatin IV continuously over 24 hours on days 1-5. Treatment repeats every 28-63 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for a minimum of 30 days, or longer.

02

Conditions studied

  • Acute Myeloid Leukemia
  • Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
  • Atypical Chronic Myeloid Leukemia
  • Chronic Myelomonocytic Leukemia
  • Essential Thrombocythemia
  • Myelodysplastic/Myeloproliferative Neoplasm
  • Myelofibrosis
  • Polycythemia Vera
  • Recurrent Acute Myeloid Leukemia
  • Refractory Acute Myeloid Leukemia
03

In context

Leukemia, Myeloid, Acute

3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.

This study's enrollment of 25 is below the median of 40 across 3,005 interventional studies indexed under Leukemia, Myeloid, Acute.

Browse Leukemia, Myeloid, Acute studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.

Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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)
  • 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
  • (ECOG) performance status =\< 2 or >= 60%
  • Total less than 2.0 mg/dL unless due to Gilbert's syndrome, then less than 5.0 mg/dL
  • (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) less than 5 x institutional
  • (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 (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 must agree to use adequate (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 document

Exclusion criteria

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 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
  • (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
Terms in these criteria, in plain language(14)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

ECOG performance status

A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.

0
Fully active, no restrictions.
1
Limited in strenuous activity, but walking and able to do light work, such as housework or office work.
2
Walking and able to look after yourself, but unable to work; up and about more than half of waking hours.
3
Able to look after yourself only in part; in bed or a chair more than half of waking hours.
4
Unable to look after yourself at all; in bed or a chair all day.

Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.

Karnofsky performance status (KPS)

A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.

Studies often ask for 60 or 70 and above. Your doctor gives the score.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

GVHD (graft-versus-host disease)

After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Single (Participant)
Enrollment
25 participants (actual)

Study arms

  • Experimental
    Arm A (veliparib, topotecan hydrochloride, carboplatin)

    Patients receive veliparib PO BID on days 1-21 and topotecan hydrochloride IV continuously over 24 hours and carboplatin IV continuously over 24 hours on days 3-7. Treatment repeats every 28-63 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.

    Drug: Carboplatin · Drug: Topotecan · Drug: Topotecan Hydrochloride · Drug: Veliparib

  • Active comparator
    Arm B (topotecan hydrochloride, carboplatin)

    Patients receive topotecan hydrochloride IV continuously over 24 hours and carboplatin IV continuously over 24 hours on days 1-5. Treatment repeats every 28-63 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.

    Drug: Carboplatin · Drug: Topotecan · Drug: Topotecan Hydrochloride

Interventions

  • Given IV

    Also known as: Blastocarb, Carboplat, Carboplatin Hexal, Carboplatino, Carboplatinum, Carbosin, Carbosol, Carbotec, CBDCA, Displata, Ercar, JM-8, JM8, Nealorin, Novoplatinum, Paraplatin, Paraplatin AQ, Paraplatine, Platinwas, Ribocarbo

  • Given IV

    Also known as: Hycamptamine, Topotecan Lactone

  • Given IV

    Also known as: Evotopin, Hycamptamine, Hycamtin, Nogitecan Hydrochloride, Potactasol, SKF S 104864 A, SKF S-104864-A, SKF S104864A, Topotec, Topotecan HCl, topotecan hydrochloride (oral)

  • Given PO

    Also known as: ABT 888, ABT-888, ABT888, PARP-1 inhibitor ABT-888

06

What researchers measure

Primary outcomes

  1. Number of Participants With a Response

    Based on published standards for acute leukemias, Complete Response (CR) means less than 5% leukemic blasts in the bone marrow, no blasts in the blood, no longer presence of cytogenetic abnormalities, no longer presence of extramedullary disease, with or without absolute neutrophil count or platelet count recovery; Partial Remission (PR) includes the criteria for Complete Remission except there are 5-25% leukemic blasts in the bone marrow and there is absolute neutrophil count and platelet count recovery; Hematologic Improvement (HI) means the disease has not gotten worse and there is at least a 20% decrease in the leukemic blasts in the bone marrow and/or a decrease in leukemia symptoms. Response = CR, PR, or HI.

    Time frame: Up to 7 months

Secondary outcomes

  1. The Highest Grade Adverse Event Experienced

    Adverse events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.

    Time frame: Up to 7 months

  2. Number of Participants Without Disease at Study Completion

    Study completion was either death or completion of all protocol-specified activities, whichever came first. Participants who came off study due to disease-related death were counted as having disease.

    Time frame: Up to 7 months

  3. Duration of Disease-free Survival

    Disease-free survival is defined as participants who are still alive and without disease at study completion. Study completion was either death or completion of all protocol-specified activities, whichever came first.

    Time frame: Up to 7 months

  4. Number of Participants Still Alive at Study Completion

    Study completion was either death or completion of all protocol-specified activities, whichever came first.

    Time frame: Up to 7 months

  5. Duration of Overall Survival at the Time of Study Completion

    Study completion was either death or completion of all protocol-specified activities, whichever came first. Duration was measured from the date of registration to the participant's study completion date.

    Time frame: Up to 7 months

  6. Number of Participants With Minimal Residual Disease (MRD) After Treatment

    Minimal residual disease (MRD) refers to a small number of leukemic cells that remain after treatment.

    Time frame: Up to 7 months

  7. Distribution of Mutations in Deoxyribonucleic Acid (DNA) Repair Defects Via Assessment in Leukemia Mutation Panel

    Will be summarized using descriptive statistics. The association response will be described with appropriate tests for continuously measured biomarkers (t tests, Wilcoxon rank sum tests) and categorical biomarkers (Fisher's exact test). Descriptive analyses will be performed for the whole cohort and also separately for Arms A and B. Differential treatment outcomes for patient subgroups may be explored using appropriate tests for interactions.

    Time frame: Baseline

  8. Frequency of Patients With Functional Impairment of DNA Damage Response Via Assessment With RAD51 Assay

    Will be reported with exact binomial 95% confidence intervals. The association response will be described with appropriate tests for continuously measured biomarkers (t tests, Wilcoxon rank sum tests) and categorical biomarkers (Fisher's exact test). Descriptive analyses will be performed for the whole cohort and also separately for Arms A and B. Differential treatment outcomes for patient subgroups may be explored using appropriate tests for interactions.

    Time frame: Baseline

  9. Topotecan-induced Stabilization of Topoisomerase I-DNA Covalent Complexes

    Topotecan-induced stabilization of topoisomerase I-DNA covalent complexes from peripheral blood

    Time frame: Up to 7 months

  10. Pharmacokinetic Sampling Studies Measured Using a Validated Liquid Chromatography/Tandem Mass Spectrometric Method in Plasma and Bone Marrow

    Plasma trough levels will be obtained weekly through the first cycle to provide a steady-state assessment. Steady-state plasma concentrations will be calculated for each patient. Exploratory correlative studies between veliparib exposure (plasma and bone marrow) with pharmacodynamic (biological endpoints, toxicity and efficacy) will be analyzed using nonparametric statistics. Significance for comparisons will be at the p \< 0.05 level.

    Time frame: Pre-treatment, day 1, day 8, day 14, day 15, and day 22 (approximately 24 hours post last dose)

07

Results

Posted Apr 2, 2024

Participant flow

Participant flow — Overall Study
MilestoneArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
Started169
Completed169
Not completed00

Outcome measures

PrimaryNumber of Participants With a Response

Based on published standards for acute leukemias, Complete Response (CR) means less than 5% leukemic blasts in the bone marrow, no blasts in the blood, no longer presence of cytogenetic abnormalities, no longer presence of extramedullary disease, with or without absolute neutrophil count or platelet count recovery; Partial Remission (PR) includes the criteria for Complete Remission except there are 5-25% leukemic blasts in the bone marrow and there is absolute neutrophil count and platelet count recovery; Hematologic Improvement (HI) means the disease has not gotten worse and there is at least a 20% decrease in the leukemic blasts in the bone marrow and/or a decrease in leukemia symptoms. Response = CR, PR, or HI.

Time frame:
Up to 7 months
Reported as:
Count of participants · Participants
Number of Participants With a Response
ParticipantsArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
Participants with a response54
Participants who did not have a response115
SecondaryThe Highest Grade Adverse Event Experienced

Adverse events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.

Time frame:
Up to 7 months
Reported as:
Count of participants · Participants
The Highest Grade Adverse Event Experienced
ParticipantsArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
Grade 5 Adverse Events01
Grade 4 Adverse Events148
Grade 3 Adverse Events20
SecondaryNumber of Participants Without Disease at Study Completion

Study completion was either death or completion of all protocol-specified activities, whichever came first. Participants who came off study due to disease-related death were counted as having disease.

Time frame:
Up to 7 months
Reported as:
Count of participants · Participants
Number of Participants Without Disease at Study Completion
ParticipantsArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
Number of Participants Without Disease at Study Completion33
SecondaryDuration of Disease-free Survival

Disease-free survival is defined as participants who are still alive and without disease at study completion. Study completion was either death or completion of all protocol-specified activities, whichever came first.

Time frame:
Up to 7 months
Reported as:
Median · days
Duration of Disease-free Survival
daysArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
Duration of Disease-free Survival120 (37 to 137)89 (27 to 137)
SecondaryNumber of Participants Still Alive at Study Completion

Study completion was either death or completion of all protocol-specified activities, whichever came first.

Time frame:
Up to 7 months
Reported as:
Count of participants · Participants
Number of Participants Still Alive at Study Completion
ParticipantsArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
Number of Participants Still Alive at Study Completion55
SecondaryDuration of Overall Survival at the Time of Study Completion

Study completion was either death or completion of all protocol-specified activities, whichever came first. Duration was measured from the date of registration to the participant's study completion date.

Time frame:
Up to 7 months
Reported as:
Median · Days
Duration of Overall Survival at the Time of Study Completion
DaysArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
Duration of Overall Survival at the Time of Study Completion83 (74 to 295)123 (82 to 295)
SecondaryNumber of Participants With Minimal Residual Disease (MRD) After Treatment

Minimal residual disease (MRD) refers to a small number of leukemic cells that remain after treatment.

Time frame:
Up to 7 months

No measurements were reported for this outcome.

SecondaryDistribution of Mutations in Deoxyribonucleic Acid (DNA) Repair Defects Via Assessment in Leukemia Mutation Panel

Will be summarized using descriptive statistics. The association response will be described with appropriate tests for continuously measured biomarkers (t tests, Wilcoxon rank sum tests) and categorical biomarkers (Fisher's exact test). Descriptive analyses will be performed for the whole cohort and also separately for Arms A and B. Differential treatment outcomes for patient subgroups may be explored using appropriate tests for interactions.

Time frame:
Baseline

No measurements were reported for this outcome.

SecondaryFrequency of Patients With Functional Impairment of DNA Damage Response Via Assessment With RAD51 Assay

Will be reported with exact binomial 95% confidence intervals. The association response will be described with appropriate tests for continuously measured biomarkers (t tests, Wilcoxon rank sum tests) and categorical biomarkers (Fisher's exact test). Descriptive analyses will be performed for the whole cohort and also separately for Arms A and B. Differential treatment outcomes for patient subgroups may be explored using appropriate tests for interactions.

Time frame:
Baseline

No measurements were reported for this outcome.

SecondaryTopotecan-induced Stabilization of Topoisomerase I-DNA Covalent Complexes

Topotecan-induced stabilization of topoisomerase I-DNA covalent complexes from peripheral blood

Time frame:
Up to 7 months

No measurements were reported for this outcome.

SecondaryPharmacokinetic Sampling Studies Measured Using a Validated Liquid Chromatography/Tandem Mass Spectrometric Method in Plasma and Bone Marrow

Plasma trough levels will be obtained weekly through the first cycle to provide a steady-state assessment. Steady-state plasma concentrations will be calculated for each patient. Exploratory correlative studies between veliparib exposure (plasma and bone marrow) with pharmacodynamic (biological endpoints, toxicity and efficacy) will be analyzed using nonparametric statistics. Significance for comparisons will be at the p \< 0.05 level.

Time frame:
Pre-treatment, day 1, day 8, day 14, day 15, and day 22 (approximately 24 hours post last dose)

Results for this outcome have not been posted.

Adverse events

Collected over Up 7 months. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Arm A (Veliparib, Topotecan Hydrochloride, Carboplatin)11/16 (68.8%)4/16 (25%)16/16 (100%)
Arm B (Topotecan Hydrochloride, Carboplatin)4/9 (44.4%)4/9 (44.4%)9/9 (100%)
Most frequent serious events
Showing 10 of 14
Most frequent serious events
EventArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
SepsisInfections and infestations2/161/9
Febrile neutropeniaBlood and lymphatic system disorders0/161/9
FeverGeneral disorders0/161/9
Skin infectionInfections and infestations0/161/9
White blood cell count increasedInvestigations0/161/9
HypoxiaRespiratory, thoracic and mediastinal disorders0/161/9
HypotensionVascular disorders0/161/9
Atrial fibrillationCardiac disorders1/160/9
Lung infectionInfections and infestations1/160/9
SinusitisInfections and infestations1/160/9
Most frequent other events
Showing 10 of 204
Most frequent other events
EventArm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)
AnemiaBlood and lymphatic system disorders14/169/9
DiarrheaGastrointestinal disorders13/169/9
Lymphocyte count decreasedInvestigations16/168/9
White blood cell decreasedInvestigations16/169/9
Platelet count decreasedInvestigations15/168/9
HypoalbuminemiaMetabolism and nutrition disorders15/167/9
HypokalemiaMetabolism and nutrition disorders15/165/9
Neutrophil count decreasedInvestigations14/167/9
NauseaGastrointestinal disorders10/167/9
FatigueGeneral disorders8/167/9

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Arm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)Total
<=18 years000
Between 18 and 65 years8412
>=65 years8513
Age, Continuous
Age, Continuous(years)Arm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)Total
Median65 (33 to 75)69 (46 to 74)66 (33 to 75)
Sex: Female, Male
Sex: Female, Male(Participants)Arm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)Total
Female415
Male12820
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Arm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)Total
Hispanic or Latino000
Not Hispanic or Latino16824
Unknown or Not Reported011
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Arm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)Total
American Indian or Alaska Native000
Asian224
Native Hawaiian or Other Pacific Islander000
Black or African American404
White10717
More than one race000
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(participants)Arm A (Veliparib, Topotecan Hydrochloride, Carboplatin)Arm B (Topotecan Hydrochloride, Carboplatin)Total
United States16925
08

Study locations

6 sites
09

References and documents

Publications

  • Richardson DR, Green SD, Foster MC, Zeidner JF. Secondary AML Emerging After Therapy with Hypomethylating Agents: Outcomes, Prognostic Factors, and Treatment Options. Curr Hematol Malig Rep. 2021 Feb;16(1):97-111. doi: 10.1007/s11899-021-00608-6. Epub 2021 Feb 20. PubMed 33609248 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Sep 2018 to primary completion in May 2023, 56 months.

Results were first posted in Apr 2024, 10 months after primary completion.

Of 385 non-industry phase 2 interventional studies under Leukemia, Myeloid, Acute, half reached primary completion within 67 months of starting (the median), counting those still running; a quarter within 40 and three quarters within 108. This study's 56 months is below that.

Counting every sponsor, half of 442 studies reached it within 60 months; a quarter within 38 and three quarters within 104.

How these figures are worked out, and which studies are left out of them.

Registered
Sep 20, 2017
First posted
Sep 21, 2017
StartActual
Sep 24, 2018
Primary completionActual
May 31, 2023
CompletionEst.
Dec 18, 2026
Results first posted
Apr 2, 2024
Last update
Jun 23, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jun 23, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03289910
Responsible party
Sponsor
First posted
Sep 21, 2017
Start date
Sep 24, 2018
Primary completion
May 31, 2023
Completion
Dec 18, 2026 (estimated)
Results posted
Apr 2, 2024
Last update
Jun 23, 2026

Study contacts

Keith W Pratz
principal investigator · JHU Sidney Kimmel Comprehensive Cancer Center LAO

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.

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Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Veliparib

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