Trial · NCT03953898
Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation
NCT03953898, Phase 2, Active not recruiting, National Cancer Institute (NCI); results first posted 2025-03-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation. Trials, record NCT03953898. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03953898
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation
- NCT number
- NCT03953898
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 14 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia Post Cytotoxic Therapy, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome, Refractory Acute Myeloid Leukemia, Refractory Myelodysplastic Syndrome
- Interventions
- Biospecimen Collection, Bone Marrow Aspiration, Olaparib
- First posted
- 2019-05-17
- Last updated
- 2026-09-10
- Results first posted
- 2025-03-30
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Cumulative Overall Response Rate (ORR) | Up to 6 cycles | Will be evaluated by Myelodysplastic Syndrome International Working Group (IWG) 2006 criteria (Cheson et al., 2006) and acute myeloid leukemia (AML) IWG 2003 criteria (Cheson et al., 2003) after 6 cycles of treatment. Cumulative ORR will include complete remission (CR), complete remission with incomplete blood count recovery (CRi), partial response (PR), and bone marrow complete remission (marrow CR) achieved at least at one point during these 6 cycles. |
| Secondary | ORR | Up to 12 months | The effectiveness of the drug in patients for each cohort will be independently assessed by ORR. The exact two-sided 95% confidence intervals (CI) for the ORR will be reported. The CI based on the Greenwoods variance will be reported. |
| Secondary | Progression-free Survival (PFS) | From first day of therapy to the time of documentation of progression, death of any cause, or last follow-up, whichever comes first, assessed up to 12 months | Will be estimated using the Kaplan-Meier method with the 95% CIs. The CI based on the Greenwoods variance will be reported. In addition, the possible risk factors will be compared for survival with log-rank test. For multivariate analysis, the proportional hazards Cox model will be applied to investigate potential prognostic factors, such as age and stage of disease on the survival data. The adjusted p-values of the odds ratios and the adjusted 95% confidence interval will be reported. |
| Secondary | Overall Survival (OS) | From first day of therapy to the time of death or last follow-up, whichever comes first, assessed up to 12 months | Will be estimated using the Kaplan-Meier method with the 95% CIs. The CI based on the Greenwoods variance will be reported. In addition, the possible risk factors will be compared for survival with log-rank test. For multivariate analysis, the proportional hazards Cox model will be applied to investigate potential prognostic factors, such as age and stage of disease on the survival data. The adjusted p-values of the odds ratios and the adjusted 95% confidence interval will be reported. |
| Secondary | Duration of Response (DOR) | From first documentation of response to the time of documentation of progression, death of any cause, or last follow-up, whichever comes first, assessed up to 12 months | |
| Secondary | Incidence of Adverse Events | Up to 12 months | Non-hematologic toxicity will be evaluated by Common Terminology Criteria for Adverse Events version 5 criteria. Presented are the count of those that experienced at least one non-serious adverse event. |
| Other | Change in 2-hydroxyglutarate (2HG) Levels | Up to 12 months | The Mann-Whitney U test will be used to test for differences in post-treatment plasma 2HG concentrations between patients with a response to treatment and those without. Will also test for differences in Delta2HG (defined as pre-treatment minus post-treatment plasma concentration) between patients with a response to treatment and those without. Differences with p =\< 0.05 will be considered significant. The area under the receiver operating characteristic curve (ROC AUC) will be calculated to determine the cutoff value of the Delta2HG difference. The optimal cutoff value will be determined at the point on the ROC curve at (sensitivity + specificity - 1) is maximized. |
| Other | Minimal Residual Disease (MRD) Assessment | Up to 12 months | Will define MRD based on the variation of the variant allele frequency of the IDH1/2 mutation in the bone marrow of the patients before and during therapy. Will evaluate two different variables: MRD negativity (defined by the absence of detection of the IDH mutant in the sample) and the molecular response (defined by the log reduction of the frequency of the mutant allele). MRD negativity is a qualitative variable and will be reported as a percentage with 95% confidence interval for each time point and mutation. Will compare the different groups using a Chi-Square test. Molecular response is a quantitative variable reported as a median, min and max for each time point and we will use a student t test for the comparison of the different groups. |
| Other | Mutant Allele Frequency | Up to 12 months | Will be estimated using Poisson distribution model as the fraction of positive reads divided by total reads containing a target. The limit of detection will be defined for each mutation as the mean value of IDH1/2 wild-type controls plus three standard deviations. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Cumulative Overall Response Rate (ORR) | Up to 6 cycles | Participants receiving treatment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | ORR | Up to 12 months | Participants receiving treatment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Progression-free Survival (PFS) | From first day of therapy to the time of documentation of progression, death of any cause, or last follow-up, whichever comes first, assessed up to 12 months | Participants receiving treatment | months | MEDIAN |
| SECONDARY | Overall Survival (OS) | From first day of therapy to the time of death or last follow-up, whichever comes first, assessed up to 12 months | Participants receiving treatment | months | MEDIAN |
| SECONDARY | Duration of Response (DOR) | From first documentation of response to the time of documentation of progression, death of any cause, or last follow-up, whichever comes first, assessed up to 12 months | no patients responded to treatment | ||
| SECONDARY | Incidence of Adverse Events | Up to 12 months | 12 pariticipants received treatment. | Participants | COUNT_OF_PARTICIPANTS |
| OTHER_PRE_SPECIFIED | Change in 2-hydroxyglutarate (2HG) Levels | Up to 12 months | |||
| OTHER_PRE_SPECIFIED | Minimal Residual Disease (MRD) Assessment | Up to 12 months | |||
| OTHER_PRE_SPECIFIED | Mutant Allele Frequency | Up to 12 months |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Cumulative Overall Response Rate (ORR) | Treatment (Olaparib) | Complete remission (CR) | 0 | Participants | |
| Cumulative Overall Response Rate (ORR) | Treatment (Olaparib) | Complete remission with incomplete hematologic response | 0 | Participants | |
| Cumulative Overall Response Rate (ORR) | Treatment (Olaparib) | Complete remission with partial hematologic response | 0 | Participants | |
| Cumulative Overall Response Rate (ORR) | Treatment (Olaparib) | Composite CR rate (CR+CRi+CRh) | 0 | Participants | |
| Cumulative Overall Response Rate (ORR) | Treatment (Olaparib) | No Response | 12 | Participants | |
| ORR | Treatment (Olaparib) | OR = Yes | 0 | Participants | |
| ORR | Treatment (Olaparib) | OR = No | 12 | Participants | |
| Progression-free Survival (PFS) | Treatment (Olaparib) | 2.3 | Full Range 0.4 to 5.6 | months | |
| Overall Survival (OS) | Treatment (Olaparib) | 3.4 | Full Range 0.4 to 35.3 | months | |
| Incidence of Adverse Events | Treatment (Olaparib) | 12 | Participants |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Treatment (Olaparib) | 14 | |
| Overall Study | Started Therapy | Treatment (Olaparib) | 12 | |
| Overall Study | COMPLETED | Treatment (Olaparib) | 0 | |
| Overall Study | NOT COMPLETED | Treatment (Olaparib) | 14 |
Source ClinicalTrials.gov
Adverse events88
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anemia | Blood and lymphatic system disorders | Treatment (Olaparib) | 6 | 12 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Treatment (Olaparib) | 4 | 12 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Treatment (Olaparib) | 2 | 12 | |
| other | Atrial fibrillation | Cardiac disorders | Treatment (Olaparib) | 1 | 12 | |
| other | Myocardial infarction | Cardiac disorders | Treatment (Olaparib) | 1 | 12 | |
| other | Sinus tachycardia | Cardiac disorders | Treatment (Olaparib) | 1 | 12 | |
| other | Vision decreased | Eye disorders | Treatment (Olaparib) | 1 | 12 | |
| other | Disease progression | General disorders | Treatment (Olaparib) | 1 | 12 | |
| other | Fatigue | General disorders | Treatment (Olaparib) | 1 | 12 | |
| other | Fever | General disorders | Treatment (Olaparib) | 1 | 12 |
1 to 10 of 88Source ClinicalTrials.gov
See all 88 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment (Olaparib) | 10 | 12 |
| Total, other adverse events | Treatment (Olaparib) | 12 | 12 |
| Total, serious adverse events | Treatment (Olaparib) | 9 | 12 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| UM1CA186689 | VICKtOrY Early Clinical Trials Consortium | LORUSSO, PATRICIA M. | YALE UNIVERSITY | NATIONAL CANCER INSTITUTE | 2014 to 2025 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03953898
GET https://conductscience.org/api/trials/v1/NCT03953898/events?page=2