Trial · NCT03727841
Marizomib for Recurrent Low-Grade and Anaplastic Supratentorial, Infratentorial, and Spinal Cord Ependymoma
NCT03727841, Phase 2, Terminated, National Cancer Institute (NCI); results first posted 2021-11-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Marizomib for Recurrent Low-Grade and Anaplastic Supratentorial, Infratentorial, and Spinal Cord Ependymoma. Trials, record NCT03727841. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03727841
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Marizomib for Recurrent Low-Grade and Anaplastic Supratentorial, Infratentorial, and Spinal Cord Ependymoma
- NCT number
- NCT03727841
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 4 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
- Anaplastic Ependymoma, Ependymoma, Ependymomas
- Interventions
- Marizomib
- First posted
- 2018-11-01
- Last updated
- 2022-07-27
- Results first posted
- 2021-11-30
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants That Are Progression-free at 6 Months Time Point After Initiation of Treatment | 6 months | Progression was assessed by the Response assessment in neuro-oncology criteria (RANO) and is defined as a 25% increase in the sum of all measurable lesions over smallest sum observed (over baseline if no decrease) using the same techniques as baseline. Appearance of new lesions, or clear worsening of any evaluable disease. Failure to return for evaluation due to death or deteriorating condition (unless clearly unrelated to this cancer). |
| Secondary | Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | At end of study, up to 16 months | To determine the safety of marizomib in recurrent ependymoma patients and in a subset of patients with more than 2 prior chemotherapies (RELA-fusion Cohort 2 and non-RELA-fusion Cohort 4). |
| Secondary | Number of Participants That Have Progressive Disease After 6 Months | 6 months | To estimate the efficacy of marizomib in recurrent ependymoma patients (RELA- fusion Cohort 2 and non RELA-fusion Cohort 4) with more than 2 prior chemotherapies as measured by progression free disease after 6 months. Progression was assessed by the Response Assessment in Neuro-oncology (RANO) criteria and is defined as a 25% increase in the sum of all measurable lesions over smallest sum observed (over baseline if no decrease) using the same techniques as baseline. Appearance of new lesions, or clear worsening of any evaluable disease. Failure to return for evaluation due to death or deteriorating condition (unless clearly unrelated to this cancer). |
| Secondary | Number of Participants With 12-month Progression-free Survival (PFS12) | 12 months | Progression was assessed by the Response Assessment in Neuro-oncology criteria (RANO) and is defined as a 25% increase in the sum of all measurable lesions over smallest sum observed (over baseline if no decrease) using the same techniques as baseline. Appearance of new lesions, or clear worsening of any evaluable disease. Failure to return for evaluation due to death or deteriorating condition (unless clearly unrelated to this cancer). |
| Secondary | Symptom Severity Using the MD Anderson Symptom Inventory- Brain Tumor (MDASI-BT) and/or MD Anderson Symptom Inventory - Spine Tumor Module (MDASI-SP) Instrument | End of study | Changes in quality of life using the MD Anderson Symptom Inventory- Brain Tumor (MDASI-BT) and/or MD Anderson Symptom Inventory - Spine Tumor Module (MDASI-SP) instrument was used to determine symptom severity.To longitudinally evaluate participant reported outcome measures using self-reported symptom severity with daily activities using the MDASI-BT and/or MDASI-SP instrument. |
| Secondary | Number of Participants Assessed Using the Response Assessment in Neuro-oncology Criteria (RANO) for Complete Response (CR) + Partial Response (PR) | Up to 16 months | To estimate the efficacy of marizomib in recurrent ependymoma patients (RELA- fusion Cohort 2 and non RELA-fusion Cohort 4) with more than 2 prior chemotherapies as measured by objective response. Complete Response is complete disappearance of all measurable and evaluable disease. No new lesions. No evidence of non-evaluable disease. Partial Response is greater than or equal to 50% decrease under baseline in the sum of products of perpendicular diameters of all measurable lesions. No progression of evaluable disease. No new lesions. |
| Secondary | Median Amount of Time Subject Survives 12 Months After Therapy | 12 months after therapy | Median amount of time subject survives 12 months after therapy |
| Secondary | Overall Survival (OS) of RELAfusion and Non RELA-fusion Recurrent Ependymoma Patients Treated With Marizomib. | Start of treatment until end of study | OS is defined as the time from treatment start date until date of death or date last known alive. |
| Secondary | Symptom Interference Using the MD Anderson Symptom Inventory- Brain Tumor (MDASI-BT) and/or MD Anderson Symptom Inventory - Spine Tumor Module (MDASI-SP) Instrument | End of study | Changes in quality of life using the MD Anderson Symptom Inventory- Brain Tumor (MDASI-BT) and/or MD Anderson Symptom Inventory - Spine Tumor Module (MDASI-SP) instrument was used to determine symptom interference.To longitudinally evaluate participant reported outcome measures using self-reported symptom interference with daily activities using the MDASI-BT and/or MDASI-SP instrument. |
| Other | Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) | Date treatment consent signed to date off study, approximately 14 months and 27 days, and 7 months and 22 days for the first and second group respectively. | Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants That Are Progression-free at 6 Months Time Point After Initiation of Treatment | 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | At end of study, up to 16 months | Toxicities | NUMBER | |
| SECONDARY | Number of Participants That Have Progressive Disease After 6 Months | 6 months | The one participant in Cohort 2 had progressive disease prior to 6 month mark and we did not enroll in Cohort 4 due to early study termination. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With 12-month Progression-free Survival (PFS12) | 12 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Symptom Severity Using the MD Anderson Symptom Inventory- Brain Tumor (MDASI-BT) and/or MD Anderson Symptom Inventory - Spine Tumor Module (MDASI-SP) Instrument | End of study | This outcome measure was not done because we were unable to determine due to low accrual and few data timepoints for which meaningful data could be extracted. | ||
| SECONDARY | Number of Participants Assessed Using the Response Assessment in Neuro-oncology Criteria (RANO) for Complete Response (CR) + Partial Response (PR) | Up to 16 months | We did not enroll to Cohort 4 due to early termination of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Median Amount of Time Subject Survives 12 Months After Therapy | 12 months after therapy | This outcome measure was not done. We were unable to determine as participants were taken off study for early study termination prior to the 12 months post therapy timepoint. | ||
| SECONDARY | Overall Survival (OS) of RELAfusion and Non RELA-fusion Recurrent Ependymoma Patients Treated With Marizomib. | Start of treatment until end of study | This outcome measure was not done. We were unable to determine for non RELA-fusion recurrent ependymoma patients since we did not enroll in those cohorts due to early study termination. And for the 4 participants with RELA-fusion recurrent ependymoma enrolled, the study was prematurely terminated pharmaceutical sponsor. | ||
| SECONDARY | Symptom Interference Using the MD Anderson Symptom Inventory- Brain Tumor (MDASI-BT) and/or MD Anderson Symptom Inventory - Spine Tumor Module (MDASI-SP) Instrument | End of study | This outcome measure was not done because we were unable to determine due to low accrual and few data timepoints for which meaningful data could be extracted. | ||
| OTHER_PRE_SPECIFIED | Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) | Date treatment consent signed to date off study, approximately 14 months and 27 days, and 7 months and 22 days for the first and second group respectively. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants That Are Progression-free at 6 Months Time Point After Initiation of Treatment | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 1 | Participants | ||
| Number of Participants That Are Progression-free at 6 Months Time Point After Initiation of Treatment | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 0 | Participants | ||
| Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | Grade 1 | 35 | Toxicities | |
| Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | Grade 1 | 13 | Toxicities | |
| Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | Grade 2 | 24 | Toxicities | |
| Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | Grade 2 | 5 | Toxicities | |
| Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | Grade 3 | 6 | Toxicities | |
| Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | Grade 3 | 0 | Toxicities | |
| Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | Grade 4 | 0 | Toxicities | |
| Number of Toxicities by Grade Using the Common CTCAE Version 5.0. | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | Grade 4 | 0 | Toxicities |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 3 | |
| Overall Study | STARTED | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 1 | |
| Overall Study | COMPLETED | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 0 | |
| Overall Study | COMPLETED | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 0 | |
| Overall Study | NOT COMPLETED | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 3 | |
| Overall Study | NOT COMPLETED | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 1 |
Source ClinicalTrials.gov
Adverse events80
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 | Agitation | Psychiatric disorders | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 1 | 3 | 1 |
| other | Agitation | Psychiatric disorders | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 0 | 1 | 0 |
| other | Anemia | Blood and lymphatic system disorders | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 0 | 3 | 0 |
| other | Anemia | Blood and lymphatic system disorders | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 1 | 1 | 1 |
| other | Anorexia | Metabolism and nutrition disorders | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 1 | 3 | 1 |
| other | Anorexia | Metabolism and nutrition disorders | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 0 | 1 | 0 |
| other | Ataxia | Nervous system disorders | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 1 | 3 | 1 |
| other | Ataxia | Nervous system disorders | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 0 | 1 | 0 |
| other | Blood lactate dehydrogenase increased | Investigations | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 1 | 3 | 1 |
| other | Blood lactate dehydrogenase increased | Investigations | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 0 | 1 | 0 |
1 to 10 of 80Source ClinicalTrials.gov
See all 80 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 0 | 3 |
| Total, all-cause mortality | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 0 | 1 |
| Total, other adverse events | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 3 | 3 |
| Total, other adverse events | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 1 | 1 |
| Total, serious adverse events | 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies | 1 | 3 |
| Total, serious adverse events | 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies | 0 | 1 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
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.
None in this release.
Trial comparisons7
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies vs 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies: Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) | Trial comparisons |
1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies vs 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies: Number of Participants That Are Progression-free at 6 Months Time Point After Initiation of Treatment | Trial comparisons |
1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies vs 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies: Number of Participants That Have Progressive Disease After 6 Months | Trial comparisons |
1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies vs 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies: Number of Participants With 12-month Progression-free Survival (PFS12) | Trial comparisons |
1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies vs 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies: Number of Participants Assessed Using the Response Assessment in Neuro-oncology Criteria (RANO) for Complete Response (CR) + Partial Response (PR) | Trial comparisons |
| 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies vs 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies: deaths from any cause | Trial comparisons |
| 1/Cohort 1 - Participants With Ependymoma Who Had None, One or Two Prior Chemotherapies vs 2/Cohort 2 - Participants With Ependymoma Who Had More Than Two Prior Chemotherapies: serious adverse events | Trial comparisons |
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/NCT03727841
GET https://conductscience.org/api/trials/v1/NCT03727841/events?page=2