Trial · NCT03257631
A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors
NCT03257631, Phase 2, Completed, Celgene; results first posted 2019-12-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors. Trials, record NCT03257631. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03257631
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors
- NCT number
- NCT03257631
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 53 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Central Nervous System Neoplasms, Medulloblastoma
- Interventions
- Pomalidomide, Pomalidomide
- First posted
- 2017-08-22
- Last updated
- 2024-04-16
- Other registries' IDs
- eudract:2016-002903-25
- Results first posted
- 2019-12-10
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With an Objective Response and Long-term Stable Disease | 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group | The percentage of participants who achieved either an objective response, defined as a complete response (CR) or partial response (PR) in the first 6 cycles of treatment (or within 3 cycles for DIPG), or long-term stable disease (SD) defined as SD maintained for ≥ 6 cycles (≥ 3 cycles for DIPG), measured from first dose date. CR: Disappearance of all lesions and no new lesions. PR: A reduction of ≥ 50% in the size of measurable lesions, and/or persistence of non-target lesions with no progression or decrease in size. SD: A decrease of \< 50% or an increase of \< 25% in the size of measurable lesions and no evidence of new lesions, response does not meet the criteria for CR, PR, or progressive disease, and/or the persistence of non-target lesions with no progression or decrease in size. Progressive Disease (PD): ≥ 25% increase in the size of the measurable lesions, or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions. |
| Secondary | Percentage of Participants Who Achieved an Objective Response (ORR) | 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group | Objective response rate was defined as the percentage of participants who achieved a complete response (CR) or partial response (PR) within the first 6 cycles of treatment (or within 3 cycles for participants in the DIPG group). Disease assessments were based on MRI and assessed by an independent central review. CR: Disappearance of all lesions and no new lesions. PR: A reduction of ≥ 50% in the size of measurable lesions compared to baseline, and/or the persistence of non-target lesions with no progression or decrease in size. Progressive Disease (PD): ≥ 25% increase in the size of the measurable lesions, or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions. |
| Secondary | Percentage of Participants With Long-term Stable Disease | 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group | Long-term stable disease (SD) rate was defined as the percentage of participants who achieved SD maintained for ≥ 6 cycles (or \> 3 cycles for DIPG), measured from the date of first dose of treatment. Disease assessments were based on MRI and assessed by an independent central review. SD: A decrease of \< 50% or an increase of \< 25% in the size of measurable lesions and no evidence of new lesions, response does not meet the criteria for CR, PR, or progressive disease, and/or the persistence of non-target lesions with no progression or decrease in size. CR: Disappearance of all lesions and no new lesions. PR: A reduction of ≥ 50% in the size of measurable lesions compared to baseline, and/or the persistence of non-target lesions with no progression or decrease in size. Progressive Disease (PD): ≥ 25% increase in the size of the measurable lesions, or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions. |
| Secondary | Duration of Response (DoR) | From the first dose of pomalidomide to the date of the first documented tumor progression or death due to any cause, whichever occurs first (Up to 71 months) | DoR is defined as the time from the date of the first objective response (complete response \[CR\] or partial response \[PR\]) to disease progression. Participants who did not have disease progression or had not died were censored at the time of their last disease assessment or at the time of start of new anticancer therapy, whichever occurred first. Progressive disease (PD): ≥ 25% increase in the size of the measurable lesions taking as a reference the smallest disease measurement recorded since the start of protocol therapy (nadir), or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions, or if spine MRI and/or lumbar cerebrospinal fluid (CSF) cytology were previously negative and became positive. CR: Disappearance of all lesions and no new lesions. PR: A reduction of ≥ 50% in the size of measurable lesions compared to baseline, and/or the persistence of non-target lesions with no progression or decrease in size. |
| Secondary | Kaplan-Meier Estimate of Progression-Free Survival (PFS) | From the first dose of pomalidomide to the date of the first documented tumor progression or death due to any cause, whichever occurs first (Up to 71 months) | Progression-free survival was defined as the time from the date of first dose of pomalidomide until the date progressive disease (PD) was first observed or until the date of death due to any cause, whichever occurred first. Participants who did not have PD or had not died at the time of analysis were censored at the time of their last disease assessment or at the start of new anticancer therapy, whichever occurred first. Progressive Disease (PD): ≥ 25% increase in the size of the measurable lesions taking as a reference the smallest disease measurement recorded since the start of protocol therapy (nadir), or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions, or if spine MRI and/or lumbar CSF cytology were previously negative and became positive. |
| Secondary | Kaplan-Meier Estimate of Overall Survival (OS) | From the first dose of pomalidomide to the date of death due to any cause (Up to 71 months) | Overall survival was defined as the time from the date of the first dose to the date of death (any cause). Participants who were alive were censored at the last known time that the participant was alive. |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From the first dose of pomalidomide until 28 days after the last dose (Up to approximately 72 months) | Treatment-emergent adverse events were defined as any adverse events (AE) occurring from the first dose of pomalidomide until 28 days after the last dose. The severity of each AE was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 4.03 and according to the following scale: Grade 1: Mild (transient or mild discomfort; no limitation in activity or medical intervention required); Grade 2: Moderate (mild to moderate limitation in activity, assistance may be needed; minimal medical intervention required); Grade 3: Severe (marked limitation in activity, assistance and medical intervention required, hospitalization possible); Grade 4: Life-threatening (extreme limitation in activity, significant assistance or medical intervention required, hospitalization or hospice care probable); Grade 5: Death. Drug-related AEs are those suspected by the Investigator as being related to administration of study drug. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With an Objective Response and Long-term Stable Disease | 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group | The response population consisted of all enrolled participants receiving at least one cycle of pomalidomide if therapy was not discontinued earlier due to progressive disease (PD) | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Achieved an Objective Response (ORR) | 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group | The response population consisted of all enrolled participants receiving at least one cycle of pomalidomide if therapy was not discontinued earlier due to progressive disease (PD) | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Long-term Stable Disease | 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group | The response population consisted of all enrolled participants receiving at least one cycle of pomalidomide if therapy was not discontinued earlier due to progressive disease (PD) | percentage of participants | NUMBER |
| SECONDARY | Duration of Response (DoR) | From the first dose of pomalidomide to the date of the first documented tumor progression or death due to any cause, whichever occurs first (Up to 71 months) | All enrolled participants with response (PR or CR), regardless of whether they received any treatment or not | weeks | MEDIAN |
| SECONDARY | Kaplan-Meier Estimate of Progression-Free Survival (PFS) | From the first dose of pomalidomide to the date of the first documented tumor progression or death due to any cause, whichever occurs first (Up to 71 months) | The intent-to-treat population includes all enrolled participants, regardless of whether they received any treatment or not | weeks | MEDIAN |
| SECONDARY | Kaplan-Meier Estimate of Overall Survival (OS) | From the first dose of pomalidomide to the date of death due to any cause (Up to 71 months) | The intent-to-treat population includes all enrolled participants, regardless of whether they received any treatment or not | months | MEDIAN |
| SECONDARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From the first dose of pomalidomide until 28 days after the last dose (Up to approximately 72 months) | The safety population included all participants who received at least 1 dose of study drug | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group62
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With an Objective Response and Long-term Stable Disease | Diffuse Intrinsic Pontine Glioma | 0 | 95% Confidence Interval 0.0 to 33.6 | percentage of participants | |
| Percentage of Participants With an Objective Response and Long-term Stable Disease | Ependymoma | 11.1 | 95% Confidence Interval 0.3 to 48.2 | percentage of participants | |
| Percentage of Participants With an Objective Response and Long-term Stable Disease | High-grade Glioma | 10.5 | 95% Confidence Interval 1.3 to 33.1 | percentage of participants | |
| Percentage of Participants With an Objective Response and Long-term Stable Disease | Medulloblastoma | 0 | 95% Confidence Interval 0.0 to 33.6 | percentage of participants | |
| Percentage of Participants Who Achieved an Objective Response (ORR) | Diffuse Intrinsic Pontine Glioma | 0 | 95% Confidence Interval 0.0 to 33.6 | percentage of participants | |
| Percentage of Participants Who Achieved an Objective Response (ORR) | Ependymoma | 0 | 95% Confidence Interval 0.0 to 33.6 | percentage of participants | |
| Percentage of Participants Who Achieved an Objective Response (ORR) | High-grade Glioma | 5.3 | 95% Confidence Interval 0.1 to 26.0 | percentage of participants | |
| Percentage of Participants Who Achieved an Objective Response (ORR) | Medulloblastoma | 0 | 95% Confidence Interval 0.0 to 33.6 | percentage of participants | |
| Percentage of Participants With Long-term Stable Disease | Diffuse Intrinsic Pontine Glioma | 0 | 95% Confidence Interval 0.0 to 33.6 | percentage of participants | |
| Percentage of Participants With Long-term Stable Disease | Ependymoma | 11.1 | 95% Confidence Interval 0.3 to 48.2 | percentage of participants |
1 to 10 of 62Source ClinicalTrials.gov
See all 62 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Diffuse Intrinsic Pontine Glioma | 11 | |
| Overall Study | STARTED | Ependymoma | 9 | |
| Overall Study | STARTED | High-grade Glioma | 23 | |
| Overall Study | STARTED | Medulloblastoma | 10 | |
| Overall Study | Received Study Drug | Diffuse Intrinsic Pontine Glioma | 11 | |
| Overall Study | Received Study Drug | Ependymoma | 9 | |
| Overall Study | Received Study Drug | High-grade Glioma | 22 | |
| Overall Study | Received Study Drug | Medulloblastoma | 10 | |
| Overall Study | COMPLETED | Diffuse Intrinsic Pontine Glioma | 0 | |
| Overall Study | COMPLETED | Ependymoma | 0 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events628
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 | Anaemia | Blood and lymphatic system disorders | Diffuse Intrinsic Pontine Glioma | 1 | 11 | |
| other | Anaemia | Blood and lymphatic system disorders | Ependymoma | 3 | 9 | |
| other | Anaemia | Blood and lymphatic system disorders | High-grade Glioma | 7 | 22 | |
| other | Anaemia | Blood and lymphatic system disorders | Medulloblastoma | 2 | 10 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Diffuse Intrinsic Pontine Glioma | 0 | 11 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Ependymoma | 0 | 9 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | High-grade Glioma | 2 | 22 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Medulloblastoma | 0 | 10 | |
| other | Leukopenia | Blood and lymphatic system disorders | Diffuse Intrinsic Pontine Glioma | 3 | 11 | |
| other | Leukopenia | Blood and lymphatic system disorders | Ependymoma | 6 | 9 |
1 to 10 of 628Source ClinicalTrials.gov
See all 628 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Diffuse Intrinsic Pontine Glioma | 11 | 11 |
| Total, all-cause mortality | Ependymoma | 6 | 9 |
| Total, all-cause mortality | High-grade Glioma | 15 | 23 |
| Total, all-cause mortality | Medulloblastoma | 9 | 10 |
| Total, other adverse events | Diffuse Intrinsic Pontine Glioma | 11 | 11 |
| Total, other adverse events | Ependymoma | 8 | 9 |
| Total, other adverse events | High-grade Glioma | 20 | 22 |
| Total, other adverse events | Medulloblastoma | 9 | 10 |
| Total, serious adverse events | Diffuse Intrinsic Pontine Glioma | 9 | 11 |
| Total, serious adverse events | Ependymoma | 4 | 9 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers2
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 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/NCT03257631
GET https://conductscience.org/api/trials/v1/NCT03257631/results?page=2