Trial · NCT01059071
Safety Study for Refractory or Relapsed Neuroblastoma With DFMO Alone and in Combination With Etoposide
NCT01059071, Phase 1, Completed, Giselle Sholler; results first posted 2016-06-20
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety Study for Refractory or Relapsed Neuroblastoma With DFMO Alone and in Combination With Etoposide. Trials, record NCT01059071. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01059071
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety Study for Refractory or Relapsed Neuroblastoma With DFMO Alone and in Combination With Etoposide
- NCT number
- NCT01059071
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 21 participants, actual
- Lead sponsor
- Giselle Sholler
- Conditions
- Neuroblastoma
- Interventions
- DFMO, Etoposide
- First posted
- 2010-01-29
- Last updated
- 2024-08-06
- Results first posted
- 2016-06-20
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events as a Measure of Safety and Tolerability | length of study plus 30 days | To determine the safety, tolerability and maximum tolerated dose (MTD) of DFMO as a single agent and in combination with etoposide in pediatric and young adult patients with refractory or recurrent neuroblastoma |
| Secondary | Progression Free Survival (PFS) | 2 years | Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions |
| Secondary | Number of Patients With an Overall Response Rate (ORR) of PR or CR | 1 year | Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. |
| Secondary | Tmax of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | Cycle 1 Day 8 at hour 0 (pre-dose), 30 minutes, 1 hour, 3 hours, and 6 hours | |
| Secondary | Cmax of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | Cycle 1 Day 8 at hour 0 (pre-dose), 30 minutes, 1 hour, 3 hours, and 6 hours | |
| Secondary | AUC of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | Cycle 1 Day 8 at hour 0 (pre-dose), 30 minutes, 1 hour, 3 hours, and 6 hours |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events as a Measure of Safety and Tolerability | length of study plus 30 days | Received at least one dose of DFMO | participants | NUMBER |
| SECONDARY | Progression Free Survival (PFS) | 2 years | 18 subjects out of the 21 enrolled were evaluable. 3 subjects did not make it to an evaluable time point. | Days | MEDIAN |
| SECONDARY | Number of Patients With an Overall Response Rate (ORR) of PR or CR | 1 year | 18 subjects out of the 21 enrolled were evaluable. 3 subjects did not make it to an evaluable time point. | participants | NUMBER |
| SECONDARY | Tmax of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | Cycle 1 Day 8 at hour 0 (pre-dose), 30 minutes, 1 hour, 3 hours, and 6 hours | Cohort at max dose of 1500mg/m2 BID | hours | MEAN |
| SECONDARY | Cmax of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | Cycle 1 Day 8 at hour 0 (pre-dose), 30 minutes, 1 hour, 3 hours, and 6 hours | Cohort at max dose of 1500mg/m2 BID | mcg/ml | MEAN |
| SECONDARY | AUC of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | Cycle 1 Day 8 at hour 0 (pre-dose), 30 minutes, 1 hour, 3 hours, and 6 hours | Cohort at max dose of 1500mg/m2 BID | (mcg/ml) * hr | MEAN |
Source ClinicalTrials.gov
Results by group9
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events as a Measure of Safety and Tolerability | Dose Level 1: 500 mg/m2 PO BID | 2 | participants | ||
| Number of Participants With Adverse Events as a Measure of Safety and Tolerability | Dose Level 2: 750 mg/m2 PO BID | 4 | participants | ||
| Number of Participants With Adverse Events as a Measure of Safety and Tolerability | Dose Level 3:1000 mg/m2 PO BID | 0 | participants | ||
| Number of Participants With Adverse Events as a Measure of Safety and Tolerability | Dose Level 4:1500 mg/m2 PO BID | 5 | participants | ||
| Progression Free Survival (PFS) | DFMO and Etoposide | 85 | 95% Confidence Interval 62 to 418 | Days | |
| Number of Patients With an Overall Response Rate (ORR) of PR or CR | DFMO and Etoposide | 4 | participants | ||
| Tmax of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | DFMO and Etoposide | 2.88 | Standard Deviation 1.45 | hours | |
| Cmax of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | DFMO and Etoposide | 28.89 | Standard Deviation 14.96 | mcg/ml | |
| AUC of DFMO in Pediatrics Using Pharmacokinetic (PK) Testing. | DFMO and Etoposide | 108.38 | Standard Deviation 53.23 | (mcg/ml) * hr |
Source ClinicalTrials.gov
Participant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Dose Level 1: 500 mg/m2 PO BID | 4 | |
| Overall Study | STARTED | Dose Level 2: 750 mg/m2 PO BID | 5 | |
| Overall Study | STARTED | Dose Level 3:1000 mg/m2 PO BID | 3 | |
| Overall Study | STARTED | Dose Level 4:1500 mg/m2 PO BID | 9 | |
| Overall Study | COMPLETED | Dose Level 1: 500 mg/m2 PO BID | 2 | |
| Overall Study | COMPLETED | Dose Level 2: 750 mg/m2 PO BID | 1 | |
| Overall Study | COMPLETED | Dose Level 3:1000 mg/m2 PO BID | 1 | |
| Overall Study | COMPLETED | Dose Level 4:1500 mg/m2 PO BID | 3 | |
| Overall Study | NOT COMPLETED | Dose Level 1: 500 mg/m2 PO BID | 2 | |
| Overall Study | NOT COMPLETED | Dose Level 2: 750 mg/m2 PO BID | 4 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events24
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 | DFMO and Etoposide | 4 | 21 | 8 |
| other | Neutorpenia | Blood and lymphatic system disorders | DFMO and Etoposide | 5 | 21 | 9 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | DFMO and Etoposide | 1 | 21 | 5 |
| other | Leukopenia | Blood and lymphatic system disorders | DFMO and Etoposide | 1 | 21 | 1 |
| other | ALT elevation | Hepatobiliary disorders | DFMO and Etoposide | 2 | 21 | 2 |
| other | Anorexia | General disorders | DFMO and Etoposide | 1 | 21 | 1 |
| other | AST elevation | Hepatobiliary disorders | DFMO and Etoposide | 3 | 21 | 3 |
| other | Conjunctivitis | Eye disorders | DFMO and Etoposide | 1 | 21 | 1 |
| other | Constipation | Gastrointestinal disorders | DFMO and Etoposide | 2 | 21 | 2 |
| other | Diarrhea | Gastrointestinal disorders | DFMO and Etoposide | 1 | 21 | 1 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DFMO and Etoposide | ||
| Total, other adverse events | DFMO and Etoposide | 11 | 21 |
| Total, serious adverse events | DFMO and Etoposide | 3 | 21 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26018967 | DERIVED |
Source PubMed
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/NCT01059071
GET https://conductscience.org/api/trials/v1/NCT01059071/events?page=2