Trial · NCT01935336
Study of Ponatinib in Patients With Lung Cancer Preselected Using Different Candidate Predictive Biomarkers
NCT01935336, Phase 2, Completed, University of Colorado, Denver; results first posted 2022-02-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of Ponatinib in Patients With Lung Cancer Preselected Using Different Candidate Predictive Biomarkers. Trials, record NCT01935336. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01935336
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Ponatinib in Patients With Lung Cancer Preselected Using Different Candidate Predictive Biomarkers
- NCT number
- NCT01935336
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 171 participants, actual
- Lead sponsor
- University of Colorado, Denver
- Conditions
Adenocarcinoma of the Lung, Extensive Stage Small Cell Lung Cancer, Limited Stage Small Cell Lung Cancer, Recurrent Non-small Cell Lung Cancer, Recurrent Small Cell Lung Cancer, Stage IIIA Non-small Cell Lung Cancer, Stage IIIB Non-small Cell Lung Cancer, Stage IV Non-small Cell Lung Cancer
- Interventions
- Ponatinib
- First posted
- 2013-09-05
- Last updated
- 2022-02-11
- Results first posted
- 2022-02-11
- Limitations and caveats
Due to poor tolerability and safety concerns regarding ponatinib after treating the first few patients, the study did not reach its accrual target (N\~70) for the treatment portion of this study and could not confirm or refute the null hypothesis (needed a minimum of 12 patients treated). This study prescreened a more heterogeneous population. As a result, all four patients that were enrolled onto the ponatinib treatment arm had either squamous or poorly differentiated lung cancer.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Biomarker FGFR1 (ISH/SISH) Score (Part A) | Baseline | Biomarker prevalence and its 95% (exact) confidence interval (CI) among the screening patients and for different histologies will be reported. Molecular cohorts for ISH and SISH positivity: FGFR1 ISH+/SISH+, FGFR1 ISH+/SISH-, FGFR1 ISH-/SISH+, and FGFR1 ISH-/SISH- |
| Primary | Overlapping Frequency of FGFR1 (ISH/SISH) Biomarkers (Part A) | Baseline | Overlapping frequency and its 95% CI between biomarkers among the screening patients and for different histologies will also be reported. |
| Primary | Objective Response Rate (ORR) Per RECIST v1.1 (Part B) | From date of first dose until date of Disease Progression or death (up to 153 days), whichever occurred first | Evaluated using Fisher's exact test with a descriptive p-value. Summarized using binomial proportions with 95% exact binomial confidence intervals. |
| Secondary | Incidence of Adverse Events, Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v4.0 | From date of first dose until date of Disease Progression (up to 153 days). Assessed at Day 1, Day 8, Day 15 of each 28 day cycle) | Adverse events will be tabulated per participant, per organ, and per visit. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Biomarker FGFR1 (ISH/SISH) Score (Part A) | Baseline | 171 cases were prescreened. Among them, 122 cases had both SISH and ISH scores reported, one case had only SISH and four cases had only ISH reported, and 44 cases lacked both SISH and ISH scores, resulting in 123 cases with SISH and 126 cases with ISH. Please note that the 126 cases with ISH scores were displayed in two different ways, one was based on ISH\<1% vs \>+1% (columns 3 and 4 above), and the other was based on ISH\<20% vs \>=20% (columns 5 and 6). | gene copy number | MEAN |
| PRIMARY | Overlapping Frequency of FGFR1 (ISH/SISH) Biomarkers (Part A) | Baseline | 171 subjects were pre-screen consented to request and submit tissue for ISH and SISH scoring. 122 of 171 subjects received ISH/SISH results and of those 122 subjects who received ISH/SISH results, 4 were enrolled to actual treatment with ponatinib. The number reported here are the eligible number of subjects in each pre-defined category. | gene copy number | MEAN |
| PRIMARY | Objective Response Rate (ORR) Per RECIST v1.1 (Part B) | From date of first dose until date of Disease Progression or death (up to 153 days), whichever occurred first | A total of 4 subjects were treated, where the 3 SISH-/ISH+ all with RET-, due to the safety concern of ponatinib and the change of inclusion and exclusion criteria. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Incidence of Adverse Events, Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v4.0 | From date of first dose until date of Disease Progression (up to 153 days). Assessed at Day 1, Day 8, Day 15 of each 28 day cycle) | Due to the safety warning of Ponatinib and the adjusted inclusion and exclusion criteria, the study only treated 4 subjects in total. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Biomarker FGFR1 (ISH/SISH) Score (Part A) | SISH- | 0.93 | 95% Confidence Interval 0.87 to 0.97 | gene copy number | |
| Biomarker FGFR1 (ISH/SISH) Score (Part A) | SISH+ | 0.07 | 95% Confidence Interval 0.03 to 0.134 | gene copy number | |
| Biomarker FGFR1 (ISH/SISH) Score (Part A) | ISH-(<1%) | 0.579 | 95% Confidence Interval 0.488 to 0.667 | gene copy number | |
| Biomarker FGFR1 (ISH/SISH) Score (Part A) | ISH+(>=1%) | 0.42 | 95% Confidence Interval 0.333 to 0.512 | gene copy number | |
| Biomarker FGFR1 (ISH/SISH) Score (Part A) | ISH<20% | 0.77 | 95% Confidence Interval 0.686 to 0.84 | gene copy number | |
| Biomarker FGFR1 (ISH/SISH) Score (Part A) | ISH>=20% | 0.23 | 95% Confidence Interval 0.16 to 0.31 | gene copy number | |
| Overlapping Frequency of FGFR1 (ISH/SISH) Biomarkers (Part A) | Ponatinib SISH+/ISH+ | 0.049 | 95% Confidence Interval 0.018 to 0.104 | gene copy number | |
| Overlapping Frequency of FGFR1 (ISH/SISH) Biomarkers (Part A) | Ponatinib SISH+/ISH- | 0.0246 | 95% Confidence Interval 0.005 to 0.070 | gene copy number | |
| Overlapping Frequency of FGFR1 (ISH/SISH) Biomarkers (Part A) | Ponatinib SISH-/ISH+ | 0.385 | 95% Confidence Interval 0.299 to 0.478 | gene copy number | |
| Overlapping Frequency of FGFR1 (ISH/SISH) Biomarkers (Part A) | Ponatinib SISH-/ISH- | 0.541 | 95% Confidence Interval 0.448 to 0.632 | gene copy number |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ponatinib SISH+/ISH+ | 6 | |
| Overall Study | STARTED | Ponatinib SISH+/ISH- | 3 | |
| Overall Study | STARTED | Ponatinib SISH-/ISH+ | 47 | |
| Overall Study | STARTED | Ponatinib SISH-/ISH- | 66 | |
| Overall Study | Started Treatment | Ponatinib SISH+/ISH+ | 1 | |
| Overall Study | Started Treatment | Ponatinib SISH+/ISH- | 0 | |
| Overall Study | Started Treatment | Ponatinib SISH-/ISH+ | 3 | |
| Overall Study | Started Treatment | Ponatinib SISH-/ISH- | 0 | |
| Overall Study | COMPLETED | Ponatinib SISH+/ISH+ | 0 | |
| Overall Study | COMPLETED | Ponatinib SISH+/ISH- | 0 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events12
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 | Grade 2 hypertension | Blood and lymphatic system disorders | Ponatinib SISH+/ISH+ | 1 | 1 | 1 |
| other | Grade 2 hypertension | Blood and lymphatic system disorders | Ponatinib SISH+/ISH- | 0 | 0 | 0 |
| other | Grade 2 hypertension | Blood and lymphatic system disorders | Ponatinib SISH-/ISH+ | 2 | 3 | 3 |
| other | Grade 2 hypertension | Blood and lymphatic system disorders | Ponatinib SISH-/ISH- | 0 | 0 | 0 |
| other | Grade 2 thrombocytopenia | Blood and lymphatic system disorders | Ponatinib SISH+/ISH+ | 1 | 1 | 1 |
| other | Grade 2 thrombocytopenia | Blood and lymphatic system disorders | Ponatinib SISH+/ISH- | 0 | 0 | 0 |
| other | Grade 2 thrombocytopenia | Blood and lymphatic system disorders | Ponatinib SISH-/ISH+ | 0 | 3 | 0 |
| other | Grade 2 thrombocytopenia | Blood and lymphatic system disorders | Ponatinib SISH-/ISH- | 0 | 0 | 0 |
| serious | Grade 3 AE | Gastrointestinal disorders | Ponatinib SISH+/ISH+ | 1 | 1 | 1 |
| serious | Grade 3 AE | Gastrointestinal disorders | Ponatinib SISH+/ISH- | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ponatinib SISH+/ISH+ | 0 | 1 |
| Total, all-cause mortality | Ponatinib SISH+/ISH- | 0 | 0 |
| Total, all-cause mortality | Ponatinib SISH-/ISH+ | 0 | 3 |
| Total, all-cause mortality | Ponatinib SISH-/ISH- | 0 | 0 |
| Total, other adverse events | Ponatinib SISH+/ISH+ | 1 | 1 |
| Total, other adverse events | Ponatinib SISH+/ISH- | 0 | 0 |
| Total, other adverse events | Ponatinib SISH-/ISH+ | 2 | 3 |
| Total, other adverse events | Ponatinib SISH-/ISH- | 0 | 0 |
| Total, serious adverse events | Ponatinib SISH+/ISH+ | 1 | 1 |
| Total, serious adverse events | Ponatinib SISH+/ISH- | 0 | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT01935336
GET https://conductscience.org/api/trials/v1/NCT01935336/registered