GIST, MalignantPhase 2POETIGNCT03171389
POETIG Trial - POnatinib After rEsisTance to Imatinib in GIST
A Phase 2 study testing Ponatinib 30 MG in people with GIST, Malignant and KIT Exon 13 Mutation, run by Sebastian Bauer at 1 site in Germany.
Trial summary
This is a non-randomized, open-label, multicenter phase 2 study to evaluate the efficacy and safety of ponatinib in patients with metastatic and/or unresectable GIST after prior failure or intolerability of imatinib. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- GIST, Malignant, KIT Exon 13 Mutation, KIT Gene Mutation
- What is being tested?
- Ponatinib 30 MG
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 81 participants
- Where
- 1 siteGermany
- Study dates
- 22 Mar 2017to 22 Sep 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients ≥18 years old
- GIST with failure or intolerance to imatinib or failure / intolerance to all three approved TKIs defined as:
- Histologically confirmed metastatic and/or unresectable GIST (harboring a primary KIT or PDGFRA-mutation) after failure or intolerance of imatinib (cohort A and B) or all three approved TKIs (cohort C). If prior TKI treatment was neoadjuvant therapy, then relapse must have occurred during the neoadjuvant therapy in order to consider it failed therapy
- Patients in Cohort A must have evidence of clinical resistance mutations in any other exon or no resistance mutation but evidence of progression by CT or MRI imaging. Patients in Cohort B must have evidence of an activating resistance mutation in KIT exon 13 (by direct sequencing of progressing lesions or by liquid biopsy).
- Measurable disease per modified RECIST 1.1 (Demetri et al., 2013). A lesion in a previously irradiated area is eligible to be considered as measurable disease as long as there is objective evidence of progression of the lesion prior to study enrollment
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
Show all 18Show fewer
- Adequate hepatic function as defined by the following criteria:
- Total serum bilirubin ≤1.5 x upper limit of normal (ULN), unless due to Gilbert's syndrome
- ALT (Alanine Aminotransferase) ≤2.5×ULN or ≤5.0xULN if liver metastases are present
- AST (Aspartate Aminotransferase) ≤2.5×ULN or ≤5.0xULN if liver metastases are present
- Adequate renal function as defined by the following criterion:
- Serum creatinine <1.5×ULN
- Adequate pancreatic function as defined by the following criterion:
- Serum lipase and amylase ≤1.5×ULN
- For patients of childbearing potential, a negative pregnancy test must be documented prior to enrollment
- Female and male patients who are fertile must agree to use an effective form of contraception with their sexual partners from signing of the informed consent form for this study through 4 months after the End-of-Treatment Provision of written informed consent
- Willingness and ability to comply with scheduled visits and study procedures
- Fully recovered (≤ Grade 1 or returned to baseline or deemed irreversible) from the acute effects of prior cancer therapy before initiation of the study drug treatment
Who cannot join
- Patients lacking primary mutations of KIT or PDGFRA (including Succinate-Dehydrogenase(SDH)-deficient GIST)
- Major surgery within 28 days prior to initiating therapy
- History of bleeding disorder
- History of acute pancreatitis within 1 year of study or history of chronic pancreatitis
- History of alcohol and /or drug abuse
- Uncontrolled hypertriglyceridemia (triglycerides >450 mg/dL)
Show all 18Show fewer
- Clinically significant, uncontrolled, or active cardiovascular disease, or other arterial or venous vascular occlusion diseases specifically including, but not restricted to: Myocardial infarction within 6 months prior to enrollment Unstable angina within 6 months prior to enrollment Congestive heart failure within 6 months prior to enrollment, or left ventricular ejection fraction (LVEF) less than lower limit of normal per local institutional standards History of clinically significant (as determined by the treating physician) atrial arrhythmia Any history of ventricular arrhythmia Cerebrovascular accident or transient ischemic attack within 6 months prior to enrollment Any history of peripheral arterial occlusive disease requiring revascularization Venous thromboembolism including deep venous thrombosis or pulmonary embolism within 6 months prior to enrollment
- Uncontrolled hypertension (diastolic blood pressure >90 mm Hg; systolic >140 mm Hg). Patients with hypertension should be under treatment on study entry to effect blood pressure control
- Taking medications that are known to be associated with Torsades de Pointes (Appendix A)
- Taking any medications or herbal supplements that are known to be strong inhibitors of CYP3A4 within at least 14 days before the first dose of ponatinib (Appendix B)
- Ongoing or active infection. This includes but is not limited to the requirement for intravenous antibiotics
- Known history of human immunodeficiency virus. Testing is not required in the absence of prior documentation or known history
- Pregnant or breastfeeding
- Malabsorption syndrome or other gastrointestinal illness that could affect oral absorption of the study drug
- Individuals with a history of a different malignancy, other than cervical cancer in situ, basal cell or squamous cell carcinoma of the skin, are ineligible, except if they have been disease-free for at least 5 years, and are deemed by the investigator to be at low risk for recurrence of that malignancy OR if the other primary malignancy is neither currently clinically significant nor requiring active intervention.
- Use of any approved TKIs or investigational agents within 2 weeks or 6 half-lives of the agent, whichever is longer, prior to receiving study drug
- Any condition or illness that, in the opinion of the investigator, would compromise patient safety or interfere with the evaluation of the drug
- History of apoplectic insult
Trial locations and contacts
1 site in 1 country.
Germany1 site, 1 recruiting
- Recruiting
West German Cancer Center
Essen, North Rhine-Westphalia, 45122
Sebastian Bauer, Prof. Dr.
About this study
Official title: Phase 2 Trial of Ponatinib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor (GIST) Following Failure or Intolerance of Prior Therapy With Imatinib
This is a non-randomized, open-label, multicenter phase 2 study to evaluate the efficacy and safety of ponatinib in patients with metastatic and/or unresectable GIST after prior failure or intolerability of imatinib. Patients will be enrolled into 1 of 2 cohorts based on absence (Cohort A) or presence (Cohort B) of KIT exon 13 resistance mutations as measured by liquid biopsy. A third cohort (Cohort C) will include patients who have received all approved lines of TKI treatments (imatinib, sunitinib and regorafenib).
Read the detailed description
Primary Objective
- To assess clinical benefit of ponatinib in patients with KIT or PDGFRA mutant GIST defined as clinical benefit rate (CBR), which is the composite of complete response (CR), partial response (PR) and stable disease (SD) at ≥16 weeks after start of treatment per modified response evaluation criteria in solid tumors (modified RECIST 1.1 [Demetri et al., 2013]) as a measure of disease control
- Two cohorts for second-line patients will be used: Cohort A: patients with secondary resistance mutations in other exons or no resistance mutations (as measured by liquid biopsy in circulating DNA); Cohort B: patients with evidence of secondary resistance mutations in exon 13 as assessed on progressing lesions or in circulating DNA
- One additional Cohort (Cohort C) will include heavily pretreated patients (failure of at least all approved lines of therapy) regardless of secondary mutation Secondary Objectives
- To assess progression-free survival (PFS) in each cohort and in the total patient population
- To assess objective response rate (ORR) in each cohort and in the total patient population
- To assess overall survival (OS) in each cohort and in the total patient population
- To evaluate the safety and tolerability of ponatinib in the total patient population
- To assess Quality of Life (QoL) Exploratory Objectives
- To assess limited elements of pharmacokinetics (PK) in the total patient population
- To explore the relationship between GIST genotype and CBR with ponatinib
- To explore the feasibility of detecting mutations in KIT and possibly other cancer-related genes using circulating nucleic acids derived from blood samples
- To explore the usefulness of "liquid biopsies" to predict treatment response and development of resistance
- To assess duration of follow-up treatment
Primary Endpoint
- CBR consisting of CR+PR+SD by modified RECIST 1.1 (Demetri et al., 2013) at 16 weeks in patients with imatinib-resistant GIST (KIT-mutant) with other or no resistance mutations (Cohort A) and secondary resistance mutation in exon 13 (Cohort B) Secondary Endpoints
- PFS in each cohort and in the total patient population
- ORR (CR + PR) in each cohort and in the total patient population
- OS in each cohort and in the total patient population
- Safety and tolerability of ponatinib
- QoL
- CBR of Cohort C Exploratory Endpoints
- Correlation of plasma levels of ponatinib and response
- Molecular genetic features of GIST at baseline and after treatment with ponatinib
- Correlation of tumor DNA from available paraffin tissue with genotypes of plasma sequencing ("liquid biopsies") and correlation of plasma genotype with treatment response, resistance and duration of follow-up treatment
- Conditions
- GIST, Malignant, KIT Exon 13 Mutation, KIT Gene Mutation
- Being tested
- Ponatinib 30 MG
- Keywords
- GIST, KIT secondary mutation, ponatinib
- Medical subject headings
- Gastrointestinal Stromal Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ponatinib 30 MGDrug
Also called Iclusiq
Ponatinib: once-daily oral dose of 30mg. A cycle of treatment is defined as 28 days. Doses may be reduced to manage drug-related adverse events and may be re-escalated once events resolve.
| Study group | Role | What they receive |
|---|---|---|
| Cohort A | Experimental | patients with primary c-KIT mutations and with either no detectable or non-exon13-secondary mutations (as measured by plasma sequencing); failure of imatinib treatment (only) Treatment with oral ponatinib 30MG (milligram) daily until progression or intolerable side effects. Receives: Drug: Ponatinib 30 MG |
| Cohort B | Experimental | GIST patients with primary c-KIT mutations and secondary c-KIT mutations in Exon 13; failure of imatinib treatment (only) Treatment with oral ponatinib 30MG daily until progression or intolerable side effects. Receives: Drug: Ponatinib 30 MG |
| Cohort C | Experimental | GIST patients with KIT-mutations and treatment failure of imatinib, sunitinib and regorafenib.
Treatment with oral ponatinib 30MG daily until progression or intolerable side effects. Receives: Drug: Ponatinib 30 MG |
What is being measured
The primary outcome is the main question the study is designed to answer.
clinical benefit rate (CBR)
CBR consisting of CR+PR+SD by modified RECIST 1.1 (Demetri et al., 2013) at 16 weeks in patients with imatinib-resistant GIST (KIT-mutant) with other or no resistance mutations (Cohort A) and secondary resistance mutation in exon 13 (Cohort B)
Measured: 16 weeks
Secondary outcomes (6)
Progression-free survival (PFS)
Assessment in each cohort and in the total patient population
Measured: through study completion, an average of 3.5 years
Objective response rate (ORR)
Assessment in each cohort and in the total patient population
Measured: 16 weeks
Overall survival (OS)
Assessment in each cohort and in the total patient population
Measured: through study completion, an average of 3.5 years
Assessment of treatment-related adverse events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Measured: 3.5 years
Quality of life assessment
Quality of life questionnaire SQLQ (Supplementary Quality of life questionnaire)
Measured: approx. 3.5 years (duration of study + 2 years follow-up period)
Fatigue assessment
Quality of life and fatigue questionnaire FACIT-F Version 4 (Functional Assessment of Chronic Illness Therapy-Fatigue)
Measured: approx. 3.5 years (duration of study + 2 years follow-up period)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2017
- Study start22 Mar 2017
- Primary completion22 Sep 2018 (estimated)
- Study completion22 Sep 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for GIST, MalignantStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 81 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sebastian Bauer
- Sponsor type
- University, hospital or other
- Collaborators
- Hannover Medical School, Helios Klinikum Berlin-Buch, University Hospital Tuebingen, Universitätsmedizin Mannheim, University Hospital, Aachen, Helios Klinikum Bad Saarow, WiSP GmbH
- Responsible party
- Sponsor-investigator, Sebastian Bauer, Universität Duisburg-Essen
- Investigators
- Sebastian Bauer, Prof. Dr., Principal investigator, University Hospital, Essen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03171389
- Sponsor's protocol ID
- POETIG
- Acronym
- POETIG
- Status last verified
- May 2017
- First submitted
- 25 Apr 2017
- Last updated
- 31 May 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Cortes JE, Kantarjian H, Shah NP, Bixby D, Mauro MJ, Flinn I, O'Hare T, Hu S, Narasimhan NI, Rivera VM, Clackson T, Turner CD, Haluska FG, Druker BJ, Deininger MW, Talpaz M. Ponatinib in refractory Philadelphia chromosome-positive leukemias. N Engl J Med. 2012 Nov 29;367(22):2075-88. doi: 10.1056/NEJMoa1205127.
PubMed 23190221Cortes JE, Kim DW, Pinilla-Ibarz J, le Coutre P, Paquette R, Chuah C, Nicolini FE, Apperley JF, Khoury HJ, Talpaz M, DiPersio J, DeAngelo DJ, Abruzzese E, Rea D, Baccarani M, Muller MC, Gambacorti-Passerini C, Wong S, Lustgarten S, Rivera VM, Clackson T, Turner CD, Haluska FG, Guilhot F, Deininger MW, Hochhaus A, Hughes T, Goldman JM, Shah NP, Kantarjian H; PACE Investigators. A phase 2 trial of ponatinib in Philadelphia chromosome-positive leukemias. N Engl J Med. 2013 Nov 7;369(19):1783-96. doi: 10.1056/NEJMoa1306494. Epub 2013 Nov 1.
PubMed 24180494Garner AP, Gozgit JM, Anjum R, Vodala S, Schrock A, Zhou T, Serrano C, Eilers G, Zhu M, Ketzer J, Wardwell S, Ning Y, Song Y, Kohlmann A, Wang F, Clackson T, Heinrich MC, Fletcher JA, Bauer S, Rivera VM. Ponatinib inhibits polyclonal drug-resistant KIT oncoproteins and shows therapeutic potential in heavily pretreated gastrointestinal stromal tumor (GIST) patients. Clin Cancer Res. 2014 Nov 15;20(22):5745-5755. doi: 10.1158/1078-0432.CCR-14-1397. Epub 2014 Sep 19.
PubMed 25239608George S, von Mehren M, Fletcher JA, Sun J, Zhang S, Pritchard JR, Hodgson JG, Kerstein D, Rivera VM, Haluska FG, Heinrich MC. Phase II Study of Ponatinib in Advanced Gastrointestinal Stromal Tumors: Efficacy, Safety, and Impact of Liquid Biopsy and Other Biomarkers. Clin Cancer Res. 2022 Apr 1;28(7):1268-1276. doi: 10.1158/1078-0432.CCR-21-2037.
PubMed 35091442
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Trial of Ponatinib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor (GIST) Following Failure or Intolerance of Prior Therapy With Imatinib
- ClinicalTrials.gov ID
- NCT03171389
- Acronym / sponsor ID
- POETIG / POETIG
- Conditions
- GIST, Malignant, KIT Exon 13 Mutation, KIT Gene Mutation
- Keywords
- GIST, KIT secondary mutation, ponatinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 81 participants (estimated)
- Lead sponsor
- Sebastian BauerUniversity, hospital or other
- Collaborators
- Hannover Medical School, Helios Klinikum Berlin-Buch, University Hospital Tuebingen, Universitätsmedizin Mannheim, University Hospital, Aachen, Helios Klinikum Bad Saarow, WiSP GmbH
- Study start
- 22 Mar 2017 (actual)
- Primary completion
- 22 Sep 2018 (estimated)
- Study completion
- 22 Sep 2020 (estimated)
- First posted
- 31 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a non-randomized, open-label, multicenter phase 2 study to evaluate the efficacy and safety of ponatinib in patients with metastatic and/or unresectable GIST after prior failure or intolerability of imatinib. Patients will be enrolled into 1 of 2 cohorts based on absence (Cohort A) or presence (Cohort B) of KIT exon 13 resistance mutations as measured by liquid biopsy. A third cohort (Cohort C) will include patients who have received all approved lines of TKI treatments (imatinib, sunitinib and regorafenib).
Detailed description
Primary Objective
- To assess clinical benefit of ponatinib in patients with KIT or PDGFRA mutant GIST defined as clinical benefit rate (CBR), which is the composite of complete response (CR), partial response (PR) and stable disease (SD) at ≥16 weeks after start of treatment per modified response evaluation criteria in solid tumors (modified RECIST 1.1 [Demetri et al., 2013]) as a measure of disease control
- Two cohorts for second-line patients will be used: Cohort A: patients with secondary resistance mutations in other exons or no resistance mutations (as measured by liquid biopsy in circulating DNA); Cohort B: patients with evidence of secondary resistance mutations in exon 13 as assessed on progressing lesions or in circulating DNA
- One additional Cohort (Cohort C) will include heavily pretreated patients (failure of at least all approved lines of therapy) regardless of secondary mutation Secondary Objectives
- To assess progression-free survival (PFS) in each cohort and in the total patient population
- To assess objective response rate (ORR) in each cohort and in the total patient population
- To assess overall survival (OS) in each cohort and in the total patient population
- To evaluate the safety and tolerability of ponatinib in the total patient population
- To assess Quality of Life (QoL) Exploratory Objectives
- To assess limited elements of pharmacokinetics (PK) in the total patient population
- To explore the relationship between GIST genotype and CBR with ponatinib
- To explore the feasibility of detecting mutations in KIT and possibly other cancer-related genes using circulating nucleic acids derived from blood samples
- To explore the usefulness of "liquid biopsies" to predict treatment response and development of resistance
- To assess duration of follow-up treatment
Primary Endpoint
- CBR consisting of CR+PR+SD by modified RECIST 1.1 (Demetri et al., 2013) at 16 weeks in patients with imatinib-resistant GIST (KIT-mutant) with other or no resistance mutations (Cohort A) and secondary resistance mutation in exon 13 (Cohort B) Secondary Endpoints
- PFS in each cohort and in the total patient population
- ORR (CR + PR) in each cohort and in the total patient population
- OS in each cohort and in the total patient population
- Safety and tolerability of ponatinib
- QoL
- CBR of Cohort C Exploratory Endpoints
- Correlation of plasma levels of ponatinib and response
- Molecular genetic features of GIST at baseline and after treatment with ponatinib
- Correlation of tumor DNA from available paraffin tissue with genotypes of plasma sequencing ("liquid biopsies") and correlation of plasma genotype with treatment response, resistance and duration of follow-up treatment
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort A | Experimental | Drug: Ponatinib 30 MGpatients with primary c-KIT mutations and with either no detectable or non-exon13-secondary mutations (as measured by plasma sequencing); failure of imatinib treatment (only) Treatment with oral ponatinib 30MG (milligram) daily until progression or intolerable side effects. |
| Cohort B | Experimental | Drug: Ponatinib 30 MGGIST patients with primary c-KIT mutations and secondary c-KIT mutations in Exon 13; failure of imatinib treatment (only) Treatment with oral ponatinib 30MG daily until progression or intolerable side effects. |
| Cohort C | Experimental | Drug: Ponatinib 30 MGGIST patients with KIT-mutations and treatment failure of imatinib, sunitinib and regorafenib. Treatment with oral ponatinib 30MG daily until progression or intolerable side effects. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Male or female patients ≥18 years old
- GIST with failure or intolerance to imatinib or failure / intolerance to all three approved TKIs defined as:
- Histologically confirmed metastatic and/or unresectable GIST (harboring a primary KIT or PDGFRA-mutation) after failure or intolerance of imatinib (cohort A and B) or all three approved TKIs (cohort C). If prior TKI treatment was neoadjuvant therapy, then relapse must have occurred during the neoadjuvant therapy in order to consider it failed therapy
- Patients in Cohort A must have evidence of clinical resistance mutations in any other exon or no resistance mutation but evidence of progression by CT or MRI imaging. Patients in Cohort B must have evidence of an activating resistance mutation in KIT exon 13 (by direct sequencing of progressing lesions or by liquid biopsy).
- Measurable disease per modified RECIST 1.1 (Demetri et al., 2013). A lesion in a previously irradiated area is eligible to be considered as measurable disease as long as there is objective evidence of progression of the lesion prior to study enrollment
Key exclusion criteria (18 listed)
- Patients lacking primary mutations of KIT or PDGFRA (including Succinate-Dehydrogenase(SDH)-deficient GIST)
- Major surgery within 28 days prior to initiating therapy
- History of bleeding disorder
- History of acute pancreatitis within 1 year of study or history of chronic pancreatitis
- History of alcohol and /or drug abuse
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ponatinib 30 MGAlso: IclusiqPonatinib: once-daily oral dose of 30mg. A cycle of treatment is defined as 28 days. Doses may be reduced to manage drug-related adverse events and may be re-escalated once events resolve. | Drug | Cohort A, Cohort B, Cohort C |
Primary outcome
clinical benefit rate (CBR)
Time frame: 16 weeks
CBR consisting of CR+PR+SD by modified RECIST 1.1 (Demetri et al., 2013) at 16 weeks in patients with imatinib-resistant GIST (KIT-mutant) with other or no resistance mutations (Cohort A) and secondary resistance mutation in exon 13 (Cohort B)
6 secondary outcomes
Progression-free survival (PFS)
Time frame: through study completion, an average of 3.5 years
Objective response rate (ORR)
Time frame: 16 weeks
Overall survival (OS)
Time frame: through study completion, an average of 3.5 years
Assessment of treatment-related adverse events
Time frame: 3.5 years
Quality of life assessment
Time frame: approx. 3.5 years (duration of study + 2 years follow-up period)
Fatigue assessment
Time frame: approx. 3.5 years (duration of study + 2 years follow-up period)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2017.
- Background: PubMed 23190221Cortes JE, Kantarjian H, Shah NP, Bixby D, Mauro MJ, Flinn I, O'Hare T, Hu S, Narasimhan NI, Rivera VM, Clackson T, Turner CD, Haluska FG, Druker BJ, Deininger MW, Talpaz M. Ponatinib in refractory Philadelphia chromosome-positive leukemias. N Engl J Med. 2012 Nov 29;367(22):2075-88. doi: 10.1056/NEJMoa1205127.
- Background: PubMed 24180494Cortes JE, Kim DW, Pinilla-Ibarz J, le Coutre P, Paquette R, Chuah C, Nicolini FE, Apperley JF, Khoury HJ, Talpaz M, DiPersio J, DeAngelo DJ, Abruzzese E, Rea D, Baccarani M, Muller MC, Gambacorti-Passerini C, Wong S, Lustgarten S, Rivera VM, Clackson T, Turner CD, Haluska FG, Guilhot F, Deininger MW, Hochhaus A, Hughes T, Goldman JM, Shah NP, Kantarjian H; PACE Investigators. A phase 2 trial of ponatinib in Philadelphia chromosome-positive leukemias. N Engl J Med. 2013 Nov 7;369(19):1783-96. doi: 10.1056/NEJMoa1306494. Epub 2013 Nov 1.
- Background: PubMed 25239608Garner AP, Gozgit JM, Anjum R, Vodala S, Schrock A, Zhou T, Serrano C, Eilers G, Zhu M, Ketzer J, Wardwell S, Ning Y, Song Y, Kohlmann A, Wang F, Clackson T, Heinrich MC, Fletcher JA, Bauer S, Rivera VM. Ponatinib inhibits polyclonal drug-resistant KIT oncoproteins and shows therapeutic potential in heavily pretreated gastrointestinal stromal tumor (GIST) patients. Clin Cancer Res. 2014 Nov 15;20(22):5745-5755. doi: 10.1158/1078-0432.CCR-14-1397. Epub 2014 Sep 19.
- Derived from this study: PubMed 35091442George S, von Mehren M, Fletcher JA, Sun J, Zhang S, Pritchard JR, Hodgson JG, Kerstein D, Rivera VM, Haluska FG, Heinrich MC. Phase II Study of Ponatinib in Advanced Gastrointestinal Stromal Tumors: Efficacy, Safety, and Impact of Liquid Biopsy and Other Biomarkers. Clin Cancer Res. 2022 Apr 1;28(7):1268-1276. doi: 10.1158/1078-0432.CCR-21-2037.
- Iclusiq (ponatinib) prescribing informationLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sebastian Bauer, Prof. Dr. principal investigator
University Hospital, Essen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ponatinib 30 MG in people with GIST, Malignant and KIT Exon 13 Mutation, run by Sebastian Bauer at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 31 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Johanna Falkenhorst (+49 201 723 84150, johanna.falkenhorst@uk-essen.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
clinical benefit rate (CBR) (time frame: 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03171389, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 May 2017.