A Phase 3 interventional study of avapritinib and regorafenib in GIST, sponsored by Blueprint Medicines Corporation. Completed at 114 sites in 18 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-10-06.
Sponsored by Blueprint Medicines Corporation (part of Sanofi) · Phase 3, Interventional, and Treatment
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Among 139 studies in Gastrointestinal Stromal Tumors with a site in United States — 19 open to participants now, 29 under way, 5 of them phase 3. Gastrointestinal Stromal Tumors · United States.
Among the 139 studies in Gastrointestinal Stromal Tumors with a site in United States, this one is the 7th largest by enrollment of the 116 interventional ones that give a count, is one of 4 that started in 2018, is one of 19 in phase 3, and was the 103rd registered.
343 studies on the registry are indexed under Gastrointestinal Stromal Tumors; 61 are open to participants now.
Browse Gastrointestinal Stromal Tumors studies →19 of the 139 studies in Gastrointestinal Stromal Tumors with a site in United States are open now. Recruiting first, the newest first:
GlaxoSmithKline · Sites in United States, Argentina, Australia and 17 more countries
Ages 18+
Kumquat Biosciences Inc. · Sites in United States
Ages 18+
Dana-Farber Cancer Institute · Sites in United States
Ages 18+
University of California, San Diego · Sites in United States
Ages 18+
GlaxoSmithKline · Sites in United States, Australia, Belgium and 16 more countries
Ages 18+
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 49 Gastrointestinal Stromal Tumors studies recruiting now, 38 (78%) set no upper age limit and 4 (8%) stop at 75 or younger; 6 take children under 18; 2 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
139 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 46 · Phase 2 63 · Phase 3 19 · Phase 4 2
First in 1997; this study’s year is marked. 4 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 476 is above the median of 50 across 252 interventional studies indexed under Gastrointestinal Stromal Tumors.
Median enrollment 55 across the 116 interventional studies, 120 across the 14 observational. Of the 105 that completed or were terminated, 49 (47%) have results posted.
This tab as a report to print or save as PDF → With an account.
62 of 139 have an industry lead sponsor. The 10 most frequent:
This study is registered under Blueprint Medicines Corporation, one of the names counted as Sanofi. Under that name alone:
Blueprint Medicines Corporation is the lead sponsor of 32 studies on the registry; 6 are open to participants now.
Of its 14 completed or terminated interventional studies of FDA-regulated products, 3 (21%) have results posted.
Sanofi has 5 studies filed under Gastrointestinal Stromal Tumors — fewer than ten, so there is no comparison to draw.
7 of the studies in Gastrointestinal Stromal Tumors with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT01991379Memorial Sloan Kettering Cancer CenterMEK162 in Combination With Imatinib Mesylate in Patients With Untreated Advanced Gastrointestinal Stromal Tumor (GIST) | 2026-11 |
| NCT05366816University of MiamictDNA-Guided Sunitinib And Regorafenib Therapy for GIST | 2026-12-31 |
| NCT05957367Deciphera Pharmaceuticals, LLCA Study of Inlexisertib (DCC-3116) in Combination With Anticancer Therapies in Participants With Advanced Malignancies | 2027-03 |
| NCT05489237IDRX, Inc., a wholly owned subsidiary of GSK, LLCFirst-in-human Study of IDRX-42 in Participants With Metastatic and/or Unresectable Gastrointestinal Stromal Tumors | 2027-03-10 |
| NCT04595747National Cancer Institute (NCI)Testing the Anti-cancer Drug, Rogaratinib (BAY 1163877), for Treatment of Advanced Sarcoma With Alteration in Fibroblast Growth Factor Receptor (FGFR 1-4), and in Patients With SDH-deficient Gastrointestinal Stromal Tumor (GIST) | 2027-07-31 |
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Among the 139 studies in Gastrointestinal Stromal Tumors with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This is an open-label, randomized, Phase 3 study in patients with locally advanced unresectable or metastatic GIST (advanced GIST) of avapritinib (also known as BLU-285) versus regorafenib in patients previously treated with imatinib and 1 or 2 other TKIs.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
300 mg PO QD
Drug: avapritinib
160 mg PO QD
Drug: regorafenib
Avapritinib tablets for oral administration. Avapritinib will be dosed at 300 mg once daily, continuously.
Also known as: BLU-285
Regorafenib tablets for oral administration. Regorafenib will be dosed at 160 mg once daily for 3 weeks out of every 4 weeks (ie. 3 weeks on/1 week off).
Also known as: Stivarga
Efficacy of Avapritinib Based on Progression-free Survival (PFS) Determined by Central Radiological Assessment Per Modified Response Evaluation Criteria in Solid Tumors (mRECIST), Version 1.1
To demonstrate the efficacy of avapritinib based on progression-free survival (PFS) determined by central radiological assessment per modified Response Evaluation Criteria in Solid Tumors (mRECIST), version 1.1 in patients with advanced GIST following 2 or 3 regimens of prior treatment with a tyrosine kinase inhibitor (TKI), including imatinib, compared to patients treated with regorafenib. A progressively growing tumor must meet the following criteria: a) the target lesions must be greater or equal to 2cm in size and be a new GIST active lesion or b) the target lesions must be expanding on at least 2 sequential imaging studies.
Time frame: 24 Months
Objective Response Rate (ORR) Determined by Central Radiology Assessment Per mRECIST, Version 1.1
To evaluate objective response rate (ORR) determined by central radiology assessment per mRECIST, version 1.1 in patients with advanced GIST treated with avapritinib compared to patients treated with regorafenib. A complete response (CR) per modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) is defined as complete disappearance of all target lesions. A partial response (PR) is defined as at least 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum of diameters. Overall Response (OR) = CR + PR
Time frame: 24 Months
Overall Survival (OS) in Patients With Advanced GIST Treated With Avapritinib Compared to Patients Treated With Regorafenib
To evaluate overall survival (OS) in patients with advanced GIST treated with avapritinib compared to patients treated with regorafenib
Time frame: 24 Months
European Organisation for Research and Treatment of Cancer Quality of Life (EORTC-QLQ-30). Change in Individual Scores in Patients With Advanced GIST Treated With Avapritinib Compared to Patients Treated With Regorafenib
The Global Health Status Score is derived from question 29 and 30 on the EORTC-QLQ-C30 tool. The change in score was assessed between baseline and week 12 in patients treated with advanced GIST treated with avapritinib compared to patients treated with regorafenib. The Global Health Status Score score range is 0 to 100 with a higher score indicating better global health status. A positive change indicates improvement in global health status.
Time frame: Difference between baseline and week 12 of treatment
| Milestone | Avapritinib | Regorafinib |
|---|---|---|
| Started | 240 | 236 |
| Completed | 0 | 0 |
| Not completed | 240 | 236 |
| Withdrew: Withdrawal by subject | 21 | 14 |
| Withdrew: Death | 89 | 87 |
| Withdrew: Lost to follow-up | 5 | 3 |
| Withdrew: Administrative/other | 33 | 32 |
| Withdrew: Not treated | 1 | 2 |
| Withdrew: Sponsor decision | 91 | 98 |
To demonstrate the efficacy of avapritinib based on progression-free survival (PFS) determined by central radiological assessment per modified Response Evaluation Criteria in Solid Tumors (mRECIST), version 1.1 in patients with advanced GIST following 2 or 3 regimens of prior treatment with a tyrosine kinase inhibitor (TKI), including imatinib, compared to patients treated with regorafenib. A progressively growing tumor must meet the following criteria: a) the target lesions must be greater or equal to 2cm in size and be a new GIST active lesion or b) the target lesions must be expanding on at least 2 sequential imaging studies.
| months | Avapritinib | Regorafinib |
|---|---|---|
| Efficacy of Avapritinib Based on Progression-free Survival (PFS) Determined by Central Radiological Assessment Per Modified Response Evaluation Criteria in Solid Tumors (mRECIST), Version 1.1 | 4.2 (3.7 to 5.6) | 5.6 (3.8 to 7.2) |
To evaluate objective response rate (ORR) determined by central radiology assessment per mRECIST, version 1.1 in patients with advanced GIST treated with avapritinib compared to patients treated with regorafenib. A complete response (CR) per modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) is defined as complete disappearance of all target lesions. A partial response (PR) is defined as at least 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum of diameters. Overall Response (OR) = CR + PR
| Participants | Avapritinib | Regorafinib |
|---|---|---|
| Responder | 41 | 17 |
| Non-Responder | 199 | 219 |
To evaluate overall survival (OS) in patients with advanced GIST treated with avapritinib compared to patients treated with regorafenib
| months | Avapritinib | Regorafinib |
|---|---|---|
| Overall Survival (OS) in Patients With Advanced GIST Treated With Avapritinib Compared to Patients Treated With Regorafenib | 19.2 (19.2 to NA) | 17.4 (15.8 to NA) |
The Global Health Status Score is derived from question 29 and 30 on the EORTC-QLQ-C30 tool. The change in score was assessed between baseline and week 12 in patients treated with advanced GIST treated with avapritinib compared to patients treated with regorafenib. The Global Health Status Score score range is 0 to 100 with a higher score indicating better global health status. A positive change indicates improvement in global health status.
| scores on a scale | Avapritinib | Regorafinib |
|---|---|---|
| European Organisation for Research and Treatment of Cancer Quality of Life (EORTC-QLQ-30). Change in Individual Scores in Patients With Advanced GIST Treated With Avapritinib Compared to Patients Treated With Regorafenib | -5.7 ± 24.29 | -4.4 ± 20.74 |
Collected over From the date of first study drug administration to 30 days after last study drug administration, approximately 6 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Avapritinib | 89/239 (37.2%) | 106/239 (44.4%) | 218/239 (91.2%) |
| Regorafinib | 87/234 (37.2%) | 91/234 (38.9%) | 224/234 (95.7%) |
| Event | Avapritinib | Regorafinib |
|---|---|---|
| AnaemiaBlood and lymphatic system disorders | 27/239 | 7/234 |
| Abdominal painGastrointestinal disorders | 13/239 | 11/234 |
| PyrexiaGeneral disorders | 4/239 | 7/234 |
| Gastrointestinal haemorrhageGastrointestinal disorders | 6/239 | 5/234 |
| DiarrhoeaGastrointestinal disorders | 4/239 | 5/234 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 3/239 | 5/234 |
| AscitesGastrointestinal disorders | 5/239 | 2/234 |
| VomitingGastrointestinal disorders | 5/239 | 2/234 |
| SepsisInfections and infestations | 5/239 | 0/234 |
| Acute kidney injuryRenal and urinary disorders | 4/239 | 4/234 |
| Event | Avapritinib | Regorafinib |
|---|---|---|
| Palmar-plantar erythrodysaesthesia syndromeSkin and subcutaneous tissue disorders | 4/239 | 143/234 |
| DysphoniaRespiratory, thoracic and mediastinal disorders | 8/239 | 71/234 |
| Periorbital oedemaEye disorders | 68/239 | 0/234 |
| Oedema peripheralGeneral disorders | 55/239 | 13/234 |
| Weight decreasedInvestigations | 24/239 | 53/234 |
| Lacrimation increasedEye disorders | 45/239 | 0/234 |
| HeadacheNervous system disorders | 29/239 | 43/234 |
| White blood cell count decreasedInvestigations | 42/239 | 7/234 |
| StomatitisGastrointestinal disorders | 7/239 | 40/234 |
| AlopeciaSkin and subcutaneous tissue disorders | 24/239 | 38/234 |
| Age, Continuous(years) | Avapritinib | Regorafinib | Total |
|---|---|---|---|
| Mean | 61.1 ± 10.96 | 61.0 ± 10.74 | 61.1 ± 10.84 |
| Sex: Female, Male(Participants) | Avapritinib | Regorafinib | Total |
|---|---|---|---|
| Female | 78 | 80 | 158 |
| Male | 162 | 156 | 318 |
| Race (NIH/OMB)(Participants) | Avapritinib | Regorafinib | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 64 | 64 | 128 |
| Native Hawaiian or Other Pacific Islander | 1 | 1 | 2 |
| Black or African American | 9 | 5 | 14 |
| White | 139 | 143 | 282 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 27 | 23 | 50 |
| Region of Enrollment(participants) | Avapritinib | Regorafinib | Total |
|---|---|---|---|
| Singapore | 2 | 2 | 4 |
| Hungary | 2 | 1 | 3 |
| United States | 71 | 62 | 133 |
| Czechia | 3 | 1 | 4 |
| United Kingdom | 12 | 12 | 24 |
| Spain | 14 | 11 | 25 |
| Canada | 2 | 6 | 8 |
| Austria | 0 | 1 | 1 |
| Netherlands | 2 | 6 | 8 |
| South Korea | 23 | 20 | 43 |
| Sweden | 6 | 7 | 13 |
| Belgium | 1 | 1 | 2 |
| China | 35 | 39 | 74 |
| Poland | 10 | 9 | 19 |
| Italy | 13 | 15 | 28 |
| Australia | 5 | 5 | 10 |
| France | 22 | 20 | 42 |
| Germany | 17 | 18 | 35 |
| Body Mass Index (BMI)(kilogram per meter square) | Avapritinib | Regorafinib | Total |
|---|---|---|---|
| Mean | 25.50 ± 5.563 | 24.69 ± 5.163 | 25.10 ± 5.378 |
Showing the first 100 of 114 sites across 18 countries. Choose a country and city above to see the rest.
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Mar 2018 to primary completion in Mar 2020, 23 months.
Results were first posted in May 2021, 1 year 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Sep 2022. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Avapritinib
Gastrointestinal Stromal Tumors →
Blueprint Medicines Corporation →