GISTNCT03862885
Early Access Program (EAP) for Avapritinib in Patients With Locally Advanced Unresectable or Metastatic GIST
A study testing Avapritinib in people with GIST, run by Blueprint Medicines Corporation.
Trial summary
This is a US, multicenter, open-label expanded access program to provide access to avapritinib until such time that avapritinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- GIST
- What is being tested?
- Avapritinib
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 16 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Patient is ≥ 16 years of age.
- Diagnosis of unresectable or metastatic GIST
- The patient is not eligible for an ongoing study of avapritinib or cannot access an ongoing study of avapritinib.
- Patient has received 3 or more TKI therapies including imatinib, or the patient has GIST that carries a mutation in exon 18 of the PDGFRA gene (such as D842V).
- The patient has adequate vital organ function, including heart, lungs, liver, kidneys, bone marrow and endocrine, and is expected to tolerate therapy with a TKI.
- Patient or legal guardian, if permitted by local regulatory authorities, provides informed consent.
Who cannot join
- Patients who have poor organ function as defined by one or more of the following laboratory parameters;
- Total bilirubin > 2 × ULN; or > 3 × ULN in the presence of Gilbert's Disease;
- Platelet count < 75 × 10^9/L.
- Patient requires therapy with a concomitant medication that is a strong inhibitor or strong inducer of cytochrome P450 (CYP) 3A4
- Patient has had a major surgical procedure (minor surgical procedures such as central venous catheter placement, tumor needle biopsy, and feeding tube placement are not considered major surgical procedures) within 14 days of the first dose of avapritinib.
- Patient has a history of a cerebrovascular accident or transient ischemic attacks within 1 year prior to the first dose of program drug.
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- Patient has a known risk of intracranial bleeding, such as a brain aneurysm or history of subdural or subarachnoid bleeding.
- Women who are unwilling, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of randomization and for at least 30 days after the last dose of avapritinib. Men who are unwilling, if not surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of first dose and for at least 90 days after the last dose of avapritinib.
- Women who are pregnant, as documented by a serum beta human chorionic gonadotropin (β-hCG) pregnancy test consistent with pregnancy, obtained within 7 days before the randomization. Females with β-hCG values that are within the range for pregnancy but are not pregnant (false-positives) may be enrolled with written consent of the Sponsor, after pregnancy has been ruled out. Females of non- childbearing potential (postmenopausal for more than 1 year; bilateral tubal ligation; bilateral oophorectomy; hysterectomy) do not require a serum β-hCG test.
- Women who are breast feeding.
About this study
Official title: Early Access Program (EAP) for Avapritinib in Patients With Locally Advanced Unresectable or Metastatic Gastrointestinal Stromal Tumor (GIST)
This is a US, multicenter, open-label expanded access program to provide access to avapritinib until such time that avapritinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.
- Conditions
- GIST
- Being tested
- Avapritinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvapritinibDrug
Also called BLU-285
Avapritinib will be administered orally (PO) at a dose of 300 mg once daily (QD) in continuous 28-day cycles.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GISTStudies 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03862885
- Sponsor's protocol ID
- BLU-285-EAP-01
- Status last verified
- Jul 2020
- First submitted
- 1 Mar 2019
- Last updated
- 27 Jul 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Access Program (EAP) for Avapritinib in Patients With Locally Advanced Unresectable or Metastatic Gastrointestinal Stromal Tumor (GIST)
- ClinicalTrials.gov ID
- NCT03862885
- Acronym / sponsor ID
- BLU-285-EAP-01
- Conditions
- GIST
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- First posted
- 5 Mar 2019 (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 US, multicenter, open-label expanded access program to provide access to avapritinib until such time that avapritinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.
Study design
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Patient is ≥ 16 years of age.
- Diagnosis of unresectable or metastatic GIST
- The patient is not eligible for an ongoing study of avapritinib or cannot access an ongoing study of avapritinib.
- Patient has received 3 or more TKI therapies including imatinib, or the patient has GIST that carries a mutation in exon 18 of the PDGFRA gene (such as D842V).
- The patient has adequate vital organ function, including heart, lungs, liver, kidneys, bone marrow and endocrine, and is expected to tolerate therapy with a TKI.
Key exclusion criteria (10 listed)
- Patients who have poor organ function as defined by one or more of the following laboratory parameters;
- Patient requires therapy with a concomitant medication that is a strong inhibitor or strong inducer of cytochrome P450 (CYP) 3A4
- Patient has had a major surgical procedure (minor surgical procedures such as central venous catheter placement, tumor needle biopsy, and feeding tube placement are not considered major surgical procedures) within 14 days of the first dose of avapritinib.
- Patient has a history of a cerebrovascular accident or transient ischemic attacks within 1 year prior to the first dose of program drug.
- Patient has a known risk of intracranial bleeding, such as a brain aneurysm or history of subdural or subarachnoid bleeding.
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 |
|---|---|---|
| AvapritinibAlso: BLU-285Avapritinib will be administered orally (PO) at a dose of 300 mg once daily (QD) in continuous 28-day cycles. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2020.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Avapritinib in people with GIST, run by Blueprint Medicines Corporation.
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 6 inclusion and 10 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 27 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
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 27 Jul 2020.