Gastrointestinal Stromal TumorsPhase 1NCT04908176
A Drug-drug Interaction Study of Avapritinib and Midazolam
A Phase 1 study testing Avapritinib and midazolam in people with Gastrointestinal Stromal Tumors and GIST, run by Blueprint Medicines Corporation at 3 sites in United States.
Trial summary
The purpose of this study is to investigate the effect of multiple dosing of avapritinib on the pharmacokinetics (PK) of midazolam in adult patients with metastatic or unresectable gastrointestinal stromal tumors (GIST), recurrent gliomas, or other KIT mutant tumors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gastrointestinal Stromal Tumors, GIST, Non-resectable Advanced Solid Tumors
- What is being tested?
- Avapritinib and midazolam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 3 sitesUnited States
- Study dates
- 24 Aug 2022to 10 May 2024
- Phase
- Phase 1
- 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
- Must be ≥18 years of age at the time of signing the informed consent
- Confirmed diagnosis of
- metastatic or unresectable KIT mutant GIST that has recurred or progressed after at least 4 lines of prior systemic SOC therapy or the Investigator has determined that treatment with SOC therapy is not appropriate for patients who failed at least 2 lines of prior SOC
- OR
- ---Non-resectable advance solid tumor with KIT mutation with progression following standard of care treatment.
- OR
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- ---Confirmed diagnosis of recurrent or unresectable CNS tumors including :IDH-mutant astrocytoma, IDH-mutant oligodendroglioma, glioblastoma, H3K27-altered diffuse midline glioma, H3G34-mutant diffuse hemispheric glioma, midline glioma (with unknown H3K27 mutation status) that has failed prior radiation or systemic SOC therapy.
- Must be able to swallow an oral medication
- Must have Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
- Patient agrees to use contraception consistent with local regulations
- Must provide signed informed consent to participate in the study
Who cannot join
- Patients with GIST that harbors a known PDGFRA mutation
- Known hypersensitivity to avapritinib, midazolam, or any of their excipients
- Have received previous therapy with avapritinib
- Have any of the following laboratory abnormalities before the first dose of study drug:
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >3 × upper limit of normal (ULN) if no hepatic metastases are present; >5 × ULN if hepatic metastases are present
- Total bilirubin >1.5 × ULN; >3 × ULN in the presence of Gilbert's Disease
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- Estimated (Cockcroft-Gault formula) or measured creatinine clearance <60 mL/min
- Platelet count <100 × 10^9/liter (L)
- Absolute neutrophil count (ANC) <1.0 × 10^9/L
- Hemoglobin <9 grams per deciliter (g/dL). Transfusion and erythropoietin may be used to reach at least 9 g/dL but must have been administered at least 2 weeks before the first dose of the study drug.
- Require therapy with a concomitant medication that is a strong and moderate CYP3A4 inhibitors or inducers
- Consumption of any nutrients known to modulate CYP3A4 enzymes activity (eg, grapefruit or grapefruit juice, pomelo juice, star fruit, or Seville [blood] orange and derivative products, cruciferous vegetables [eg, broccoli, cauliflower, cabbage, brussel sprouts]) within 14 days before screening and during the study until the end of the Main Treatment Period
- Have received a prior anticancer drug less than 5 half-lives or 14 days (whichever is shorter) before screening
- Have had a major surgical procedure within 14 days of the first dose of study drug or have significant traumatic injury within 28 days before screening
- Have history of a cerebrovascular accident or transient ischemic attacks within 1 year before screening
- Have known risk of intracranial bleeding, such as a brain aneurysm or history of subdural or subarachnoid bleeding
- Have corrected QT interval using Fridericia's formula (QTcF) >450 msec
- Have clinically significant, uncontrolled, cardiovascular disease, including congestive heart failure Grades 2, 3, or 4 according to the New York Heart Association classification, myocardial infarction, or unstable angina within the previous 6 months, or uncontrolled hypertension
- Have experienced any hemorrhage or bleeding event National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0 Grade ≥3 within 4 weeks before screening. Exceptions are patients with primary CNS tumors who are eligible if the Grade ≥3 bleeding event was in the CNS and it occurred 2 weeks or more prior to the first dose of avapritinib.
- Patients who have a symptomatic nonhealing wound, ulcer, GI perforation, or bone fracture
- Have received organ or allogenic bone marrow or peripheral blood stem cell transplant
- Have known diagnosis of human immunodeficiency virus infection or active viral hepatitis; viral testing is not required
- History of alcohol consumption exceeding 2 standard drinks per day on average (1 standard drink = 14 grams of alcohol). Alcohol consumption will be prohibited 48 hours before screening and throughout the entire the Main Treatment Period
- Use of tobacco- or nicotine-containing products within 3 months of enrollment
- Is a female patient who is unwilling, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of informed consent and for until at least 6 weeks after the last dose of study drug. Males who are unwilling, if not surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of informed consent and for at least 6 weeks after the last dose of study drug.
- Is a female patient who is pregnant, as documented by a serum beta human chorionic gonadotropin (β-hCG) pregnancy test consistent with pregnancy obtained within 7 days before the first dose of study drug. Patients 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 nonchildbearing potential (postmenopausal for more than 12 months, bilateral tubal ligation, bilateral oophorectomy, or hysterectomy) do not require a serum β-hCG test.
- Female who is breastfeeding
- Have a prior or ongoing clinically significant illness, medical condition, surgical history, physical finding, or laboratory abnormality that, in the Investigator's opinion, could affect the safety of the patient, alter the absorption, distribution, metabolism or excretion of the study drugs, or impair the assessment of study results.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Michigan
Ann Arbor, Michigan, 48103
Mayo Clinic Florida
Jacksonville, Florida, 32224
Thomas Jefferson University, Sidney Kimmel Cancer Center
Philadelphia, Pennsylvania, 19107
About this study
Official title: A Drug-drug Interaction Study to Investigate the Effect of Avapritinib on the Pharmacokinetics of Midazolam in Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumors (GIST) and Other Advanced Solid Tumors
The purpose of this study is to investigate the effect of multiple dosing of avapritinib on the pharmacokinetics (PK) of midazolam in adult patients with metastatic or unresectable gastrointestinal stromal tumors (GIST), recurrent gliomas, or other KIT mutant tumors.
- Conditions
- Gastrointestinal Stromal Tumors, GIST, Non-resectable Advanced Solid Tumors, Recurrent or Unresectable Central Nervous System (CNS) Tumors
- Being tested
- Avapritinib, midazolam
- Keywords
- Avapritinib, Midazolam, GIST, BLU-285, KIT, IDH-mutant astrocytoma, IDH-mutant oligodendroglioma, glioblastoma, H3K27-altered diffuse midline glioma, H3G34-mutant diffuse hemispheric glioma, midline glioma
- Medical subject headings
- Gastrointestinal Stromal Tumors, Recurrence, Neoplasms, Glioblastoma
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 tablets
midazolamDrug
midazolam syrup
| Study group | Role | What they receive |
|---|---|---|
| Participants with metastatic, unresectable GIST, non-CNS solid tumors, or CNS tumors | Experimental | Participants will receive 5 mg of midazolam orally on Day 1 and Day 17. Participants will receive avapritinib 300 mg daily, orally on starting on Day 3. Participants with CNS tumors will receive avapritinib 300 mg daily orally, until Day 56. Receives: Drug: Avapritinib; Drug: midazolam |
What is being measured
The primary outcome is the main question the study is designed to answer.
maximum plasma concentration (Cmax) of midazolam
Measured: Day 1 and Day 18
time of maximum plasma concentration (tmax) of midazolam
Measured: Day 1 and Day 18
area under the plasma concentration-time curve (AUC) of midazolam
Measured: Day 1 and Day 18
Secondary outcomes (5)
Number of adverse events (AEs), serious AEs (SAEs),
Measured: up to approximately 2 months
Cmax at steady state (Cmax, ss) of avapritinib
Measured: Day 18
Cmax at steady state (Cmax, ss) of metabolite
Measured: Day 18
area under the plasma concentration-time curve at steady state (AUC,ss) of avapritinib
Measured: Day 18
area under the plasma concentration-time curve at steady state (AUC,ss) of metabolite
Measured: Day 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2021
- Study start24 Aug 2022
- Primary completion1 Mar 2024
- Study completion10 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gastrointestinal Stromal TumorsStudies 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 1
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04908176
- Sponsor's protocol ID
- BLU-285-1107
- Status last verified
- Dec 2024
- First submitted
- 27 May 2021
- Last updated
- 13 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Drug-drug Interaction Study to Investigate the Effect of Avapritinib on the Pharmacokinetics of Midazolam in Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumors (GIST) and Other Advanced Solid Tumors
- ClinicalTrials.gov ID
- NCT04908176
- Acronym / sponsor ID
- BLU-285-1107
- Conditions
- Gastrointestinal Stromal Tumors, GIST, Non-resectable Advanced Solid Tumors, Recurrent or Unresectable Central Nervous System (CNS) Tumors
- Keywords
- Avapritinib, Midazolam, GIST, BLU-285, KIT, IDH-mutant astrocytoma, IDH-mutant oligodendroglioma, glioblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Study start
- 24 Aug 2022 (actual)
- Primary completion
- 1 Mar 2024 (actual)
- Study completion
- 10 May 2024 (actual)
- First posted
- 1 Jun 2021 (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.
The purpose of this study is to investigate the effect of multiple dosing of avapritinib on the pharmacokinetics (PK) of midazolam in adult patients with metastatic or unresectable gastrointestinal stromal tumors (GIST), recurrent gliomas, or other KIT mutant tumors.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with metastatic, unresectable GIST, non-CNS solid tumors, or CNS tumors | Experimental | Drug: Avapritinib, Drug: midazolamParticipants will receive 5 mg of midazolam orally on Day 1 and Day 17. Participants will receive avapritinib 300 mg daily, orally on starting on Day 3. Participants with CNS tumors will receive avapritinib 300 mg daily orally, until Day 56. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Must be ≥18 years of age at the time of signing the informed consent
- Confirmed diagnosis of
- OR
- ---Non-resectable advance solid tumor with KIT mutation with progression following standard of care treatment.
- OR
Key exclusion criteria (28 listed)
- Patients with GIST that harbors a known PDGFRA mutation
- Known hypersensitivity to avapritinib, midazolam, or any of their excipients
- Have received previous therapy with avapritinib
- Have any of the following laboratory abnormalities before the first dose of study drug:
- Require therapy with a concomitant medication that is a strong and moderate CYP3A4 inhibitors or inducers
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 tablets | Drug | Participants with metastatic, unresectable GIST, non-CNS solid tumors, or CNS tumors |
| midazolammidazolam syrup | Drug | Participants with metastatic, unresectable GIST, non-CNS solid tumors, or CNS tumors |
Primary outcome
maximum plasma concentration (Cmax) of midazolam
Time frame: Day 1 and Day 18
time of maximum plasma concentration (tmax) of midazolam
Time frame: Day 1 and Day 18
area under the plasma concentration-time curve (AUC) of midazolam
Time frame: Day 1 and Day 18
5 secondary outcomes
Number of adverse events (AEs), serious AEs (SAEs),
Time frame: up to approximately 2 months
Cmax at steady state (Cmax, ss) of avapritinib
Time frame: Day 18
Cmax at steady state (Cmax, ss) of metabolite
Time frame: Day 18
area under the plasma concentration-time curve at steady state (AUC,ss) of avapritinib
Time frame: Day 18
area under the plasma concentration-time curve at steady state (AUC,ss) of metabolite
Time frame: Day 18
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2024.
- Individual participant dataSharing planned: No
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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.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Avapritinib and midazolam in people with Gastrointestinal Stromal Tumors and GIST, run by Blueprint Medicines Corporation at 3 sites in United States.
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 11 inclusion and 28 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: finished, as of the sponsor's last update on 13 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
What does the primary outcome measure?
maximum plasma concentration (Cmax) of midazolam (time frame: Day 1 and Day 18). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
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 13 Dec 2024.