Brain TumorEarly Phase 1NCT07039760
Asciminib With or Without Sildenafil for Brain Tumors
A Early Phase 1 study testing Asciminib, Sildenafil and Surgical resection or biopsy in people with Brain Tumor, run by Washington University School of Medicine at 1 site in United States.
Trial summary
Dissemination of medulloblastoma is an independent risk factor of poor prognosis. Dissemination of medulloblastoma at recurrence is nearly universally fatal. ABL1 and 2 have been recently found to mediate the dissemination of medulloblastoma. Read more about this study
- Who is it for?
- Ages 18 to 39 years
- Conditions the study is for
- Brain Tumor
- What is being tested?
- Asciminib, Sildenafil and Surgical resection or biopsy
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Oct 2026to 31 May 2028
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 to 39 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Ages 18-39 years old, inclusive.
- Radiographic evidence of a recurrent/progressive brain tumor.
- Tumor must be predominantly in an intraparenchymal location.
- Deemed operable (able to be resected or have an open or stereotactic needle biopsy) by treating neurosurgeon.
- ECOG Performance Status of ≥ 2. Patients who are unable to walk because of paralysis but who are up in a wheelchair will be considered ambulatory for the purposes of the performance score.
- Bone Marrow:
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- ANC (Absolute neutrophil count) ≥ 1000/µl (unsupported).
- Platelets ≥ 100,000/µl (may be supported by transfusion).
- Hemoglobin > 8 g/dL (may be supported by transfusion).
- Renal:
- Serum creatinine ≤ upper limit of institutional normal.
- Hepatic:
- Bilirubin ≤ 1.5 times upper limit of normal for age.
- ALT (SGPT) ≤ 3 times institutional upper limit of normal for age.
- AST (SGOT) ≤ 3 times institutional upper limit of normal for age.
- Ability to understand and willingness to sign an IRB approved written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants. All patients and/or their parents or legal guardians must sign an IRB approved written informed consent document.
Who cannot join
- Tumors suspected to be pituitary tumors or tumors of the meninges.
- Tumors that are suspected of a non-CNS primary location or history of non-CNS malignancy).
- Unable to take tablets orally
- Pregnant and/or breastfeeding. Subjects of childbearing potential must have a negative serum or urine pregnancy test within 10 days prior to Day 1.
- Active infection requiring treatment or an unexplained febrile (> 101.5o F) illness.
- Known immunosuppressive disease or human immunodeficiency virus infection.
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- Any active renal, cardiac (congestive cardiac failure, myocardial infarction, myocarditis), or pulmonary disease.
- Any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction).
- Inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Washington University School of Medicine/St. Louis Children's Hospital
St Louis, Missouri, 63110
Eric Thompson, M.D., 314-454-4707, pedshemonctrialreferral@wustl.edu
Eric Thompson, M.D.
About this study
Official title: An Early Phase 1 Study of Asciminib With or Without Sildenafil for Brain Tumors
Dissemination of medulloblastoma is an independent risk factor of poor prognosis. Dissemination of medulloblastoma at recurrence is nearly universally fatal. ABL1 and 2 have been recently found to mediate the dissemination of medulloblastoma. Genetically inactivating ABL1 and 2 resulted in decreased leptomeningeal medulloblastoma and improved overall survival (OS) in rodent models. ABL kinases have also been shown to play a role in the malignant properties of glioblastoma. Asciminib is an FDA approved for the treatment of chronic myeloid leukemia and is well tolerated, likely due to its specificity for ABL1 and ABL2. Asciminib is a P-glycoprotein (P-gp) substrate and thus may be susceptible to being pumped out of tumor cells and brain endothelial cells. It is unclear if asciminib can enter the central nervous system (CNS) and brain tumors in adequate concentration to have anti-tumor effects.
- Conditions
- Brain Tumor
- Being tested
- Asciminib, Sildenafil, Surgical resection or biopsy
- Keywords
- Asciminib, Sildenafil, ABL1, ABL2, Tyrosine kinase inhibitor, Pediatric brain tumor, Brain tumor, Pharmacokinetics, Pharmacodynamics
- Medical subject headings
- Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AsciminibDrug
Commercially available stock
SildenafilDrug
Commercially available stock
Surgical resection or biopsyProcedure or surgery
Standard of care
| Study group | Role | What they receive |
|---|---|---|
| Group A: Asciminib | Experimental | Patients will receive two doses of 40 mg oral asciminib taken 12 hours apart. Surgery or biopsy should occur approximately 3 hours after last dose of asciminib. Receives: Drug: Asciminib; Procedure: Surgical resection or biopsy |
| Group B: Asciminib + Sildenafil | Experimental | Patients will receive two doses of 40 mg oral asciminib taken 12 hours apart plus two doses of 20 mg oral sildenafil taken 12 hours apart. Surgery or biopsy should occur approximately 3 hours after last dose of asciminib and sildenafil. Receives: Drug: Asciminib; Drug: Sildenafil; Procedure: Surgical resection or biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tumor:plasma ratio of asciminib
Measured: At time of surgical resection or biopsy (day 1)
Tumor:plasma ratio of asciminib with sildenafil
Measured: At time of surgical resection or biopsy (day 1)
Secondary outcomes (4)
Change in plasma levels of asciminib
Measured: Baseline, time of tumor resection/biopsy (day 1), and 8 (+/- 4 hours) after surgical resection or biopsy
Expression of c-MYC in brain tumor specimens
Measured: At time of surgical resection or biopsy (day 1)
Expression of p-CRKL in brain tumor specimens
Measured: At time of surgical resection or biopsy (day 1)
Proportion of patients with unacceptable toxicity
Measured: From start of treatment (day 1) through 3 weeks following asciminib
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2025
- Study start31 Oct 2026 (estimated)
- Primary completion30 Apr 2028 (estimated)
- Study completion31 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brain TumorStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Eric Thompson, M.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07039760
- Sponsor's protocol ID
- 202509139
- Status last verified
- Sep 2026
- First submitted
- 17 Jun 2025
- Last updated
- 11 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Early Phase 1 Study of Asciminib With or Without Sildenafil for Brain Tumors
- ClinicalTrials.gov ID
- NCT07039760
- Acronym / sponsor ID
- 202509139
- Conditions
- Brain Tumor
- Keywords
- Asciminib, Sildenafil, ABL1, ABL2, Tyrosine kinase inhibitor, Pediatric brain tumor, Brain tumor, Pharmacokinetics
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- 31 Oct 2026 (estimated)
- Primary completion
- 30 Apr 2028 (estimated)
- Study completion
- 31 May 2028 (estimated)
- First posted
- 26 Jun 2025 (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.
Dissemination of medulloblastoma is an independent risk factor of poor prognosis. Dissemination of medulloblastoma at recurrence is nearly universally fatal. ABL1 and 2 have been recently found to mediate the dissemination of medulloblastoma. Genetically inactivating ABL1 and 2 resulted in decreased leptomeningeal medulloblastoma and improved overall survival (OS) in rodent models. ABL kinases have also been shown to play a role in the malignant properties of glioblastoma. Asciminib is an FDA approved for the treatment of chronic myeloid leukemia and is well tolerated, likely due to its specificity for ABL1 and ABL2. Asciminib is a P-glycoprotein (P-gp) substrate and thus may be susceptible to being pumped out of tumor cells and brain endothelial cells. It is unclear if asciminib can enter the central nervous system (CNS) and brain tumors in adequate concentration to have anti-tumor effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A: Asciminib | Experimental | Drug: Asciminib, Procedure: Surgical resection or biopsyPatients will receive two doses of 40 mg oral asciminib taken 12 hours apart. Surgery or biopsy should occur approximately 3 hours after last dose of asciminib. |
| Group B: Asciminib + Sildenafil | Experimental | Drug: Asciminib, Drug: Sildenafil, Procedure: Surgical resection or biopsyPatients will receive two doses of 40 mg oral asciminib taken 12 hours apart plus two doses of 20 mg oral sildenafil taken 12 hours apart. Surgery or biopsy should occur approximately 3 hours after last dose of asciminib and sildenafil. |
Who may qualify
- Ages
- 18 to 39 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (16 listed)
- Ages 18-39 years old, inclusive.
- Radiographic evidence of a recurrent/progressive brain tumor.
- Tumor must be predominantly in an intraparenchymal location.
- Deemed operable (able to be resected or have an open or stereotactic needle biopsy) by treating neurosurgeon.
- ECOG Performance Status of ≥ 2. Patients who are unable to walk because of paralysis but who are up in a wheelchair will be considered ambulatory for the purposes of the performance score.
Key exclusion criteria (9 listed)
- Tumors suspected to be pituitary tumors or tumors of the meninges.
- Tumors that are suspected of a non-CNS primary location or history of non-CNS malignancy).
- Unable to take tablets orally
- Pregnant and/or breastfeeding. Subjects of childbearing potential must have a negative serum or urine pregnancy test within 10 days prior to Day 1.
- Active infection requiring treatment or an unexplained febrile (> 101.5o F) illness.
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 |
|---|---|---|
| AsciminibCommercially available stock | Drug | Group A: Asciminib, Group B: Asciminib + Sildenafil |
| SildenafilCommercially available stock | Drug | Group B: Asciminib + Sildenafil |
| Surgical resection or biopsyStandard of care | Procedure or surgery | Group A: Asciminib, Group B: Asciminib + Sildenafil |
Primary outcome
Tumor:plasma ratio of asciminib
Time frame: At time of surgical resection or biopsy (day 1)
Tumor:plasma ratio of asciminib with sildenafil
Time frame: At time of surgical resection or biopsy (day 1)
4 secondary outcomes
Change in plasma levels of asciminib
Time frame: Baseline, time of tumor resection/biopsy (day 1), and 8 (+/- 4 hours) after surgical resection or biopsy
Expression of c-MYC in brain tumor specimens
Time frame: At time of surgical resection or biopsy (day 1)
Expression of p-CRKL in brain tumor specimens
Time frame: At time of surgical resection or biopsy (day 1)
Proportion of patients with unacceptable toxicity
Time frame: From start of treatment (day 1) through 3 weeks following asciminib
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2026.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- 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.
Eric Thompson, M.D. principal investigator
Washington University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Asciminib, Sildenafil and Surgical resection or biopsy in people with Brain Tumor, run by Washington University School of Medicine at 1 site 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 16 inclusion and 9 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: opening soon, as of the sponsor's last update on 11 Sep 2026. 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 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Eric Thompson, M.D. (314-454-4707, pedshemonctrialreferral@wustl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tumor:plasma ratio of asciminib (time frame: At time of surgical resection or biopsy (day 1)). The study lists 2 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 11 Sep 2026.