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Asciminib With or Without Sildenafil for Brain Tumors

ConditionBrain Tumor
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-39
SponsorWashington University School of Medicine

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Asciminib

    Drug

    Commercially available stock

  • Sildenafil

    Drug

    Commercially available stock

  • Surgical resection or biopsy

    Procedure/Surgery

    Standard of care

Treatment groups

12 Participants
are divided into 2 treatment groups
Group A: Group A: AsciminibExperimental treatment 2 interventions
Group B: Group B: Asciminib + SildenafilExperimental treatment 3 interventions

Trial outcomes

Primary outcomes

1

Tumor:plasma ratio of asciminib

Time frame
At time of surgical resection or biopsy (day 1)
2

Tumor:plasma ratio of asciminib with sildenafil

Time frame
At time of surgical resection or biopsy (day 1)

Secondary outcomes

1

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
2

Expression of c-MYC in brain tumor specimens

Time frame
At time of surgical resection or biopsy (day 1)
3

Expression of p-CRKL in brain tumor specimens

Time frame
At time of surgical resection or biopsy (day 1)
4

Proportion of patients with unacceptable toxicity

Time frame
From start of treatment (day 1) through 3 weeks following asciminib

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Washington University School of Medicine

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: