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Treatment with MTX110

Phase 1

Medulloblastoma | Unknown | Oncology |Biodexa Pharmaceuticals plc|Last Updated: Mar 10, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMC
Total Trials1
Total Enrollment2
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04315064Infusion of Panobinostat (MTX110) Into the Fourth Ventricle in Children and Adults with Recurrent MedulloblastomaEARLY_PHASE1 COMPLETED 2Apr 21, 2020May 2, 2023Mar 10, 20251 United States
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Study Endpoints
Primary Endpoints
Number of participants with grade 3 through grade 5 new neurological adverse events that are related to study drug
4 months post intervention

Anew neurological deficit will be defined as new cranial neuropathy, change in level of consciousness, motor weakness, gait change or cerebellar finding (ataxia, dysmetria, dysdiadochokinesis) and will be graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 4.0

Secondary Endpoints
Change in disease progression as measured by an MRI
prior to first infusion; after last infusion (last infusion is about 6 weeks after the first infusion)
Change in disease progression as measured by lumbar CSF cytology
prior to first infusion; after last infusion (last infusion is about 6 weeks after the first infusion)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Treatment with MTX110EXPERIMENTAL -
Interventions
NameTypeDescription
Treatment with MTX110DRUGPatients will undergo surgery for maximum safe tumor resection with simultaneous placement of a ventricular access device (VAD) into the fourth ventricle or posterior fossa tumor resection cavity.The specific ventricular access device that will be placed is Medtronic Reference number 44102, "Medtronic CSF-Ventricular Reservoir Side Inlet, 18 mm.If a patient already has a ventricular access device in place and additional tumor resection is not required, then that patient can proceed with intraventricular chemotherapy infusions after study enrollment. If the patient has metastatic disease in the brain and/or spine without resectable tumor in the posterior fossa, then only catheter and ventricular access device placement will be performed.Enrolled patients will receive 4 infusions per week of panobinostat (MTX110; 0.25 ml of 300 μM = 26.2 μg), per infusion into the ventricular access device for 6 consecutive weeks for a total of 24 infusions.
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Eligibility Criteria
Age Range1 Year — 80 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Diagnosis: Patients with histologically verified medulloblastoma with recurrence or progression involving anywhere in the brain and/or spine. * Patient must have either measurable or evaluable tumor as assessed by MRI of the brain and total spine * An implanted catheter in the...

Countries:United States
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