Clinical trial NCT04315064: Infusion of Panobinostat (MTX110) Into the Fourth Ventricle in Children and Adults with Recurrent Medulloblastoma
Official title: Infusion of Panobinostat (MTX110) Into the Fourth Ventricle or Tumor Resection Cavity in Children and Adults with Recurrent Medulloblastoma: a Pilot Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Medulloblastoma
Keywords: medulloblastoma
Malignant Neoplasms, Brain
MTX110
panobinostat
Antineoplastic
Lead sponsor: The University of Texas Health Science Center, Houston
Sponsor class: OTHER
Collaborators: name: Midatech Pharma plc
First posted: 2020-03-19
Enrollment: 2
Sex: ALL
Minimum age (years): 1
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to establish the safety of infusions of panobinostat (MTX110) into the fourth ventricle of the brain or tumor resection cavity in patients with recurrent medulloblastoma and to assess the antitumor activity of simultaneous infusions of panobinostat (MTX110) into the fourth ventricle of the brain or resection cavity in patients with recurrent medulloblastoma based upon MRI scans and lumbar cerebrospinal fluid (CSF) cytology.


Eligibility criteria: 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 fourth ventricle or posterior fossa tumor cavity attached to a ventricular access device or agreement to have one placed.
* A minimum of 7 days between last dose of systemic chemotherapy and/or radiation therapy and first infusion of chemotherapy into fourth ventricle
* Life expectancy of at least 12 weeks in the opinion of the principal investigator
* Lansky score of 50 or greater if ≤16 years of age or Karnofsky score of 50 or greater if \> 16 years of age
* Existing neurological deficits must have been stable for a minimum of 1 week prior to study enrollment
* Patients must have recovered from the acute toxic effects of all prior anticancer chemotherapy
* Adequate bone marrow function defined by peripheral absolute neutrophil count (ANC) ≥ 500/µL, platelet count ≥ 50,000/µL (transfusion independent), and hemoglobin ≥ 9.0 gm/dL (may receive red blood cells(RBC) transfusions)
* Patient or patient's legal representative, parent(s), or guardian able to provide written informed consent.
* Patient with prolonged QT interval on screening EKG will need cardiology consultation prior to enrollment
* Patients with abnormal liver function tests (ALT, Aspartate Aminotransferase(AST),or total bilirubin) will need gastroenterology consultation prior to enrollment

Exclusion Criteria:

* Enrolled in another treatment protocol
* Has received another investigational or chemotherapy agent or radiation therapy within 7 days prior to intraventricular chemotherapy infusions
* Evidence of untreated infection
* Pregnant or lactating women

Primary outcomes:
measure: Number of participants with grade 3 through grade 5 new neurological adverse events that are related to study drug
timeFrame: 4 months post intervention
Secondary outcomes:
measure: Change in disease progression as measured by an MRI
timeFrame: prior to first infusion; after last infusion (last infusion is about 6 weeks after the first infusion)
measure: Change in disease progression as measured by lumbar CSF cytology
timeFrame: prior to first infusion; after last infusion (last infusion is about 6 weeks after the first infusion)
Other outcomes:
Locations:
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
Study officials:
affiliation: The University of Texas Health Science Center, Houston
name: David IIan Sandberg, MD
role: PRINCIPAL_INVESTIGATOR
References: