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Infusion of Panobinostat (MTX110) Into the Fourth Ventricle in Children and Adults With Recurrent Medulloblastoma

Primary Purpose

Medulloblastoma

Status

Recruiting

Phase

Early Phase 1

Locations

United States

Study Type

Interventional

Intervention

Treatment with MTX110

About this trial

This is an interventional treatment trial for Medulloblastoma focused on measuring medulloblastoma, Malignant Neoplasms, Brain, MTX110, panobinostat, Antineoplastic

Eligibility Criteria

1 Year - 80 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

The University of Texas Health Science Center at HoustonRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment with MTX110

Arm Description

Outcomes

Primary Outcome Measures

Number of participants with grade 3 through grade 5 new neurological adverse events that are related to study drug

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 Outcome Measures

Change in disease progression as measured by an MRI

Change in disease progression as measured by lumbar CSF cytology

Full Information

NCT ID

NCT04315064

First Posted

March 17, 2020

Last Updated

June 20, 2023

Sponsor

The University of Texas Health Science Center, Houston

Collaborators

Midatech Pharma plc

1. Study Identification

Unique Protocol Identification Number

NCT04315064

Brief Title

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

2. Study Status

Record Verification Date

June 2023

Overall Recruitment Status

Recruiting

Study Start Date

April 21, 2020 (Actual)

Primary Completion Date

December 30, 2025 (Anticipated)

Study Completion Date

December 30, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

The University of Texas Health Science Center, Houston

Collaborators

Midatech Pharma plc

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Medulloblastoma

Keywords

medulloblastoma, Malignant Neoplasms, Brain, MTX110, panobinostat, Antineoplastic

7. Study Design

Primary Purpose

Treatment

Study Phase

Early Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Treatment with MTX110

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Treatment with MTX110

Intervention Description

Patients 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.

Primary Outcome Measure Information:

Title

Number of participants with grade 3 through grade 5 new neurological adverse events that are related to study drug

Description

Time Frame

4 months post intervention

Secondary Outcome Measure Information:

Title

Change in disease progression as measured by an MRI

Description

Change in disease progression as measured by an MRI

Time Frame

prior to first infusion; after last infusion (last infusion is about 6 weeks after the first infusion)

Title

Change in disease progression as measured by lumbar CSF cytology

Description

Change in disease progression as measured by lumbar CSF cytology

Time Frame

prior to first infusion; after last infusion (last infusion is about 6 weeks after the first infusion)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

David IIan Sandberg, MD

Phone

(713) 500-7370

David.I.Sandberg@uth.tmc.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Bangning Yu

Phone

(713) 500-7363

Bangning.Yu@uth.tmc.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

David IIan Sandberg, MD

Organizational Affiliation

The University of Texas Health Science Center, Houston

Official's Role

Principal Investigator

Facility Information:

Facility Name

The University of Texas Health Science Center at Houston

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

David Sandberg, MD

Phone

713-500-7370

David.I.Sandberg@uth.tmc.edu

First Name & Middle Initial & Last Name & Degree

Bangning yu

Phone

(713) 500-7363

Bangning.Yu@uth.tmc.edu

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Infusion of Panobinostat (MTX110) Into the Fourth Ventricle in Children and Adults With Recurrent Medulloblastoma

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